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The schedule for this year's International Papillomavirus Conference in Berlin Germany has been finalized. My presentation, HPV - The Patient Experience is the first in the experience of the conference given by a patient and I commend the organizers of this conference in recognizing the need to include the voice of those most affected by HPV - the patients.
It is my hope that this will set a new standard by which future conferences will continue to include the voice of the patient. There is much that can be learned from the patient's input however for too long the medical community has not appreciated that they could learn anything useful from them aside from the results provided regarding physical research. But HPV is not solely a physical condition but a psychological one as well.
In finalizing my presentation I realize just how much of the psychological aspects of an HPV diagnosis is relevant to the development of guidelines currently only focused on scientific physical evidence. While this is certainly a significant factor, the psychological aspect simply cannot be ignored if the patient is going to be treated as a whole.
We are not just physical nor just psychological beings but a delicate combination of both. It is important for me to dispel the myth that providing the patient with information will only result in hysteria and upset. In my experience it is exactly the opposite. It is that lack of education of the patient which results in psychological distress. The feelings of not only the unknown but the loss of control over their bodies as well The loss of control is especially true in the case of persistent disease in which the patient cannot in many instances effectuate a change in the progression of precancerous lesions. Younger individuals, once feeling invincible with the world ahead of them are now fearful and concerned about the potential for loss of future fertility and possibly death - something which, because of their age they typically would never have had to face if not for HPV.
It is also important that women not continue to have to bear the burden of HPV diagnosis themselves and a test for men must be developed. In addition the development of a therapeutic vaccine for those already infected with the virus is paramount. The percentage of individuals who actually complete the three doses of the current vaccine in the US is only 30% and without a widespread public education campaign far more individuals will become infected than those protected.
This will certainly prove to be a precedent setting conference.
13 Ekim 2012 Cumartesi
Is Cancer Contagious? The ACS Says No, but is This the Truth?
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In an article by the American Cancer Society titled “Is Cancer Contagious”, the comment is emphatically made that it is not. It goes on to say,
“If cancer were contagious, we would have cancer epidemics just as we have flu epidemics -- cancer would spread like measles, polio, or the common cold. We would expect a high rate of cancer among the families and friends of cancer patients and among health professionals to reflect their exposure to the disease. This is not the case.”
In the case of the human papillomavirus I would have to disagree. HPV is known to cause numerous cancers including cervical, vaginal, vulvar, anal and oral, as well as penile. Its main method of transmission is through intimate skin-to-skin contact. It does not require intercourse however this only increased the chances of contracting the virus especially for women through small tears or abrasions in the vagina during intercourse.
Family members don’t have intimate relations with each other and they don’t typically have them with their friends either. And it’s obvious that they aren’t having intimate relations with their physicians. So the theory as related in this article borders on nothing but the absurd.
While HPV does not transmit cancer in the literal sense of the word, it does introduce the precursors known, under the right conditions, to cause cancer. We know that HPV is a sexually transmitted disease and can also be transmitted through oral sex and some have indicated that kissing is not beyond the realm of possibilities as well as through foamites (inanimate objects, fingers, sex toys etc).
The article goes on to state:
“There is no evidence that close contact or things like sex, kissing, touching …… can spread cancer from one person to another.” On the contrary, more and more research is confirming that transmission of HPV is far more complex and involves multiple modes of transmission and one is certainly more likely to develop one of these cancers if they have been exposed to HPV than not.
Today, 99.9% of cervical cancers are the result of HPV and over 90% of anal cancers. Oral cancers continue to rise, particularly in younger men, at a staggering rate which has already exceeded the number of cervical cancers on an annual basis.
As with other HPV related cancers, it is not a difficulty in diagnosing these cancers that are the problem, but a lack of education on the part of physicians and the lack of public awareness and early screening opportunities.
Cancer in the broader sense of the conversation, or shall I say the potential for cancer, has shown to be very much contagious as it relates to HPV. The Hepatitis viruses have long been known to result in liver and pancreatic cancers. Perhaps we may ultimately find that all cancers are the result of various viruses, but until then, perhaps the ACS should take a bit more pragmatic view on its current position.
" Is Cancer Contagious? ." American Cancer Society :: Information and Resources for Cancer: Breast, Colon, Prostate, Lung and Other Forms. N.p., n.d. Web. 28 Aug. 2011. http://www.cancer.org/Cancer/CancerBasics/is-cancer-contagious
"Hepatitis B Foundation: Hepatitis B and Primary Liver Cancer." Hepatitis B Foundation. N.p., n.d. Web. 28 Aug. 2011. http://www.hepb.org/professionals
The Oral Cancer Foundation. Web.28 Aug. 2011. http://www.oralcancerfoundation.com/
“If cancer were contagious, we would have cancer epidemics just as we have flu epidemics -- cancer would spread like measles, polio, or the common cold. We would expect a high rate of cancer among the families and friends of cancer patients and among health professionals to reflect their exposure to the disease. This is not the case.”
In the case of the human papillomavirus I would have to disagree. HPV is known to cause numerous cancers including cervical, vaginal, vulvar, anal and oral, as well as penile. Its main method of transmission is through intimate skin-to-skin contact. It does not require intercourse however this only increased the chances of contracting the virus especially for women through small tears or abrasions in the vagina during intercourse.
Family members don’t have intimate relations with each other and they don’t typically have them with their friends either. And it’s obvious that they aren’t having intimate relations with their physicians. So the theory as related in this article borders on nothing but the absurd.
While HPV does not transmit cancer in the literal sense of the word, it does introduce the precursors known, under the right conditions, to cause cancer. We know that HPV is a sexually transmitted disease and can also be transmitted through oral sex and some have indicated that kissing is not beyond the realm of possibilities as well as through foamites (inanimate objects, fingers, sex toys etc).
The article goes on to state:
“There is no evidence that close contact or things like sex, kissing, touching …… can spread cancer from one person to another.” On the contrary, more and more research is confirming that transmission of HPV is far more complex and involves multiple modes of transmission and one is certainly more likely to develop one of these cancers if they have been exposed to HPV than not.
Today, 99.9% of cervical cancers are the result of HPV and over 90% of anal cancers. Oral cancers continue to rise, particularly in younger men, at a staggering rate which has already exceeded the number of cervical cancers on an annual basis.
As with other HPV related cancers, it is not a difficulty in diagnosing these cancers that are the problem, but a lack of education on the part of physicians and the lack of public awareness and early screening opportunities.
Cancer in the broader sense of the conversation, or shall I say the potential for cancer, has shown to be very much contagious as it relates to HPV. The Hepatitis viruses have long been known to result in liver and pancreatic cancers. Perhaps we may ultimately find that all cancers are the result of various viruses, but until then, perhaps the ACS should take a bit more pragmatic view on its current position.
" Is Cancer Contagious? ." American Cancer Society :: Information and Resources for Cancer: Breast, Colon, Prostate, Lung and Other Forms. N.p., n.d. Web. 28 Aug. 2011. http://www.cancer.org/Cancer/CancerBasics/is-cancer-contagious
"Hepatitis B Foundation: Hepatitis B and Primary Liver Cancer." Hepatitis B Foundation. N.p., n.d. Web. 28 Aug. 2011. http://www.hepb.org/professionals
The Oral Cancer Foundation. Web.28 Aug. 2011. http://www.oralcancerfoundation.com/
FDA Approves New Cobas High Risk HPV Test
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On April 20, 2011, Roche Diagnostics announced that the FDA has approved its new HPV test, cobas. This new test detects the presence of DNA for fourteen high risk strains of the virus. There are a few differences between the Roche test and the current digene HPV test. Roche’s test will check for fourteen of the high risk HPV strains while the digene test checks for thirteen. Once this is completed, the Roche test will then concurrently genotype the specimen for types 16 and 18. These two HPV strains re shown to cause approximately 70 percent of all cervical cancers.
Roche representatives, Christoph Majewski , Head of HPV molecular science and Dr. Catherine Behrens, Director of Medical Affairs of the women involved in the ATHENA study with a Pap result of ASCUS (atypical squamous cells of undetermined significance) 90 percent were shown on biopsy to have CIN2/3 lesions. Of those, 46 percent were CIN3.
The National Cancer Institute indicates that 10 percent of women who are positive for HPV types 16 and 18 have been shown to develop CIN3 (cervical intraepithelial neoplasia grade 3) within three years. CIN3/CIS (carcinoma in situ) is the most severe type of abnormality prior to the lesion becoming an invasive cancer.
The ATHENA study was utilized to determine the effectiveness of this new test, cobas. The study was conducted throughout the United States and involved a total of 47,000 women. It showed that one in ten women who tested positive for types 16 and 18 genotyping had precancerous lesions despite having a negative result on their Pap smears.
The current digene HPV test in existence for over ten years does not, at this point, genotype within its 13 panel assay. It has however since its inception performed over forty million tests and had over three-hundred pier review articles with respect to its methods.
Cervista*, the HPV test from Hologic, approved by the FDA, like Roche, tests for fourteen high risk strains. While Cervista* HR does not concurrently genotype for 16 and 18 as cobas does, they do offer a separate test to genotype for both 16 and 18.
It may seem more convenient to have all testing done relatively simultaneously in one HPV test. However, since much of the cost of testing is ultimately determined by the laboratories performing them, it will be interesting to see just which test will take the lead.
*This blog has been changed (with our apologies) to correctly reflect the name of the Hologic HPV test which is Cervista and not Cervarix as previously mentioned. Cervarix, along with Gardasil is one of the two FDA approved HPV vaccines.
Roche representatives, Christoph Majewski , Head of HPV molecular science and Dr. Catherine Behrens, Director of Medical Affairs of the women involved in the ATHENA study with a Pap result of ASCUS (atypical squamous cells of undetermined significance) 90 percent were shown on biopsy to have CIN2/3 lesions. Of those, 46 percent were CIN3.
The National Cancer Institute indicates that 10 percent of women who are positive for HPV types 16 and 18 have been shown to develop CIN3 (cervical intraepithelial neoplasia grade 3) within three years. CIN3/CIS (carcinoma in situ) is the most severe type of abnormality prior to the lesion becoming an invasive cancer.
The ATHENA study was utilized to determine the effectiveness of this new test, cobas. The study was conducted throughout the United States and involved a total of 47,000 women. It showed that one in ten women who tested positive for types 16 and 18 genotyping had precancerous lesions despite having a negative result on their Pap smears.
The current digene HPV test in existence for over ten years does not, at this point, genotype within its 13 panel assay. It has however since its inception performed over forty million tests and had over three-hundred pier review articles with respect to its methods.
Cervista*, the HPV test from Hologic, approved by the FDA, like Roche, tests for fourteen high risk strains. While Cervista* HR does not concurrently genotype for 16 and 18 as cobas does, they do offer a separate test to genotype for both 16 and 18.
It may seem more convenient to have all testing done relatively simultaneously in one HPV test. However, since much of the cost of testing is ultimately determined by the laboratories performing them, it will be interesting to see just which test will take the lead.
*This blog has been changed (with our apologies) to correctly reflect the name of the Hologic HPV test which is Cervista and not Cervarix as previously mentioned. Cervarix, along with Gardasil is one of the two FDA approved HPV vaccines.
Human Genome Sciences Gains FDA Recommendation for Benlysta. Shares Soar 10% in Afterhours Trading
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FDA panel favors lupus drug from Human Genome Sciences despite mixed results in studies
By: MATTHEW PERRONE
Associated Press
11/16/10 7:00 PM EST ADELPHI, MD. — Federal health experts on Tuesday voted in favor of the first new drug to treat Lupus in over 50 years, setting aside concerns that the experimental therapy does not work in some key patient groups, including African-Americans.
The recommendation from a panel of Food and Drug Administration advisers brings the biotech drug from Human Genome Sciences one step closer to market. The drug was co-developed with GlaxoSmithKline PLC.
Known as Benlysta, the drug is designed to treat flare-ups and pain caused by lupus, a little-understood and potentially fatal ailment in which the body attacks its own tissue and organs. Ninety percent of lupus patients are women. The disease causes skin rashes, joint pain and inflammation of the kidneys and the fibrous tissue surrounding the heart.
The FDA panel voted 13-2 in favor of approving the drug. The agency is not required to follow the advice of its panelists, though it often does.
The FDA is scheduled to make a decision on Benlysta by Dec. 9, and company executives suggested the drug could be available in the first quarter 2011.
Shares of Human Genome Sciences rose $2.41, or 9.3 percent, to $28.29 in after-hours trading, while GlaxoSmithKline stock gained 94 cents, or 2.4 percent, to $39.48.
Throughout Tuesday's meeting, FDA's experts stressed that Benlysta will not work for all patients and that more effective therapies are still needed.
"I think it's very exciting that this is the first new drug in five decades, but it's not magic bullet," said Dr. Maria Suarez-Almazor of the MD Anderson Cancer Center in Houston.
Panelists who voted against the drug's approval said the studies submitted by Human Genome Sciences did not show that patients taking Benlysta fared significantly better than those taking older therapies.
"I have all the hopes and dreams that we have heard expressed today, and I know my lupus patients want a medicine that is more effective, but I have not seen data that says this drug is it," said Dr. David Blumenthal of Case Western Reserve University.
Only 30 percent of patients studied by Human Genome Sciences responded to the drug, as measured by a comprehensive list of lupus symptoms. The drug did not help African-Americans — who are three times more likely to have lupus than Caucasians. Additionally, studies conducted in Latin America and Eastern Europe showed significantly better results than those conducted in the U.S. and Canada, leading some panelists to question the accuracy of the results.
Despite such concerns, panelists appeared swayed by more than 30 lupus patients — several of whom currently take Benlysta — who urged the FDA to approve the drug during a public comment session. Many of the patients said the side effects of current drugs are as debilitating as the disease itself. Current treatments include steroids, which can cause bone fractures, and chemotherapy drugs, which cause hair loss, anemia and diarrhea.
The drugs help keep the immune system in check, but aren't designed to treat lupus itself.
"Living with lupus is a daily fight simply to see another day, and often survival means letting go of things you love and wanted to do," said Wendy Rogers, a spokeswoman for the Lupus Foundation of America.
In separate votes the panel voted 10-5 in favor of the drug's effectiveness and 14-1 in favor of its safety.
"I found the personal stories today particularly compelling, and I see this as an important opportunity to advance a challenging area of medicine and serve a previously underserved population," said Dr. Robert Kerns of the Veterans Affairs Connecticut Healthcare System.
An estimated 1.5 million people in the U.S. have lupus. Benlysta, known chemically as belimumab, is a once-a-month infusion that works by blocking the antibodies that cause the disease in a subset of patients.
If approved, analysts estimate Benlysta could eventually post sales of over $2 billion annually. Human Genome Sciences Inc., of Rockville, Md., and GlaxoSmithKline PLC, of Britain, which would split the profits, are also seeking approval to market the injectable drug in Europe.
Human Genome Sciences and GlaxoSmithKline Announce Vote of FDA Advisory Committee to Recommend Approval of BENLYSTA(R) for Systemic Lupus Erythematosus
ROCKVILLE, Md., & LONDON, Nov 16, 2010 (BUSINESS WIRE) -- Human Genome Sciences, Inc. (HGSI 28.60, +2.72, +10.51%) and GlaxoSmithKline PLC (GSK) today announced that the Arthritis Advisory Committee of the U.S. Food and Drug Administration (FDA) has voted 13 to 2 to recommend that the FDA approve BENLYSTA(R) (belimumab) for the treatment of autoantibody-positive patients with active systemic lupus erythematosus (SLE).
The FDA Arthritis Advisory Committee is convened to provide the FDA with independent expert advice on a broad range of issues related to rheumatology drug products. The committee provides non-binding recommendations for consideration by FDA, with the final decision on approval made by FDA. The FDA has assigned BENLYSTA a Prescription Drug User Fee Act target date of December 9, 2010.
Belimumab is an investigational drug and the first in a new class of drugs called BLyS-specific inhibitors. HGS and GSK are developing belimumab under a definitive co-development and co-commercialization agreement entered into in 2006. Under the agreement, HGS has responsibility for conducting the belimumab Phase 3 trials, with assistance from GSK. The companies will share equally in Phase 3/4 development costs, sales and marketing expenses, and profits of any product commercialized under the current agreement.
ABOUT GLAXOSMITHKLINE
GlaxoSmithKline -- one of the world's leading research-based pharmaceutical and healthcare companies -- is committed to improving the quality of human life by enabling people to do more, feel better and live longer. For further information please visit www.gsk.com. GSK Biopharm R&D is employing novel approaches to harness the therapeutic potential of biopharmaceuticals for the benefit of patients with serious autoimmune disease.
ABOUT HUMAN GENOME SCIENCES
The mission of HGS is to apply great science and great medicine to bring innovative drugs to patients with unmet medical needs.
For more information about HGS, please visit the Company's web site at http://www.hgsi.com/. Health professionals and patients interested in clinical trials of HGS products may inquire via e-mail to medinfo@hgsi.com or by calling HGS at (877) 822-8472.
Related Articles
Good news today for Human Genome Sciences and those who suffer from Lupus. FDA voted in favor of Benlysta with a strong vote of 13-2. Although the FDA doesn't always approve drugs that are recommended by the panel, many times they do, and more so with an Approval Recommendation than without one. Shares of Human Genome Sciences were up 10.51% to 28.60 in afterhours trading.
Trading was halted throughout the day and will resume normal trading tomorrow. I expect a strong day tomorrow and it should be watched closely tomorrow. It's partner Glaxo SmithKline was trading slightly higher at 39.45 +0.91 a 2.36% gain. This is really good news for investors who have watched HGSI drop from a high of 30.18 in September to a low of 23.60 this past Friday from a Doctor scandal, a CRL for Zalbin, and safety concerns from their Phase III clinical trial results for Benlysta.
It did report positive 3Q 2010 results in the following press release, Human Genome Sciences Announces Third Quarter 2010 Financial Results and Key Developments. I see HGSI stock to rise towards the 32-35 mark with possible Full Approval coming December 9th.
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FDA panel favors lupus drug from Human Genome Sciences despite mixed results in studies
By: MATTHEW PERRONE
Associated Press
11/16/10 7:00 PM EST ADELPHI, MD. — Federal health experts on Tuesday voted in favor of the first new drug to treat Lupus in over 50 years, setting aside concerns that the experimental therapy does not work in some key patient groups, including African-Americans.
The recommendation from a panel of Food and Drug Administration advisers brings the biotech drug from Human Genome Sciences one step closer to market. The drug was co-developed with GlaxoSmithKline PLC.
Known as Benlysta, the drug is designed to treat flare-ups and pain caused by lupus, a little-understood and potentially fatal ailment in which the body attacks its own tissue and organs. Ninety percent of lupus patients are women. The disease causes skin rashes, joint pain and inflammation of the kidneys and the fibrous tissue surrounding the heart.
The FDA panel voted 13-2 in favor of approving the drug. The agency is not required to follow the advice of its panelists, though it often does.
The FDA is scheduled to make a decision on Benlysta by Dec. 9, and company executives suggested the drug could be available in the first quarter 2011.
Shares of Human Genome Sciences rose $2.41, or 9.3 percent, to $28.29 in after-hours trading, while GlaxoSmithKline stock gained 94 cents, or 2.4 percent, to $39.48.
Throughout Tuesday's meeting, FDA's experts stressed that Benlysta will not work for all patients and that more effective therapies are still needed.
"I think it's very exciting that this is the first new drug in five decades, but it's not magic bullet," said Dr. Maria Suarez-Almazor of the MD Anderson Cancer Center in Houston.
Panelists who voted against the drug's approval said the studies submitted by Human Genome Sciences did not show that patients taking Benlysta fared significantly better than those taking older therapies.
"I have all the hopes and dreams that we have heard expressed today, and I know my lupus patients want a medicine that is more effective, but I have not seen data that says this drug is it," said Dr. David Blumenthal of Case Western Reserve University.
Only 30 percent of patients studied by Human Genome Sciences responded to the drug, as measured by a comprehensive list of lupus symptoms. The drug did not help African-Americans — who are three times more likely to have lupus than Caucasians. Additionally, studies conducted in Latin America and Eastern Europe showed significantly better results than those conducted in the U.S. and Canada, leading some panelists to question the accuracy of the results.
Despite such concerns, panelists appeared swayed by more than 30 lupus patients — several of whom currently take Benlysta — who urged the FDA to approve the drug during a public comment session. Many of the patients said the side effects of current drugs are as debilitating as the disease itself. Current treatments include steroids, which can cause bone fractures, and chemotherapy drugs, which cause hair loss, anemia and diarrhea.
The drugs help keep the immune system in check, but aren't designed to treat lupus itself.
"Living with lupus is a daily fight simply to see another day, and often survival means letting go of things you love and wanted to do," said Wendy Rogers, a spokeswoman for the Lupus Foundation of America.
In separate votes the panel voted 10-5 in favor of the drug's effectiveness and 14-1 in favor of its safety.
"I found the personal stories today particularly compelling, and I see this as an important opportunity to advance a challenging area of medicine and serve a previously underserved population," said Dr. Robert Kerns of the Veterans Affairs Connecticut Healthcare System.
An estimated 1.5 million people in the U.S. have lupus. Benlysta, known chemically as belimumab, is a once-a-month infusion that works by blocking the antibodies that cause the disease in a subset of patients.
If approved, analysts estimate Benlysta could eventually post sales of over $2 billion annually. Human Genome Sciences Inc., of Rockville, Md., and GlaxoSmithKline PLC, of Britain, which would split the profits, are also seeking approval to market the injectable drug in Europe.
Human Genome Sciences and GlaxoSmithKline Announce Vote of FDA Advisory Committee to Recommend Approval of BENLYSTA(R) for Systemic Lupus Erythematosus
ROCKVILLE, Md., & LONDON, Nov 16, 2010 (BUSINESS WIRE) -- Human Genome Sciences, Inc. (HGSI 28.60, +2.72, +10.51%) and GlaxoSmithKline PLC (GSK) today announced that the Arthritis Advisory Committee of the U.S. Food and Drug Administration (FDA) has voted 13 to 2 to recommend that the FDA approve BENLYSTA(R) (belimumab) for the treatment of autoantibody-positive patients with active systemic lupus erythematosus (SLE).
The FDA Arthritis Advisory Committee is convened to provide the FDA with independent expert advice on a broad range of issues related to rheumatology drug products. The committee provides non-binding recommendations for consideration by FDA, with the final decision on approval made by FDA. The FDA has assigned BENLYSTA a Prescription Drug User Fee Act target date of December 9, 2010.
Belimumab is an investigational drug and the first in a new class of drugs called BLyS-specific inhibitors. HGS and GSK are developing belimumab under a definitive co-development and co-commercialization agreement entered into in 2006. Under the agreement, HGS has responsibility for conducting the belimumab Phase 3 trials, with assistance from GSK. The companies will share equally in Phase 3/4 development costs, sales and marketing expenses, and profits of any product commercialized under the current agreement.
ABOUT GLAXOSMITHKLINE
GlaxoSmithKline -- one of the world's leading research-based pharmaceutical and healthcare companies -- is committed to improving the quality of human life by enabling people to do more, feel better and live longer. For further information please visit www.gsk.com. GSK Biopharm R&D is employing novel approaches to harness the therapeutic potential of biopharmaceuticals for the benefit of patients with serious autoimmune disease.
ABOUT HUMAN GENOME SCIENCES
The mission of HGS is to apply great science and great medicine to bring innovative drugs to patients with unmet medical needs.
For more information about HGS, please visit the Company's web site at http://www.hgsi.com/. Health professionals and patients interested in clinical trials of HGS products may inquire via e-mail to medinfo@hgsi.com or by calling HGS at (877) 822-8472.
Related Articles
- FDA panel backs first new lupus drug in 50 years (thenewstribune.com)
- FDA Panel Backs First New Lupus Drug In 50 Years (huffingtonpost.com)
- BREAKING NEWS: HGS wins crucial panel backing for Benlysta (fiercebiotech.com)
- Benlysta Shows Promise for Lupus (lupus.webmd.com)
Pfizer's To Present Lung Cancer Data July 3-7
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Image via CrunchBase Pfizer Inc. will present early and mid-stage data from its lung cancer portfolio, including PF-00299804 (PF-299) an investigational, oral, pan-HER inhibitor;1 and crizotinib, an investigational, oral, first-in-class compound that inhibits the anaplastic lymphoma kinase, or ALK,2 at the International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), July 3-7 in Amsterdam, The Netherlands.
“While lung cancer remains a difficult-to-treat disease, we’re learning more about how therapies like crizotinib and PF-299 may be able to specifically target ALK or the HER pathway, respectively, and how this may lead to more rationally selected and personalized therapy,” said Maurizio Voi, MD, Thoracic Tumor Strategy Lead, Pfizer Oncology. “Data being presented show survival outcomes for PF-299 and crizotinib, as well as quality-of-life or patient-reported outcomes after treatment for patients with non small cell lung cancer, which represent important considerations in determining the best treatment option for these patients.”
First Presentation of PF-299 Preliminary Overall Survival Data
Continued....
Pfizer will present, for the first time, preliminary overall survival data from a Phase 2 study evaluating PF-299 vs erlotinib in patients with advanced non-small cell lung cancer (NSCLC) after progression on at least one chemotherapy regimen (oral presentation, Abstract #745, Monday, July 4).1
Pfizer also will present patient-reported outcomes (PRO) from clinical trials of PF-299 in refractory and second-/third-line NSCLC, which provide a better understanding of the patient’s perspective of the burden of adverse events associated with treatment and how it may change over time.3,4
PF-299 targets multiple receptors of the HER pathway. PF-299 is an irreversible inhibitor of HER-1 (EGFR), HER-2 and HER-4 tyrosine kinases. 6
Crizotinib Data to be presented:
At the WCLC, data on the anti-tumor activity, safety, overall survival, patient-reported and quality-of-life outcomes observed in clinical trials of Pfizer’s crizotinib will be presented.2,7,8
About Pfizer Oncology
Pfizer Oncology is committed to the discovery, investigation and development of innovative treatment options to improve the outlook for cancer patients worldwide. Our strong pipeline, one of the most robust in the industry, is studied with precise focus on identifying and translating the best scientific breakthroughs into clinical application for patients across a wide range of cancers. Pfizer Oncology has biologics and small molecules in clinical development and more than 100 clinical trials underway. By working collaboratively with academic institutions, individual researchers, cooperative research groups, governments, and licensing partners, Pfizer Oncology strives to cure or control cancer with breakthrough medicines, to deliver the right drug for each patient at the right time. For more information please visit www.Pfizer.com.
DISCLOSURE NOTICE: The information contained in this release is as of June 28, 2011. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.
This release contains forward-looking information about various oncology product candidates, including their potential benefits, that involves substantial risks and uncertainties. Such risks and uncertainties include, among other things, the uncertainties inherent in research and development; decisions by regulatory authorities regarding whether and when to approve any drug applications that have been or may be filed for any such oncology product candidates as well as their decisions regarding labeling and other matters that could affect their availability or commercial potential; and competitive developments.
A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2010 and in its reports on Form 10-Q and Form 8-K.
1 World Lung Accepted Abstract #745. Overall Survival (OS) Results of a Randomized Phase 2 Trial of PF299804 versus Erlotinib in Patients with Advanced Non-Small Cell Lung Cancer (NSCLC) After Failure of Chemotherapy. Oral Session, Monday July 4, 2011: 3:35 PM – 3:45 PM CEST. M. Boyer – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
2 World Lung Accepted Abstract #1618. Phase 2 Data for Crizotinib (PF-02341066) in ALK-Positive Advanced Non-Small Cell Lung Cancer (NSCLC): PROFILE 1005. Oral Session, Wednesday July 6, 2011: 3:10 PM – 3:20 PM CEST. G. Riely – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
3 World Lung Accepted Abstract #957. Gastrointestinal Toxicity of the Pan-HER Tyrosine Kinase Inhibitor (TKI) PF299804: Assessment by Patient-Reported Outcomes in 2nd/3rd-Line and Refractory Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. A. Campbell – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
4 World Lung Accepted Abstract #702. Dermatologic Adverse Events of the Pan-HER Tyrosine Kinase Inhibitor (TKI) PF299804: Assessment by Patient-Reported Outcomes in 2nd/3rd-line and Refractory Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. A. Campbell – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
5 Clinicaltrials.gov. ARCHER 1009: A Phase 3 Study of PF-00299804, a Pan-HER Inhibitor, Vs. Erolotinib in the Treatment of Advanced Non-Small Cell Lung Cancer. Available here: http://www.clinicaltrials.gov/ct2/show/NCT01360554?term=ARCHER&rank=1. Accessed June 21, 2011.
6 Gonzales AJ, Hook KE, Althaus IW et al. Antitumor activity and pharmacokinetic properties of PF-00299804, a second-generation irreversible pan-erbBreceptor tyrosine kinase inhibitor. Mol Cancer Ther. 2008;7:1880-89.
7 World Lung Accepted Abstract #1510. PROFILE 1005: Preliminary Patient-Reported Outcomes (PROs) from an Ongoing Phase 2 Study of Crizotinib (PF-02341066) in Anaplastic Lymphoma Kinase (ALK)-Positive Advanced Non-Small Cell Lung Cancer (NSCLC). Oral Session, Wednesday July 6, 2011: 3:30 PM – 3:40 PM CEST. F. Blackhall – Presenter. Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
8 World Lung Accepted Abstract #1207. Crizotinib improves overall survival of ALK-positive patients with advanced NSCLC compared with historical controls. Oral Session, Wednesday July 6, 2011: 3:20 PM – 3:30 PM CEST. A. Shaw – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
9 World Lung Accepted Abstract #1349. Efficacy of Crizotinib in Retrospective Comparisons with Standard-Of-Care (SOC) Regimens from Three Pfizer-Sponsored Clinical Trials in Patients with Advanced Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. Y. Tang – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
10 Bang Y et al. Clinical Activity of the Oral ALK Inhibitor, Crizotinib (PF-02341066), in Patients with ALK-positive Non-Small Cell Lung Cancer. Accepted Plenary Presentation at the American Society of Clinical Oncology Annual Meeting, June 4-8, 2010. Chicago, IL.
11 Zou HY, Li Q, Lee JH, et al. An orally available small-molecule inhibitor of c-MET,
PF-2341066, exhibits cytoreductive antitumor efficacy through antiproliferative and antiangiogenic mechanisms. Cancer Res. 2007;67:4408-4417.
12 Chiarle R, Voena C, Ambrogio C et al. The anaplastic lymphoma kinase in the pathogenesis of cancer. Nat Rev Cancer. 2008;8(1): 11-23.
Image via CrunchBase Pfizer Inc. will present early and mid-stage data from its lung cancer portfolio, including PF-00299804 (PF-299) an investigational, oral, pan-HER inhibitor;1 and crizotinib, an investigational, oral, first-in-class compound that inhibits the anaplastic lymphoma kinase, or ALK,2 at the International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), July 3-7 in Amsterdam, The Netherlands. “While lung cancer remains a difficult-to-treat disease, we’re learning more about how therapies like crizotinib and PF-299 may be able to specifically target ALK or the HER pathway, respectively, and how this may lead to more rationally selected and personalized therapy,” said Maurizio Voi, MD, Thoracic Tumor Strategy Lead, Pfizer Oncology. “Data being presented show survival outcomes for PF-299 and crizotinib, as well as quality-of-life or patient-reported outcomes after treatment for patients with non small cell lung cancer, which represent important considerations in determining the best treatment option for these patients.”
First Presentation of PF-299 Preliminary Overall Survival Data
Continued....
Pfizer will present, for the first time, preliminary overall survival data from a Phase 2 study evaluating PF-299 vs erlotinib in patients with advanced non-small cell lung cancer (NSCLC) after progression on at least one chemotherapy regimen (oral presentation, Abstract #745, Monday, July 4).1
Pfizer also will present patient-reported outcomes (PRO) from clinical trials of PF-299 in refractory and second-/third-line NSCLC, which provide a better understanding of the patient’s perspective of the burden of adverse events associated with treatment and how it may change over time.3,4
- Gastrointestinal toxicity of the pan-HER tyrosine kinase inhibitor (TKI) PF299804: Assessment by patient-reported outcomes in second-/third-line and refractory NSCLC (poster session, Abstract #957, Wednesday, July 6)3
- Dermatologic adverse events of the pan-HER tyrosine kinase inhibitor (TKI) PF299804: Assessment by patient-reported outcomes in second-/third-line and refractory NSCLC (poster session, Abstract #702, Wednesday, July 6)4
PF-299 targets multiple receptors of the HER pathway. PF-299 is an irreversible inhibitor of HER-1 (EGFR), HER-2 and HER-4 tyrosine kinases. 6
Crizotinib Data to be presented:
At the WCLC, data on the anti-tumor activity, safety, overall survival, patient-reported and quality-of-life outcomes observed in clinical trials of Pfizer’s crizotinib will be presented.2,7,8
- Phase 2 data for crizotinib in ALK-positive advanced NSCLC: PROFILE 1005 (oral presentation, Abstract #1618, Wednesday, July 6)2
- PROFILE 1005: Preliminary patient-reported outcomes (PROs) from an ongoing Phase 2 study of crizotinib in ALK-positive advanced NSCLC (oral presentation, Abstract #1510, Wednesday, July 6)7
- Crizotinib improves overall survival of ALK-positive patients with advanced NSCLC compared with historical controls (oral presentation, Abstract #1207, Wednesday, July 6)8
- Efficacy of crizotinib in retrospective comparisons with standard-of-care (SOC) regimens from three Pfizer-sponsored clinical trials in patients with advanced NSCLC (poster session, Abstract #1349, Wednesday, July 6)9
About Pfizer Oncology
Pfizer Oncology is committed to the discovery, investigation and development of innovative treatment options to improve the outlook for cancer patients worldwide. Our strong pipeline, one of the most robust in the industry, is studied with precise focus on identifying and translating the best scientific breakthroughs into clinical application for patients across a wide range of cancers. Pfizer Oncology has biologics and small molecules in clinical development and more than 100 clinical trials underway. By working collaboratively with academic institutions, individual researchers, cooperative research groups, governments, and licensing partners, Pfizer Oncology strives to cure or control cancer with breakthrough medicines, to deliver the right drug for each patient at the right time. For more information please visit www.Pfizer.com.
DISCLOSURE NOTICE: The information contained in this release is as of June 28, 2011. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.
This release contains forward-looking information about various oncology product candidates, including their potential benefits, that involves substantial risks and uncertainties. Such risks and uncertainties include, among other things, the uncertainties inherent in research and development; decisions by regulatory authorities regarding whether and when to approve any drug applications that have been or may be filed for any such oncology product candidates as well as their decisions regarding labeling and other matters that could affect their availability or commercial potential; and competitive developments.
A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2010 and in its reports on Form 10-Q and Form 8-K.
1 World Lung Accepted Abstract #745. Overall Survival (OS) Results of a Randomized Phase 2 Trial of PF299804 versus Erlotinib in Patients with Advanced Non-Small Cell Lung Cancer (NSCLC) After Failure of Chemotherapy. Oral Session, Monday July 4, 2011: 3:35 PM – 3:45 PM CEST. M. Boyer – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
2 World Lung Accepted Abstract #1618. Phase 2 Data for Crizotinib (PF-02341066) in ALK-Positive Advanced Non-Small Cell Lung Cancer (NSCLC): PROFILE 1005. Oral Session, Wednesday July 6, 2011: 3:10 PM – 3:20 PM CEST. G. Riely – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
3 World Lung Accepted Abstract #957. Gastrointestinal Toxicity of the Pan-HER Tyrosine Kinase Inhibitor (TKI) PF299804: Assessment by Patient-Reported Outcomes in 2nd/3rd-Line and Refractory Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. A. Campbell – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
4 World Lung Accepted Abstract #702. Dermatologic Adverse Events of the Pan-HER Tyrosine Kinase Inhibitor (TKI) PF299804: Assessment by Patient-Reported Outcomes in 2nd/3rd-line and Refractory Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. A. Campbell – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
5 Clinicaltrials.gov. ARCHER 1009: A Phase 3 Study of PF-00299804, a Pan-HER Inhibitor, Vs. Erolotinib in the Treatment of Advanced Non-Small Cell Lung Cancer. Available here: http://www.clinicaltrials.gov/ct2/show/NCT01360554?term=ARCHER&rank=1. Accessed June 21, 2011.
6 Gonzales AJ, Hook KE, Althaus IW et al. Antitumor activity and pharmacokinetic properties of PF-00299804, a second-generation irreversible pan-erbBreceptor tyrosine kinase inhibitor. Mol Cancer Ther. 2008;7:1880-89.
7 World Lung Accepted Abstract #1510. PROFILE 1005: Preliminary Patient-Reported Outcomes (PROs) from an Ongoing Phase 2 Study of Crizotinib (PF-02341066) in Anaplastic Lymphoma Kinase (ALK)-Positive Advanced Non-Small Cell Lung Cancer (NSCLC). Oral Session, Wednesday July 6, 2011: 3:30 PM – 3:40 PM CEST. F. Blackhall – Presenter. Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
8 World Lung Accepted Abstract #1207. Crizotinib improves overall survival of ALK-positive patients with advanced NSCLC compared with historical controls. Oral Session, Wednesday July 6, 2011: 3:20 PM – 3:30 PM CEST. A. Shaw – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
9 World Lung Accepted Abstract #1349. Efficacy of Crizotinib in Retrospective Comparisons with Standard-Of-Care (SOC) Regimens from Three Pfizer-Sponsored Clinical Trials in Patients with Advanced Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. Y. Tang – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
10 Bang Y et al. Clinical Activity of the Oral ALK Inhibitor, Crizotinib (PF-02341066), in Patients with ALK-positive Non-Small Cell Lung Cancer. Accepted Plenary Presentation at the American Society of Clinical Oncology Annual Meeting, June 4-8, 2010. Chicago, IL.
11 Zou HY, Li Q, Lee JH, et al. An orally available small-molecule inhibitor of c-MET,
PF-2341066, exhibits cytoreductive antitumor efficacy through antiproliferative and antiangiogenic mechanisms. Cancer Res. 2007;67:4408-4417.
12 Chiarle R, Voena C, Ambrogio C et al. The anaplastic lymphoma kinase in the pathogenesis of cancer. Nat Rev Cancer. 2008;8(1): 11-23.
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- Pfizer Lung Cancer Drug to Get Priority Review (xconomy.com)
- Benefit of targeted lung cancer therapy confirmed (eurekalert.org)
- Pfizer drug shows double survival time for certain lung cancer patients (nj.com)
- Analyst bets on approval of Pfizer's lung cancer drug (fiercebiotech.com)
12 Ekim 2012 Cuma
Human Genome Sciences Shares Decline After FDA Recommend Benlysta
To contact us Click HERE
Just after Tuesday's FDA Recommendation for Human Genome Sciences' Lupus Therapy Benlysta, Citigroup and Bank of America downgraded the stock. Obviously, they mistakenly shorted the stocks and wanted to get their money back after guessing wrong on the FDA decision. The drug was recommended by a 13-2 vote. It is very similar to other cases in recent FDA Approvals, where the stock has actually has gone down on good news, not up.
Manipulating shorts cover their positions by making sure they got analysts on their side to downgrade a stock in case a Panel vote goes against them. Here is a perfect example of how Hedge Fund Shorts cover their positions:
Two downgrades Wednesday morning, one each from Citi and Bank of America/Merrill Lynch, reflected these new concerns and sent Human Genome shares down 7% to $23.98 in early trading.
An advisory panel convened by the U.S. Food and Drug Administration voted 13-2 Tuesday night to recommend Benlysta's approval as the first new lupus therapy in 50 years.
The large margin of victory represented in the vote count, however, belied the contentiousness of the panel's discussion over Benlysta's efficacy. Many of the panelists expressed concern that Benlysta provided only a marginal benefit for U.S. lupus patients and that some specific types of lupus patients -- African Americans or those with lupus that attacks the kidneys or the central nervous system -- were not helped by the drug at all.
Despite recommending Benlysta's approval, the FDA panel expressed support for a label that potentially restricts the drug's use to a smaller segment of U.S. lupus patients than Wall Street had expected.
"The label will likely have noticeable restrictions and thin efficacy is already drawing a mixed reception from rheumatologists. We are concerned about the long-term sales potential given (1) modest benefit, (2) poorer activity in U.S. pts than we originally expected, and (3) now more restricted label," wrote Citi biotech analyst Yaron Werber in note Wednesday in which he downgraded Human Genome to a hold from a buy.
Werber cut his peak sales estimate for Benlysta to $1.6 billion from $2.6 billion and as a result his Human Genome price target dropped to $30 from $35.
Similarly, Bank of America/Merrill Lynch downgraded Human Genome to underperform with a $24 price target.
Investors are also concerned that FDA and Human Genome may not be able to resolve the outstanding issues needed to complete the Benlysta review in time to meet the Dec. 9 approval decision date. This increases the likelihood that Benlysta's approval could be delayed 1-3 months.
ISI Group biotech analyst Mark Schoenebaum defended Human Genome Wednesday, calling concerns about a significant cut in Benlysta peak sales overblown. Even with a narrower label, Benlysta peak sales worldwide can still exceed $3 billion, he wrote in an email to clients Wednesday.
Human Genome downgraded over lupus drug potential
Nov. 17, 2010
SAN FRANCISCO (MarketWatch) — Two analysts downgraded Human Genome Sciences Wednesday after a U.S. Food and Drug Administration panel showed support for a lupus drug Human Genome is co-developing with GlaxoSmithKline PLC.
The key concern: Sales for the lupus drug called Benlysta may not be as big as previously thought, according to analysts at Citigroup and Bank of America who both downgraded Human Genome and cut their stock-price targets before the stock market opened.
By midmorning trades Wednesday, Human Genome (HGSI 23.93, -0.58, -2.37%) shares had already been on a rollercoaster ride as investors tried to digest the long range implications for the lupus drug called Benlysta. The stock fell 8% in opening trades but recovered all those losses later in the session.
By late morning, Human Genome was up 1% at $26.32.
Lupus is an inflammatory connective tissue disease. It occurs mostly in women and is characterized by fever, skin rash, and arthritis.
An FDA panel late Tuesday voted 13-2 to recommend the FDA approve Benlysta. A separate vote on the effectiveness of drug was 10-5.
In a research note, B. of. A. analyst Rachel McMinn said FDA panel feedback at Tuesday afternoon’s meeting “underscored concerns that Benlysta will have a smaller than expected role in lupus treatment.”
McMinn had previously assumed treatment using the lupus drug would run $40,000 per patient. She lowered that calculation to $35,000.
She cut Human Genome to “underperform” from “buy” and shaved her long-range price target to $24 a share, from $33.
Citigroup analyst Yaron Werber downgraded Human Genome to “hold” from “buy” and cut his price target to $30 a share, from $35.
He now expects Benlysta to reach global sales of $1.6 billion by 2015, down from a previous assumption of $2.6 billion
 |
| Elmo the Investment Banker is killing traders. Stick to Sesame Street Elmo. You aren't cut out for Wall St. |
Just after Tuesday's FDA Recommendation for Human Genome Sciences' Lupus Therapy Benlysta, Citigroup and Bank of America downgraded the stock. Obviously, they mistakenly shorted the stocks and wanted to get their money back after guessing wrong on the FDA decision. The drug was recommended by a 13-2 vote. It is very similar to other cases in recent FDA Approvals, where the stock has actually has gone down on good news, not up.
Manipulating shorts cover their positions by making sure they got analysts on their side to downgrade a stock in case a Panel vote goes against them. Here is a perfect example of how Hedge Fund Shorts cover their positions:
Two downgrades Wednesday morning, one each from Citi and Bank of America/Merrill Lynch, reflected these new concerns and sent Human Genome shares down 7% to $23.98 in early trading.
An advisory panel convened by the U.S. Food and Drug Administration voted 13-2 Tuesday night to recommend Benlysta's approval as the first new lupus therapy in 50 years.
The large margin of victory represented in the vote count, however, belied the contentiousness of the panel's discussion over Benlysta's efficacy. Many of the panelists expressed concern that Benlysta provided only a marginal benefit for U.S. lupus patients and that some specific types of lupus patients -- African Americans or those with lupus that attacks the kidneys or the central nervous system -- were not helped by the drug at all.
Despite recommending Benlysta's approval, the FDA panel expressed support for a label that potentially restricts the drug's use to a smaller segment of U.S. lupus patients than Wall Street had expected.
"The label will likely have noticeable restrictions and thin efficacy is already drawing a mixed reception from rheumatologists. We are concerned about the long-term sales potential given (1) modest benefit, (2) poorer activity in U.S. pts than we originally expected, and (3) now more restricted label," wrote Citi biotech analyst Yaron Werber in note Wednesday in which he downgraded Human Genome to a hold from a buy.
Werber cut his peak sales estimate for Benlysta to $1.6 billion from $2.6 billion and as a result his Human Genome price target dropped to $30 from $35.
Similarly, Bank of America/Merrill Lynch downgraded Human Genome to underperform with a $24 price target.
Investors are also concerned that FDA and Human Genome may not be able to resolve the outstanding issues needed to complete the Benlysta review in time to meet the Dec. 9 approval decision date. This increases the likelihood that Benlysta's approval could be delayed 1-3 months.
ISI Group biotech analyst Mark Schoenebaum defended Human Genome Wednesday, calling concerns about a significant cut in Benlysta peak sales overblown. Even with a narrower label, Benlysta peak sales worldwide can still exceed $3 billion, he wrote in an email to clients Wednesday.
Human Genome downgraded over lupus drug potential
Nov. 17, 2010
SAN FRANCISCO (MarketWatch) — Two analysts downgraded Human Genome Sciences Wednesday after a U.S. Food and Drug Administration panel showed support for a lupus drug Human Genome is co-developing with GlaxoSmithKline PLC.
The key concern: Sales for the lupus drug called Benlysta may not be as big as previously thought, according to analysts at Citigroup and Bank of America who both downgraded Human Genome and cut their stock-price targets before the stock market opened.
By midmorning trades Wednesday, Human Genome (HGSI 23.93, -0.58, -2.37%) shares had already been on a rollercoaster ride as investors tried to digest the long range implications for the lupus drug called Benlysta. The stock fell 8% in opening trades but recovered all those losses later in the session.
By late morning, Human Genome was up 1% at $26.32.
Lupus is an inflammatory connective tissue disease. It occurs mostly in women and is characterized by fever, skin rash, and arthritis.
An FDA panel late Tuesday voted 13-2 to recommend the FDA approve Benlysta. A separate vote on the effectiveness of drug was 10-5.
In a research note, B. of. A. analyst Rachel McMinn said FDA panel feedback at Tuesday afternoon’s meeting “underscored concerns that Benlysta will have a smaller than expected role in lupus treatment.”
McMinn had previously assumed treatment using the lupus drug would run $40,000 per patient. She lowered that calculation to $35,000.
She cut Human Genome to “underperform” from “buy” and shaved her long-range price target to $24 a share, from $33.
Citigroup analyst Yaron Werber downgraded Human Genome to “hold” from “buy” and cut his price target to $30 a share, from $35.
He now expects Benlysta to reach global sales of $1.6 billion by 2015, down from a previous assumption of $2.6 billion
Related articles
- "Human Genome Sciences dissed: Lupus drug disappoints Wall Street" and related posts (personalmoneystore.com)
- The Ratings Game: Human Genome downgraded over lupus drug potential (marketwatch.com)
- Human Genome Sciences and GlaxoSmithKline Announce Vote of FDA Advisory Committee to Recommend Approval of BENLYSTA for Systemic Lupus Erythematosus (eon.businesswire.com)
- Benlysta Shows Promise for Lupus (lupus.webmd.com)
- "Advisers recommend Human Genome lupus drug" and related posts (health.am)
- These Bankers Knew What They Were Selling (blogs.forbes.com)
Aastrom Announces Phase II Clinical Results, Shares Sink
To contact us Click HERE
Good and Bad news for Aastrom Biosciences today. Shares dropped heavily after Reuters reports that Aastrom's potential stem cell therapy did not meet a secondary endpoint for it's Phase II clinical trial for the treatment of Critical Limb Ischemia which can cause amputation in those who suffer from the disease. I have seen this a few times lately, where a stock receives generally positive news, only to come crumbling down. Aastrom simply rose too fast, too soon.
Yahoo Finance: Aastrom Biosciences--(ASTM)
Aastrom Ischemia Therapy Data Disappoint; Shares Tank
* Says study meets main safety and efficacy goal
* Did not show significant amputation free survival
* Shares fall 36 pct
Nov 18 (Reuters) - Aastrom Biosciences Inc (ASTM.O) reported its experimental ischemia treatment met its main goals, but the company's shares, which had soared in anticipation of the data, crashed as the secondary goal of the trial was not met.
The study met the primary goals of safety and efficacy in patients with critical limb ischemia (CLI), a cardiovascular condition that often leads to amputation of the limbs.
CLI is a painful condition caused by obstructions in the arteries that decreases blood flow to the limbs with no medical therapy available at present.
Interim data from the second mid-stage trial failed to show statistically significant amputation-free survival in patients, although data published in June from an earlier mid-stage study had shown promising results. [ID:nSGE65A0F9]
McNicoll, Lewis & Vlak analyst George Zavoico said the discrepancies with the earlier data was due to the size and scope of the trial.
"Its too small a trial, its underpowered and I think people are forgetting that. You have tremendous variations with fewer patients," he said.
The analyst said data on the time to treatment failure events, which include amputation, deepening of wounds and gangrene, was encouraging.
"The phase 2 trial did exactly what it was supposed to do, that is inform on the phase 3 trial plan," analyst Zavoico said.
Last month, the company submitted a special protocol assessment (SPA) to U.S. health regulators for a late-stage trial on the drug and said it plans to initiate the study in early 2011
The stem cell research company's tissue repair cell technology (TRC) delivers the patient's own bone marrow directly to the damaged tissues.
A special protocol assessment provides the company with a written agreement that the design and analysis of the trial are adequate to support a marketing application submission.
The company's shares, which have more than doubled in value the last one month, closed down 36 percent at $2.71 Thursday on Nasdaq. They had touched a 52-week high of $4.45 prior to the announcement.
(Reporting by Krishnakali Sengupta in Bangalore;Editing by Prem Udayabhanu) (krishnakali.sengupta@thomsonreuters.com; within U.S. +1 646 223 8780; outside U.S. +91 80 4135 5800
Reuters Messaging: krishnakali.sengupta.reuters.com@reuters.net))
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Investors buy ASTM on rumor, sell on news that study met main safety and efficacy goal
Shares were halted this afternoon for Aastrom Biosciences, Inc. (Nasdaq:ASTM), the regenerative medicine company engaged in the development of autologous cell products for treatment of severe, chronic cardiovascular diseases as the company announced that an interim analysis of all 86 patients enrolled in the company's Phase 2b RESTORE-CLI clinical trial shows that the study achieved both its primary safety endpoint and primary efficacy endpoint of time to first occurrence of treatment failure.
The findings were statistically significant (p=0.0132) and close analyses showed a clinically meaningful reduction of 56% in treatment failure events. The RESTORE-CLI trial is the largest fully controlled cell-therapy study ever conducted in critical limb ischemia (CLI)- a severe blockage in the arteries of the lower extremities, which markedly reduces blood-flow. It is a serious form of peripheral arterial disease. The most prominent features of critical limb ischemia are severe pain in the legs and feet while a person is not moving, or non-healing sores on the feet or legs. ASTM is the first company to make it this far in clinical trials with such positive results.
Shares had climbed significantly in advance of today's news presentation but most investors chose to take profits off the table upon release of the data when Reuters reported that the study failed to meet a key secondary goal- something which the study was not designed to demonstrate. That data showed a clinically meaningful reduction in event rates (amputation) of 24% but did not show statistical significance. Phase III will be designed to focus on that secondary data, but at least momentarily the article panicked investors and sent shares tumbling. Others swooped in for the suddenly discounted shares and took them back from $2.86 to the $3.85 range. Moments later, shares continued a slow decline as investors began to digest the news and try to decide how to best intrepret the study data over the next few days.
Aastrom Biosciences has become is a strong stem cell play, but fear of dillution has also impacted shares after a mid-November registration with the U.S. Securities and Exchange Commission to sell, from time to time, up to $75 million of Common Stock, Preferred Stock, Debt Securities, Warrants, Units, or any combination thereof. It appears that the fear that the company could sell shares on the good news played a role in the mass exodus.
The company has made great strides in their pipeline and over the next few months are scheduled to share more information with the investment community about their break-through treatment candidates. According to the their latest shareholder presentation, in December they are expecting to announce the complete enrollment in Phase II for their DCM (Dilated Cardiomyopathy catheter trial along with:
Phase 2 Surgical-DCM 6-month results in January,
SPA negotiations for Phase 3 in CLI with FDA between now and early 2011
Phase 2b CLI 12-month results in Q2
Phase 2 Surgical-DCM 12 month results in Q2/Q3
The value of Aastrom’s pipeline will be more clear going forward, as these results and other news start to trickle in. Success in CLI has set ASTM apart from other stem cell companies as other pharmaceutical and biotechnology firms have recently abandoned programs aiming to treat CLI.
In February 2010, Aastrom had reported results from an interim analysis of data from this trial that showed a statistically significant clinical benefit favoring Aastrom's autologous cellular therapy for the first 46 patients enrolled in the trial, in both time to first occurrence of treatment failure (a composite endpoint consisting of major amputation of treated leg, all-cause mortality, doubling of total wound surface area from baseline and de novo gangrene; p=0.005) and amputation-free survival (p=0.038).
Results of the second interim analysis were presented today in a non-CME satellite session of the VEITHsymposium(TM) in New York City by principal investigator Richard Powell, M.D., chief of vascular surgery at Dartmouth-Hitchcock Medical Center in Lebanon, NH. An archived webcast of the presentation will be available at www.aastrom.com/investor.cfm.
"I am very optimistic about the treatment response rates we have seen in patients in this trial so far. These interim data suggest that there is a clear and clinically meaningful therapeutic effect at work in treated patients. I look forward to examining the benefits of this treatment in the context of a larger Phase 3 study," said Dr. Powell.
The two interim analyses were done to provide information to support the design and execution of Aastrom's Phase 3 CLI program. In October, Aastrom announced plans to initiate a Phase 3 CLI clinical development program under special protocol assessments (SPA) with a Fast Track designation by the FDA.
"We believe these interim results provide further evidence supporting the use of our autologous cell therapy to treat patients with this devastating disease," said Tim Mayleben, president and CEO of Aastrom Biosciences. "As expected, these data provide essential guidance as we refine and finalize the sample sizes and patient selection criteria for our Phase 3 program."
Good and Bad news for Aastrom Biosciences today. Shares dropped heavily after Reuters reports that Aastrom's potential stem cell therapy did not meet a secondary endpoint for it's Phase II clinical trial for the treatment of Critical Limb Ischemia which can cause amputation in those who suffer from the disease. I have seen this a few times lately, where a stock receives generally positive news, only to come crumbling down. Aastrom simply rose too fast, too soon.
Yahoo Finance: Aastrom Biosciences--(ASTM)
Aastrom Ischemia Therapy Data Disappoint; Shares Tank
* Says study meets main safety and efficacy goal
* Did not show significant amputation free survival
* Shares fall 36 pct
Nov 18 (Reuters) - Aastrom Biosciences Inc (ASTM.O) reported its experimental ischemia treatment met its main goals, but the company's shares, which had soared in anticipation of the data, crashed as the secondary goal of the trial was not met.
The study met the primary goals of safety and efficacy in patients with critical limb ischemia (CLI), a cardiovascular condition that often leads to amputation of the limbs.
CLI is a painful condition caused by obstructions in the arteries that decreases blood flow to the limbs with no medical therapy available at present.
Interim data from the second mid-stage trial failed to show statistically significant amputation-free survival in patients, although data published in June from an earlier mid-stage study had shown promising results. [ID:nSGE65A0F9]
McNicoll, Lewis & Vlak analyst George Zavoico said the discrepancies with the earlier data was due to the size and scope of the trial.
"Its too small a trial, its underpowered and I think people are forgetting that. You have tremendous variations with fewer patients," he said.
The analyst said data on the time to treatment failure events, which include amputation, deepening of wounds and gangrene, was encouraging.
"The phase 2 trial did exactly what it was supposed to do, that is inform on the phase 3 trial plan," analyst Zavoico said.
Last month, the company submitted a special protocol assessment (SPA) to U.S. health regulators for a late-stage trial on the drug and said it plans to initiate the study in early 2011
The stem cell research company's tissue repair cell technology (TRC) delivers the patient's own bone marrow directly to the damaged tissues.
A special protocol assessment provides the company with a written agreement that the design and analysis of the trial are adequate to support a marketing application submission.
The company's shares, which have more than doubled in value the last one month, closed down 36 percent at $2.71 Thursday on Nasdaq. They had touched a 52-week high of $4.45 prior to the announcement.
(Reporting by Krishnakali Sengupta in Bangalore;Editing by Prem Udayabhanu) (krishnakali.sengupta@thomsonreuters.com; within U.S. +1 646 223 8780; outside U.S. +91 80 4135 5800
Reuters Messaging: krishnakali.sengupta.reuters.com@reuters.net))
More From Reuters
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Investors buy ASTM on rumor, sell on news that study met main safety and efficacy goal
Shares were halted this afternoon for Aastrom Biosciences, Inc. (Nasdaq:ASTM), the regenerative medicine company engaged in the development of autologous cell products for treatment of severe, chronic cardiovascular diseases as the company announced that an interim analysis of all 86 patients enrolled in the company's Phase 2b RESTORE-CLI clinical trial shows that the study achieved both its primary safety endpoint and primary efficacy endpoint of time to first occurrence of treatment failure.
The findings were statistically significant (p=0.0132) and close analyses showed a clinically meaningful reduction of 56% in treatment failure events. The RESTORE-CLI trial is the largest fully controlled cell-therapy study ever conducted in critical limb ischemia (CLI)- a severe blockage in the arteries of the lower extremities, which markedly reduces blood-flow. It is a serious form of peripheral arterial disease. The most prominent features of critical limb ischemia are severe pain in the legs and feet while a person is not moving, or non-healing sores on the feet or legs. ASTM is the first company to make it this far in clinical trials with such positive results.
Shares had climbed significantly in advance of today's news presentation but most investors chose to take profits off the table upon release of the data when Reuters reported that the study failed to meet a key secondary goal- something which the study was not designed to demonstrate. That data showed a clinically meaningful reduction in event rates (amputation) of 24% but did not show statistical significance. Phase III will be designed to focus on that secondary data, but at least momentarily the article panicked investors and sent shares tumbling. Others swooped in for the suddenly discounted shares and took them back from $2.86 to the $3.85 range. Moments later, shares continued a slow decline as investors began to digest the news and try to decide how to best intrepret the study data over the next few days.
Aastrom Biosciences has become is a strong stem cell play, but fear of dillution has also impacted shares after a mid-November registration with the U.S. Securities and Exchange Commission to sell, from time to time, up to $75 million of Common Stock, Preferred Stock, Debt Securities, Warrants, Units, or any combination thereof. It appears that the fear that the company could sell shares on the good news played a role in the mass exodus.
The company has made great strides in their pipeline and over the next few months are scheduled to share more information with the investment community about their break-through treatment candidates. According to the their latest shareholder presentation, in December they are expecting to announce the complete enrollment in Phase II for their DCM (Dilated Cardiomyopathy catheter trial along with:
Phase 2 Surgical-DCM 6-month results in January,
SPA negotiations for Phase 3 in CLI with FDA between now and early 2011
Phase 2b CLI 12-month results in Q2
Phase 2 Surgical-DCM 12 month results in Q2/Q3
The value of Aastrom’s pipeline will be more clear going forward, as these results and other news start to trickle in. Success in CLI has set ASTM apart from other stem cell companies as other pharmaceutical and biotechnology firms have recently abandoned programs aiming to treat CLI.
In February 2010, Aastrom had reported results from an interim analysis of data from this trial that showed a statistically significant clinical benefit favoring Aastrom's autologous cellular therapy for the first 46 patients enrolled in the trial, in both time to first occurrence of treatment failure (a composite endpoint consisting of major amputation of treated leg, all-cause mortality, doubling of total wound surface area from baseline and de novo gangrene; p=0.005) and amputation-free survival (p=0.038).
Results of the second interim analysis were presented today in a non-CME satellite session of the VEITHsymposium(TM) in New York City by principal investigator Richard Powell, M.D., chief of vascular surgery at Dartmouth-Hitchcock Medical Center in Lebanon, NH. An archived webcast of the presentation will be available at www.aastrom.com/investor.cfm.
"I am very optimistic about the treatment response rates we have seen in patients in this trial so far. These interim data suggest that there is a clear and clinically meaningful therapeutic effect at work in treated patients. I look forward to examining the benefits of this treatment in the context of a larger Phase 3 study," said Dr. Powell.
The two interim analyses were done to provide information to support the design and execution of Aastrom's Phase 3 CLI program. In October, Aastrom announced plans to initiate a Phase 3 CLI clinical development program under special protocol assessments (SPA) with a Fast Track designation by the FDA.
"We believe these interim results provide further evidence supporting the use of our autologous cell therapy to treat patients with this devastating disease," said Tim Mayleben, president and CEO of Aastrom Biosciences. "As expected, these data provide essential guidance as we refine and finalize the sample sizes and patient selection criteria for our Phase 3 program."
Related articles
- Aastrom Gets FDA Fast Track Status, Gears Up for Pivotal Study of Cell Therapy (xconomy.com)
- Aastrom Maps Out Pivotal Trial Strategy With Adult Stem Cell Therapy (fool.com)
- Aastrom Gets FDA Fast Track Status, Gears Up for Pivotal Study of Cell Therapy - Xconomy (news.google.com)
BioPharma Investor Is Back In Business-Apologies for the Lack of Posts Last Month
To contact us Click HERE
Sorry for any delays. I was out of town for the past month and was not able to keep up with the website. The website will be back up and running in the next few days with new BioPharma News and Articles. Again I apologize for a lack of quality posts last month, but again I will be working diligently throughout this year to provide the most up to date news and information on the hottest BioPharma Stocks and Clinical Trial news throughout 2011 without any more delays. Thanks for your patience and support.
Sincerely,
BioPharma Investor
Sincerely,
BioPharma Investor
2011 Biotech Preview: JP Morgan Healthcare Conference
To contact us Click HERE
JP Morgan Healthcare Conference 2011 Preview
Stocks Mentioned: AMAG, AMGN, AMLN, ARIA, CELG, DNDN, GENZ, GILD, HGSI, ITMN, KERX, MNKD, NKTR, OREX, SGEN, SNY, SVNT, TEVA, VRTX, VVUS, MRK
The Street: Biotech 2011 Kickoff Party Investor Preview
By: Adam Feuerstein
The biotech industry descends on the City by the Bay starting Sunday for a week of investor meetings, networking, deal making and partying all centered on the 29th annual J.P. Morgan Healthcare Conference.
Biotech investor conferences are a dime a dozen but the J.P. Morgan confab is the oldest, largest and still the most important because of tradition, location (in the birthplace of biotech) and timing. Companies and investors come into this early January meeting with clean slates and use the forum to set goals and agendas for the next 12 months.
2010 was a mixed year for biotech. The biotech sector outperformed the S&P 500 (barely) but sentiment for a large part of the year was negative, dragged down by healthcare reform concerns, European drug price cuts and overall economic uncertainty. Many large institutional investors abandoned or scaled back healthcare investments in 2010 as a result. Drug approvals and merger-and-acquisition activity -- two vital measures of the health of the biotech sector -- were down in 2010.
What's in store for biotech in 2011? J.P. Morgan's senior biotech analyst Geoff Meacham, who will emcee next week's conference, is bullish:
"Looking to 2011, we think the tone in the sector will improve, and as a result, we are broadly bullish on the biotech sector, more than we have been over the past two years," Meacham told his clients in a Monday research note. "Historically, revenue upside (not EPS upside) has been a critical determinant of biotech outperformance, and in this regard the consensus revenue growth for the sector looks too low to us at 8%. More than 25 products could be in launch mode in 2011, which should fuel higher top-line growth for the sector and may drive multiple expansion. Couple this with a favorable M&A and capital markets environment as well as well-known risks from US/EU pricing that are largely assumed in models and we think that the biotech sector is well positioned in 2011." Continued
I'll be in San Francisco next week to cover and analyze all the breaking biotech news, rumors and goings-on at the J.P. Morgan Healthcare conference.
Biotech investor conferences are a dime a dozen but the J.P. Morgan confab is the oldest, largest and still the most important because of tradition, location (in the birthplace of biotech) and timing. Companies and investors come into this early January meeting with clean slates and use the forum to set goals and agendas for the next 12 months.
2010 was a mixed year for biotech. The biotech sector outperformed the S&P 500 (barely) but sentiment for a large part of the year was negative, dragged down by healthcare reform concerns, European drug price cuts and overall economic uncertainty. Many large institutional investors abandoned or scaled back healthcare investments in 2010 as a result. Drug approvals and merger-and-acquisition activity -- two vital measures of the health of the biotech sector -- were down in 2010.
What's in store for biotech in 2011? J.P. Morgan's senior biotech analyst Geoff Meacham, who will emcee next week's conference, is bullish:
"Looking to 2011, we think the tone in the sector will improve, and as a result, we are broadly bullish on the biotech sector, more than we have been over the past two years," Meacham told his clients in a Monday research note. "Historically, revenue upside (not EPS upside) has been a critical determinant of biotech outperformance, and in this regard the consensus revenue growth for the sector looks too low to us at 8%. More than 25 products could be in launch mode in 2011, which should fuel higher top-line growth for the sector and may drive multiple expansion. Couple this with a favorable M&A and capital markets environment as well as well-known risks from US/EU pricing that are largely assumed in models and we think that the biotech sector is well positioned in 2011."
I'll be in San Francisco next week to cover and analyze all the breaking biotech news, rumors and goings-on at the J.P. Morgan Healthcare conference.
To prep, I'm busy putting together my schedule and trying to figure out a way to clone myself so that I can be in two or three places simultaneously. The following is a capsule look at some (certainly not all) of the major story lines and questions for biotech and drug companies presenting at the J.P. Morgan conference next week.
Monday, Jan. 10
Celgene (CELG): By tradition, this big-cap biotech kicks off the JP Morgan conference every year with financial guidance for 2011 and a pre-announcement of fourth-quarter earnings. Celgene tends to be conservative with its forward guidance, which gives the company flexibility to beat and raise forecasts later in the year. [It also creates a fair amount of volatility in the stock price.] At present, the Street's consensus estimate has Celgene earning $3.38 a share on total revenue of $4.42 billion in 2011. Sales of Revlimid, the company's key multiple myeloma drug, are expected to reach $3.01 billion in 2011.
Vertex Pharmaceuticals (VRTX): The focus of the company's presentation -- and investor questions -- will be on the big year ahead for the hepatitis C drug telaprevir. FDA will likely hold an advisory panel in the second quarter, followed by a May approval decision date. Telaprevir's full approval is widely expected, so most investors are already looking ahead to the drug's commercial launch and the looming market share battle with Merck (MRK) and its hepatitis C drug boceprevir.
Dendreon (DNDN): Management is supposed to update investors on regulatory plans for Provenge in Europe so the J.P. Morgan conference might be the venue for that important news. The key question: Will European regulators require Dendreon to conduct another Provenge clinical trial? Here in the U.S., investors are focused on the complete build out later this year of Dendreon's three manufacturing plants and how Provenge sales will ramp once supply constraints are lifted.
Amylin Pharmaceuticals (AMLN): This year's JP Morgan conference was supposed to be where Amylin management talked excitedly about the early Bydureon commercial launch. Instead, the company will field questions about how fast it can respond to FDA's October rejection letter.
AMAG Pharmaceuticals (AMAG): Can AMAG right the disastrous commercial launch of the intravenous iron replacement therapy Feraheme? Does CEO Brian Pereira have any credibility left with investors, or will someone at the conference suggest he step aside? At AMAG's current stock price, Feraheme is essentially valued at nothing, so maybe there's value to be found here if/when the company's problems are ironed out.
Human Genome Sciences (HGSI): The FDA's approval decision date for the company's lupus drug is March 10. The big questions for Human Genome is not whether or not FDA approves Benlysta, but what kind of restrictions, if any, does FDA place on the drug's label and how does that affect the drug's commercial launch later in the year.
Gilead Sciences (GILD): Management usually waits for its fourth-quarter call later in January to provide forward product sales guidance, but right now, the Street consensus has Gilead earning $4.04 a share on total revenue of $8.41 billion in 2011. That implies about 10% earnings growth over 2010 -- a relatively low bar and well below the company's average growth rate. Meaningful HIV catalysts this year include FDA approval in the second quarter for "Btripla" [Johnson & Johnson's TMC278 combined with Gilead's Truvada] and phase III data from the HIV "Quad" pill in the second half of the year.
Tuesday, Jan. 11
Amgen (AMGN): Expect a lot of discussion and questions about the Prolia osteoporosis launch and the expectations/potential for Xgeva in cancer, particularly in the prevention of prostate cancer bone metastases. Given the persistent rumors about Amgen's interest in overseas acquisitions, someone will invariably ask management about M&A strategy. Historically, Amgen has not provided earnings guidance at the JP Morgan conference.
Nektar Therapeutics (NKTR): Management might better explain or justify the decision announced in December to not seek a partner for its cancer drug NKTR-102.
Genzyme (GENZ): "Henri, when will you stop playing hard to get with Sanofi-Aventis and just sell the company?"
Seattle Genetics (SGEN): An update on timing of the approval filing for brentuximab vedotin, now expected in the first quarter. I'd expect management to field questions about the size of the commercial markets in Hodgkins lymphoma and anaplastic large cell lymphoma.
Sanofi-Aventis (SNY): "Chris, when are you going to raise your bid price for Genzyme and get this deal done?"
Teva Pharmaceuticals (TEVA): The impact Novartis' Gilenya is having on Copaxone's market share in multiple sclerosis. What is the probability of a generic version of Copaxone? And an update on Teva's own oral MS drug laquinimod.
Savient Pharmaceuticals (SVNT): An update on the early solo efforts selling the gout drug Krystexxa.
Biogen Idec (BIIB): Data from two phase III studies of the oral MS drug BG-12 are the most important catalysts in 2011. Biogen sometimes, but not always, pre-announces fourth quarter earnings and offers forward guidance at the J.P. Morgan conference. Current consensus is for Biogen to earn $5.67 a share on $4.69 billion in revenue.
Keryx Pharmaceuticals (KERX): Meaningful clinical data from the ongoing phase III studies of perifosine are more likely to be 2012 events, so 2011 might be a bit of a dead year. The company did recently file a new registration statement with the SEC, so could money raising be a near-term event?
Wednesday, January 12:
Vivus (VVUS) and Orexigen Therapeutics (OREX): Obesity drugs? Will the FDA ever approve one or both?
Ariad Pharmaceuticals (ARIA): Data from the phase III study of ridaforolimus in sarcoma is expected in the first quarter. Given that Merck essentially controls that drug, most of the investors at JP Morgan will be much more interested in hearing about Ariad's leukemia drug ponatinib -- a potential blockbuster in the making.
InterMune (ITMN): The decision by European regulators last month to approve pirfenidone for idiopathic pulmonary fibrosis should put the company's executives in a much happier frame of mind as they update investors at the conference.
Mannkind (MNKD): Thirty minutes in a crowded breakout room with Al Mann just days or weeks before the FDA issues an approval decision on Afrezza. This might be the don't-miss event of the conference.
Arena Pharmaceuticals (ARNA): Rat cancer. By the time CEO Jack Lief is ready to speak at 4 pm, most of the investors (at least those still in San Francisco) are likely to be in a bar.
Thursday, January 13:
My condolences to any company forced to present on the last day of the JP Morgan conference, when dust bunnies and crumpled cocktail napkins outnumber investors in the audience. Try for a better slot next year by giving J.P. Morgan more of your banking business. I'll be on a flight home.
--Written by Adam Feuerstein in Boston.
Related articles
JP Morgan Healthcare Conference 2011 PreviewStocks Mentioned: AMAG, AMGN, AMLN, ARIA, CELG, DNDN, GENZ, GILD, HGSI, ITMN, KERX, MNKD, NKTR, OREX, SGEN, SNY, SVNT, TEVA, VRTX, VVUS, MRK
The Street: Biotech 2011 Kickoff Party Investor Preview
By: Adam Feuerstein
The biotech industry descends on the City by the Bay starting Sunday for a week of investor meetings, networking, deal making and partying all centered on the 29th annual J.P. Morgan Healthcare Conference.
Biotech investor conferences are a dime a dozen but the J.P. Morgan confab is the oldest, largest and still the most important because of tradition, location (in the birthplace of biotech) and timing. Companies and investors come into this early January meeting with clean slates and use the forum to set goals and agendas for the next 12 months.
2010 was a mixed year for biotech. The biotech sector outperformed the S&P 500 (barely) but sentiment for a large part of the year was negative, dragged down by healthcare reform concerns, European drug price cuts and overall economic uncertainty. Many large institutional investors abandoned or scaled back healthcare investments in 2010 as a result. Drug approvals and merger-and-acquisition activity -- two vital measures of the health of the biotech sector -- were down in 2010.
What's in store for biotech in 2011? J.P. Morgan's senior biotech analyst Geoff Meacham, who will emcee next week's conference, is bullish:
"Looking to 2011, we think the tone in the sector will improve, and as a result, we are broadly bullish on the biotech sector, more than we have been over the past two years," Meacham told his clients in a Monday research note. "Historically, revenue upside (not EPS upside) has been a critical determinant of biotech outperformance, and in this regard the consensus revenue growth for the sector looks too low to us at 8%. More than 25 products could be in launch mode in 2011, which should fuel higher top-line growth for the sector and may drive multiple expansion. Couple this with a favorable M&A and capital markets environment as well as well-known risks from US/EU pricing that are largely assumed in models and we think that the biotech sector is well positioned in 2011." Continued
I'll be in San Francisco next week to cover and analyze all the breaking biotech news, rumors and goings-on at the J.P. Morgan Healthcare conference.
Biotech investor conferences are a dime a dozen but the J.P. Morgan confab is the oldest, largest and still the most important because of tradition, location (in the birthplace of biotech) and timing. Companies and investors come into this early January meeting with clean slates and use the forum to set goals and agendas for the next 12 months.
2010 was a mixed year for biotech. The biotech sector outperformed the S&P 500 (barely) but sentiment for a large part of the year was negative, dragged down by healthcare reform concerns, European drug price cuts and overall economic uncertainty. Many large institutional investors abandoned or scaled back healthcare investments in 2010 as a result. Drug approvals and merger-and-acquisition activity -- two vital measures of the health of the biotech sector -- were down in 2010.
What's in store for biotech in 2011? J.P. Morgan's senior biotech analyst Geoff Meacham, who will emcee next week's conference, is bullish:
"Looking to 2011, we think the tone in the sector will improve, and as a result, we are broadly bullish on the biotech sector, more than we have been over the past two years," Meacham told his clients in a Monday research note. "Historically, revenue upside (not EPS upside) has been a critical determinant of biotech outperformance, and in this regard the consensus revenue growth for the sector looks too low to us at 8%. More than 25 products could be in launch mode in 2011, which should fuel higher top-line growth for the sector and may drive multiple expansion. Couple this with a favorable M&A and capital markets environment as well as well-known risks from US/EU pricing that are largely assumed in models and we think that the biotech sector is well positioned in 2011."
I'll be in San Francisco next week to cover and analyze all the breaking biotech news, rumors and goings-on at the J.P. Morgan Healthcare conference.
To prep, I'm busy putting together my schedule and trying to figure out a way to clone myself so that I can be in two or three places simultaneously. The following is a capsule look at some (certainly not all) of the major story lines and questions for biotech and drug companies presenting at the J.P. Morgan conference next week.
Monday, Jan. 10
Celgene (CELG): By tradition, this big-cap biotech kicks off the JP Morgan conference every year with financial guidance for 2011 and a pre-announcement of fourth-quarter earnings. Celgene tends to be conservative with its forward guidance, which gives the company flexibility to beat and raise forecasts later in the year. [It also creates a fair amount of volatility in the stock price.] At present, the Street's consensus estimate has Celgene earning $3.38 a share on total revenue of $4.42 billion in 2011. Sales of Revlimid, the company's key multiple myeloma drug, are expected to reach $3.01 billion in 2011.
Vertex Pharmaceuticals (VRTX): The focus of the company's presentation -- and investor questions -- will be on the big year ahead for the hepatitis C drug telaprevir. FDA will likely hold an advisory panel in the second quarter, followed by a May approval decision date. Telaprevir's full approval is widely expected, so most investors are already looking ahead to the drug's commercial launch and the looming market share battle with Merck (MRK) and its hepatitis C drug boceprevir.
Dendreon (DNDN): Management is supposed to update investors on regulatory plans for Provenge in Europe so the J.P. Morgan conference might be the venue for that important news. The key question: Will European regulators require Dendreon to conduct another Provenge clinical trial? Here in the U.S., investors are focused on the complete build out later this year of Dendreon's three manufacturing plants and how Provenge sales will ramp once supply constraints are lifted.
Amylin Pharmaceuticals (AMLN): This year's JP Morgan conference was supposed to be where Amylin management talked excitedly about the early Bydureon commercial launch. Instead, the company will field questions about how fast it can respond to FDA's October rejection letter.
AMAG Pharmaceuticals (AMAG): Can AMAG right the disastrous commercial launch of the intravenous iron replacement therapy Feraheme? Does CEO Brian Pereira have any credibility left with investors, or will someone at the conference suggest he step aside? At AMAG's current stock price, Feraheme is essentially valued at nothing, so maybe there's value to be found here if/when the company's problems are ironed out.
Human Genome Sciences (HGSI): The FDA's approval decision date for the company's lupus drug is March 10. The big questions for Human Genome is not whether or not FDA approves Benlysta, but what kind of restrictions, if any, does FDA place on the drug's label and how does that affect the drug's commercial launch later in the year.
Gilead Sciences (GILD): Management usually waits for its fourth-quarter call later in January to provide forward product sales guidance, but right now, the Street consensus has Gilead earning $4.04 a share on total revenue of $8.41 billion in 2011. That implies about 10% earnings growth over 2010 -- a relatively low bar and well below the company's average growth rate. Meaningful HIV catalysts this year include FDA approval in the second quarter for "Btripla" [Johnson & Johnson's TMC278 combined with Gilead's Truvada] and phase III data from the HIV "Quad" pill in the second half of the year.
Tuesday, Jan. 11
Amgen (AMGN): Expect a lot of discussion and questions about the Prolia osteoporosis launch and the expectations/potential for Xgeva in cancer, particularly in the prevention of prostate cancer bone metastases. Given the persistent rumors about Amgen's interest in overseas acquisitions, someone will invariably ask management about M&A strategy. Historically, Amgen has not provided earnings guidance at the JP Morgan conference.
Nektar Therapeutics (NKTR): Management might better explain or justify the decision announced in December to not seek a partner for its cancer drug NKTR-102.
Genzyme (GENZ): "Henri, when will you stop playing hard to get with Sanofi-Aventis and just sell the company?"
Seattle Genetics (SGEN): An update on timing of the approval filing for brentuximab vedotin, now expected in the first quarter. I'd expect management to field questions about the size of the commercial markets in Hodgkins lymphoma and anaplastic large cell lymphoma.
Sanofi-Aventis (SNY): "Chris, when are you going to raise your bid price for Genzyme and get this deal done?"
Teva Pharmaceuticals (TEVA): The impact Novartis' Gilenya is having on Copaxone's market share in multiple sclerosis. What is the probability of a generic version of Copaxone? And an update on Teva's own oral MS drug laquinimod.
Savient Pharmaceuticals (SVNT): An update on the early solo efforts selling the gout drug Krystexxa.
Biogen Idec (BIIB): Data from two phase III studies of the oral MS drug BG-12 are the most important catalysts in 2011. Biogen sometimes, but not always, pre-announces fourth quarter earnings and offers forward guidance at the J.P. Morgan conference. Current consensus is for Biogen to earn $5.67 a share on $4.69 billion in revenue.
Keryx Pharmaceuticals (KERX): Meaningful clinical data from the ongoing phase III studies of perifosine are more likely to be 2012 events, so 2011 might be a bit of a dead year. The company did recently file a new registration statement with the SEC, so could money raising be a near-term event?
Wednesday, January 12:
Vivus (VVUS) and Orexigen Therapeutics (OREX): Obesity drugs? Will the FDA ever approve one or both?
Ariad Pharmaceuticals (ARIA): Data from the phase III study of ridaforolimus in sarcoma is expected in the first quarter. Given that Merck essentially controls that drug, most of the investors at JP Morgan will be much more interested in hearing about Ariad's leukemia drug ponatinib -- a potential blockbuster in the making.
InterMune (ITMN): The decision by European regulators last month to approve pirfenidone for idiopathic pulmonary fibrosis should put the company's executives in a much happier frame of mind as they update investors at the conference.
Mannkind (MNKD): Thirty minutes in a crowded breakout room with Al Mann just days or weeks before the FDA issues an approval decision on Afrezza. This might be the don't-miss event of the conference.
Arena Pharmaceuticals (ARNA): Rat cancer. By the time CEO Jack Lief is ready to speak at 4 pm, most of the investors (at least those still in San Francisco) are likely to be in a bar.
Thursday, January 13:
My condolences to any company forced to present on the last day of the JP Morgan conference, when dust bunnies and crumpled cocktail napkins outnumber investors in the audience. Try for a better slot next year by giving J.P. Morgan more of your banking business. I'll be on a flight home.
--Written by Adam Feuerstein in Boston.
Related articles
- Heads Up, Europe: Dendreon's Provenge Is Coming (blogs.wsj.com)
- Focus Turns To Biotech Next Week - Daily Markets (news.google.com)
- Dendreon Plans to Go It Alone in Europe, Build Provenge Factory in Germany (xconomy.com)
- Focus Turns To Biotech Next Week - Analyst Blog - Stock Markets Review (news.google.com)
- Dendreon Outlines European Strategy for Provenge (cenblog.org)
- Be a Better Biotech Investor - Investorplace.com (news.google.com)
- Off to JPMorgan Healthcare Conference... (cenblog.org)
Pfizer's To Present Lung Cancer Data July 3-7
To contact us Click HERE
Image via CrunchBase Pfizer Inc. will present early and mid-stage data from its lung cancer portfolio, including PF-00299804 (PF-299) an investigational, oral, pan-HER inhibitor;1 and crizotinib, an investigational, oral, first-in-class compound that inhibits the anaplastic lymphoma kinase, or ALK,2 at the International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), July 3-7 in Amsterdam, The Netherlands.
“While lung cancer remains a difficult-to-treat disease, we’re learning more about how therapies like crizotinib and PF-299 may be able to specifically target ALK or the HER pathway, respectively, and how this may lead to more rationally selected and personalized therapy,” said Maurizio Voi, MD, Thoracic Tumor Strategy Lead, Pfizer Oncology. “Data being presented show survival outcomes for PF-299 and crizotinib, as well as quality-of-life or patient-reported outcomes after treatment for patients with non small cell lung cancer, which represent important considerations in determining the best treatment option for these patients.”
First Presentation of PF-299 Preliminary Overall Survival Data
Continued....
Pfizer will present, for the first time, preliminary overall survival data from a Phase 2 study evaluating PF-299 vs erlotinib in patients with advanced non-small cell lung cancer (NSCLC) after progression on at least one chemotherapy regimen (oral presentation, Abstract #745, Monday, July 4).1
Pfizer also will present patient-reported outcomes (PRO) from clinical trials of PF-299 in refractory and second-/third-line NSCLC, which provide a better understanding of the patient’s perspective of the burden of adverse events associated with treatment and how it may change over time.3,4
PF-299 targets multiple receptors of the HER pathway. PF-299 is an irreversible inhibitor of HER-1 (EGFR), HER-2 and HER-4 tyrosine kinases. 6
Crizotinib Data to be presented:
At the WCLC, data on the anti-tumor activity, safety, overall survival, patient-reported and quality-of-life outcomes observed in clinical trials of Pfizer’s crizotinib will be presented.2,7,8
About Pfizer Oncology
Pfizer Oncology is committed to the discovery, investigation and development of innovative treatment options to improve the outlook for cancer patients worldwide. Our strong pipeline, one of the most robust in the industry, is studied with precise focus on identifying and translating the best scientific breakthroughs into clinical application for patients across a wide range of cancers. Pfizer Oncology has biologics and small molecules in clinical development and more than 100 clinical trials underway. By working collaboratively with academic institutions, individual researchers, cooperative research groups, governments, and licensing partners, Pfizer Oncology strives to cure or control cancer with breakthrough medicines, to deliver the right drug for each patient at the right time. For more information please visit www.Pfizer.com.
DISCLOSURE NOTICE: The information contained in this release is as of June 28, 2011. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.
This release contains forward-looking information about various oncology product candidates, including their potential benefits, that involves substantial risks and uncertainties. Such risks and uncertainties include, among other things, the uncertainties inherent in research and development; decisions by regulatory authorities regarding whether and when to approve any drug applications that have been or may be filed for any such oncology product candidates as well as their decisions regarding labeling and other matters that could affect their availability or commercial potential; and competitive developments.
A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2010 and in its reports on Form 10-Q and Form 8-K.
1 World Lung Accepted Abstract #745. Overall Survival (OS) Results of a Randomized Phase 2 Trial of PF299804 versus Erlotinib in Patients with Advanced Non-Small Cell Lung Cancer (NSCLC) After Failure of Chemotherapy. Oral Session, Monday July 4, 2011: 3:35 PM – 3:45 PM CEST. M. Boyer – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
2 World Lung Accepted Abstract #1618. Phase 2 Data for Crizotinib (PF-02341066) in ALK-Positive Advanced Non-Small Cell Lung Cancer (NSCLC): PROFILE 1005. Oral Session, Wednesday July 6, 2011: 3:10 PM – 3:20 PM CEST. G. Riely – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
3 World Lung Accepted Abstract #957. Gastrointestinal Toxicity of the Pan-HER Tyrosine Kinase Inhibitor (TKI) PF299804: Assessment by Patient-Reported Outcomes in 2nd/3rd-Line and Refractory Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. A. Campbell – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
4 World Lung Accepted Abstract #702. Dermatologic Adverse Events of the Pan-HER Tyrosine Kinase Inhibitor (TKI) PF299804: Assessment by Patient-Reported Outcomes in 2nd/3rd-line and Refractory Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. A. Campbell – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
5 Clinicaltrials.gov. ARCHER 1009: A Phase 3 Study of PF-00299804, a Pan-HER Inhibitor, Vs. Erolotinib in the Treatment of Advanced Non-Small Cell Lung Cancer. Available here: http://www.clinicaltrials.gov/ct2/show/NCT01360554?term=ARCHER&rank=1. Accessed June 21, 2011.
6 Gonzales AJ, Hook KE, Althaus IW et al. Antitumor activity and pharmacokinetic properties of PF-00299804, a second-generation irreversible pan-erbBreceptor tyrosine kinase inhibitor. Mol Cancer Ther. 2008;7:1880-89.
7 World Lung Accepted Abstract #1510. PROFILE 1005: Preliminary Patient-Reported Outcomes (PROs) from an Ongoing Phase 2 Study of Crizotinib (PF-02341066) in Anaplastic Lymphoma Kinase (ALK)-Positive Advanced Non-Small Cell Lung Cancer (NSCLC). Oral Session, Wednesday July 6, 2011: 3:30 PM – 3:40 PM CEST. F. Blackhall – Presenter. Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
8 World Lung Accepted Abstract #1207. Crizotinib improves overall survival of ALK-positive patients with advanced NSCLC compared with historical controls. Oral Session, Wednesday July 6, 2011: 3:20 PM – 3:30 PM CEST. A. Shaw – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
9 World Lung Accepted Abstract #1349. Efficacy of Crizotinib in Retrospective Comparisons with Standard-Of-Care (SOC) Regimens from Three Pfizer-Sponsored Clinical Trials in Patients with Advanced Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. Y. Tang – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
10 Bang Y et al. Clinical Activity of the Oral ALK Inhibitor, Crizotinib (PF-02341066), in Patients with ALK-positive Non-Small Cell Lung Cancer. Accepted Plenary Presentation at the American Society of Clinical Oncology Annual Meeting, June 4-8, 2010. Chicago, IL.
11 Zou HY, Li Q, Lee JH, et al. An orally available small-molecule inhibitor of c-MET,
PF-2341066, exhibits cytoreductive antitumor efficacy through antiproliferative and antiangiogenic mechanisms. Cancer Res. 2007;67:4408-4417.
12 Chiarle R, Voena C, Ambrogio C et al. The anaplastic lymphoma kinase in the pathogenesis of cancer. Nat Rev Cancer. 2008;8(1): 11-23.
Image via CrunchBase Pfizer Inc. will present early and mid-stage data from its lung cancer portfolio, including PF-00299804 (PF-299) an investigational, oral, pan-HER inhibitor;1 and crizotinib, an investigational, oral, first-in-class compound that inhibits the anaplastic lymphoma kinase, or ALK,2 at the International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), July 3-7 in Amsterdam, The Netherlands. “While lung cancer remains a difficult-to-treat disease, we’re learning more about how therapies like crizotinib and PF-299 may be able to specifically target ALK or the HER pathway, respectively, and how this may lead to more rationally selected and personalized therapy,” said Maurizio Voi, MD, Thoracic Tumor Strategy Lead, Pfizer Oncology. “Data being presented show survival outcomes for PF-299 and crizotinib, as well as quality-of-life or patient-reported outcomes after treatment for patients with non small cell lung cancer, which represent important considerations in determining the best treatment option for these patients.”
First Presentation of PF-299 Preliminary Overall Survival Data
Continued....
Pfizer will present, for the first time, preliminary overall survival data from a Phase 2 study evaluating PF-299 vs erlotinib in patients with advanced non-small cell lung cancer (NSCLC) after progression on at least one chemotherapy regimen (oral presentation, Abstract #745, Monday, July 4).1
Pfizer also will present patient-reported outcomes (PRO) from clinical trials of PF-299 in refractory and second-/third-line NSCLC, which provide a better understanding of the patient’s perspective of the burden of adverse events associated with treatment and how it may change over time.3,4
- Gastrointestinal toxicity of the pan-HER tyrosine kinase inhibitor (TKI) PF299804: Assessment by patient-reported outcomes in second-/third-line and refractory NSCLC (poster session, Abstract #957, Wednesday, July 6)3
- Dermatologic adverse events of the pan-HER tyrosine kinase inhibitor (TKI) PF299804: Assessment by patient-reported outcomes in second-/third-line and refractory NSCLC (poster session, Abstract #702, Wednesday, July 6)4
PF-299 targets multiple receptors of the HER pathway. PF-299 is an irreversible inhibitor of HER-1 (EGFR), HER-2 and HER-4 tyrosine kinases. 6
Crizotinib Data to be presented:
At the WCLC, data on the anti-tumor activity, safety, overall survival, patient-reported and quality-of-life outcomes observed in clinical trials of Pfizer’s crizotinib will be presented.2,7,8
- Phase 2 data for crizotinib in ALK-positive advanced NSCLC: PROFILE 1005 (oral presentation, Abstract #1618, Wednesday, July 6)2
- PROFILE 1005: Preliminary patient-reported outcomes (PROs) from an ongoing Phase 2 study of crizotinib in ALK-positive advanced NSCLC (oral presentation, Abstract #1510, Wednesday, July 6)7
- Crizotinib improves overall survival of ALK-positive patients with advanced NSCLC compared with historical controls (oral presentation, Abstract #1207, Wednesday, July 6)8
- Efficacy of crizotinib in retrospective comparisons with standard-of-care (SOC) regimens from three Pfizer-sponsored clinical trials in patients with advanced NSCLC (poster session, Abstract #1349, Wednesday, July 6)9
About Pfizer Oncology
Pfizer Oncology is committed to the discovery, investigation and development of innovative treatment options to improve the outlook for cancer patients worldwide. Our strong pipeline, one of the most robust in the industry, is studied with precise focus on identifying and translating the best scientific breakthroughs into clinical application for patients across a wide range of cancers. Pfizer Oncology has biologics and small molecules in clinical development and more than 100 clinical trials underway. By working collaboratively with academic institutions, individual researchers, cooperative research groups, governments, and licensing partners, Pfizer Oncology strives to cure or control cancer with breakthrough medicines, to deliver the right drug for each patient at the right time. For more information please visit www.Pfizer.com.
DISCLOSURE NOTICE: The information contained in this release is as of June 28, 2011. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.
This release contains forward-looking information about various oncology product candidates, including their potential benefits, that involves substantial risks and uncertainties. Such risks and uncertainties include, among other things, the uncertainties inherent in research and development; decisions by regulatory authorities regarding whether and when to approve any drug applications that have been or may be filed for any such oncology product candidates as well as their decisions regarding labeling and other matters that could affect their availability or commercial potential; and competitive developments.
A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2010 and in its reports on Form 10-Q and Form 8-K.
1 World Lung Accepted Abstract #745. Overall Survival (OS) Results of a Randomized Phase 2 Trial of PF299804 versus Erlotinib in Patients with Advanced Non-Small Cell Lung Cancer (NSCLC) After Failure of Chemotherapy. Oral Session, Monday July 4, 2011: 3:35 PM – 3:45 PM CEST. M. Boyer – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
2 World Lung Accepted Abstract #1618. Phase 2 Data for Crizotinib (PF-02341066) in ALK-Positive Advanced Non-Small Cell Lung Cancer (NSCLC): PROFILE 1005. Oral Session, Wednesday July 6, 2011: 3:10 PM – 3:20 PM CEST. G. Riely – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
3 World Lung Accepted Abstract #957. Gastrointestinal Toxicity of the Pan-HER Tyrosine Kinase Inhibitor (TKI) PF299804: Assessment by Patient-Reported Outcomes in 2nd/3rd-Line and Refractory Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. A. Campbell – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
4 World Lung Accepted Abstract #702. Dermatologic Adverse Events of the Pan-HER Tyrosine Kinase Inhibitor (TKI) PF299804: Assessment by Patient-Reported Outcomes in 2nd/3rd-line and Refractory Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. A. Campbell – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
5 Clinicaltrials.gov. ARCHER 1009: A Phase 3 Study of PF-00299804, a Pan-HER Inhibitor, Vs. Erolotinib in the Treatment of Advanced Non-Small Cell Lung Cancer. Available here: http://www.clinicaltrials.gov/ct2/show/NCT01360554?term=ARCHER&rank=1. Accessed June 21, 2011.
6 Gonzales AJ, Hook KE, Althaus IW et al. Antitumor activity and pharmacokinetic properties of PF-00299804, a second-generation irreversible pan-erbBreceptor tyrosine kinase inhibitor. Mol Cancer Ther. 2008;7:1880-89.
7 World Lung Accepted Abstract #1510. PROFILE 1005: Preliminary Patient-Reported Outcomes (PROs) from an Ongoing Phase 2 Study of Crizotinib (PF-02341066) in Anaplastic Lymphoma Kinase (ALK)-Positive Advanced Non-Small Cell Lung Cancer (NSCLC). Oral Session, Wednesday July 6, 2011: 3:30 PM – 3:40 PM CEST. F. Blackhall – Presenter. Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
8 World Lung Accepted Abstract #1207. Crizotinib improves overall survival of ALK-positive patients with advanced NSCLC compared with historical controls. Oral Session, Wednesday July 6, 2011: 3:20 PM – 3:30 PM CEST. A. Shaw – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
9 World Lung Accepted Abstract #1349. Efficacy of Crizotinib in Retrospective Comparisons with Standard-Of-Care (SOC) Regimens from Three Pfizer-Sponsored Clinical Trials in Patients with Advanced Non-Small Cell Lung Cancer (NSCLC). Poster Session, Wednesday July 6, 2011: 12:15 PM – 2:15 PM CEST. Y. Tang – Presenter. International Association for the Study of Lung Cancer’s (IASLC) 14th World Conference on Lung Cancer (WCLC), Amsterdam, The Netherlands. July 3-7, 2011.
10 Bang Y et al. Clinical Activity of the Oral ALK Inhibitor, Crizotinib (PF-02341066), in Patients with ALK-positive Non-Small Cell Lung Cancer. Accepted Plenary Presentation at the American Society of Clinical Oncology Annual Meeting, June 4-8, 2010. Chicago, IL.
11 Zou HY, Li Q, Lee JH, et al. An orally available small-molecule inhibitor of c-MET,
PF-2341066, exhibits cytoreductive antitumor efficacy through antiproliferative and antiangiogenic mechanisms. Cancer Res. 2007;67:4408-4417.
12 Chiarle R, Voena C, Ambrogio C et al. The anaplastic lymphoma kinase in the pathogenesis of cancer. Nat Rev Cancer. 2008;8(1): 11-23.
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- Pfizer Lung Cancer Drug to Get Priority Review (xconomy.com)
- Benefit of targeted lung cancer therapy confirmed (eurekalert.org)
- Pfizer drug shows double survival time for certain lung cancer patients (nj.com)
- Analyst bets on approval of Pfizer's lung cancer drug (fiercebiotech.com)
11 Ekim 2012 Perşembe
Spectrum Pharmaceuticals is Making Headlines in Early 2011
To contact us Click HERE
Spectrum Pharmaceuticals Submits Supplemental New Drug Application (sNDA) For Ready-to-Use Formulation Of FUSILEV(R) In Colorectal Cancer
Main Category: Colorectal Cancer
Also Included In: Cancer / Oncology
Article Date: 06 Jan 2011 - 1:00 PST
Spectrum Pharmaceuticals (Nasdaq: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in oncology, announced it submitted a supplemental New Drug Application with the U.S. Food and Drug Administration (FDA) for a Ready-to-Use formulation of FUSILEV (levoleucovorin) for Injection. This submission is in support of FUSILEV's use in colorectal cancer, which is currently under review by the FDA with a Prescription Drug User Fee Act (PDUFA) action date of April 29 2011. FDA has up to 60 days to formally accept the submission.
"If approved, we believe a RTU formulation of FUSILEV, as the name implies, will be more convenient for the staff and easy to use. RTU formulation requires no reconstitution, and since it will also be of a higher strength than the currently available lyophilized formulation, the healthcare provider's time to administer FUSILEV to colorectal cancer patients will be considerably reduced," said Rajesh C. Shrotriya, MD, Chairman of the Board of Directors, Chief Executive Officer, and President of Spectrum Pharmaceuticals. Continued
FUSILEV is currently FDA approved and marketed by Spectrum for rescue after high-dose methotrexate therapy in osteosarcoma. FUSILEV is also indicated to diminish the toxicity and counteract the effects of impaired methotrexate elimination, and of inadvertent overdosage of folic acid antagonists.
About FUSILEV® (levoleucovorin) for Injection
FUSILEV, a novel folate analog, is available in vials as freeze-dried powder. FUSILEV rescue is indicated after high-dose methotrexate therapy in osteosarcoma. FUSILEV is also indicated to diminish the toxicity and counteract the effects of impaired methotrexate elimination and of inadvertent overdosage of folic acid antagonists. FUSILEV has been marketed outside the United States by Wyeth, Sanofi-Aventis, Takeda, and others for more than 10 years.
Important FUSILEV® (levoleucovorin) for Injection Safety Considerations
FUSILEV is contraindicated for patients who have had previous allergic reactions attributed to folic acid or folinic acid. Due to calcium content, no more than 16-mL (160-mg) of levoleucovorin solution should be injected intravenously per minute. FUSILEV enhances the cyto-toxicity of fluorouracil. Concomitant use of d,l-leucovorin with trimethoprim-sulfamethoxazole for pneumocystis carinii pneumonia in HIV patients was associated with increased rates of treatment failure in a placebo-controlled study. Allergic reactions were reported in patients receiving FUSILEV.
Vomiting (38%), stomatitis (38%) and nausea (19%) were reported in patients receiving FUSILEV as rescue after high dose methotrexate therapy. FUSILEV may counteract the antiepileptic effect of phenobarbital, phenytoin and primidone, and increase the frequency of seizures in susceptible patients.
Source:
Spectrum Pharmaceuticals: Website http://www.spectrumpharm.com/
Product Pipeline for Spectrum Pharmaceuticals
View drug information on Fusilev.
UPDATE: Spectrum Pharmaceuticals In Deal With Viropro For Cancer Drug
DOW JONES NEWSWIRES
Spectrum Pharmaceuticals Inc. (SPPI) signed a deal with Viropro Inc. (VPRO) to develop a biosimilar version of Swiss pharmaceutical giant Roche Holding AG's (RHHBY, ROG.VX) cancer drug rituximab ahead of its U.S. patent expiration in 2015.
The agreement comes as many patent expirations loom over large pharmaceutical companies. Rituximab, used in the treatment of many lymphomas, leukemias, transplant rejection and some autoimmune disorders, saw worldwide sales of about $5.6 billion in 2009.
Financial terms weren't disclosed.
Viropro, a biotech drug maker, said Spectrum will license the rights to certain technology and it should get milestone payments over 36 months and royalty payments from ensuing sales.
Biosimilar drugs are officially approved versions of biopharmaceutical products made by a different company as patent and exclusivity expire on the product.
Biotech company Spectrum, which has a focus on cancer treatment, has been focused on making another push to improve the weak sales of its leading drug Zevalin. In its most recent quarterly results, Spectrum swung to a loss as it spent more to market Zevalin and another drug, Fusilev, which treats a vitamin deficiency in patients who have been treated with chemotherapy for colorectal cancer.
Meanwhile, Roche, the world's biggest maker of cancer drugs, is facing pressure following the U.S. Food and Drug Administration December decision to revoke approval of blockbuster drug Avastin for the treatment of breast cancer. The company has said it will appeal.
Spectrum shares were up 4 cents at $6.92. The stock is up 49% over 12 months.
Spectrum Pharma (NASDAQ: SPPI) Posts Large Volume Increase, Hits $6.34
January 7th, 2011
Shares of Spectrum Pharma (NASDAQ: SPPI) saw unusually high trading volume on Friday. Approximately 1.492 million shares changed hands during mid-day trading. During the most recent quarter, the stock had an average daily volume of 540,149 shares. The stock last traded at $6.34.
On a related note, analysts at Zacks Investment Research reiterated a “neutral” rating on shares of Spectrum Pharma in a research note to investors on Wednesday, December 29th. Separately, analysts at Morgan Joseph raised their price target on shares of Spectrum Pharma from $8.00 to $10.00 in a research note to investors on Wednesday, December 8th. They now have a “buy” rating on the stock.
Spectrum Pharmaceuticals, Inc. ( Spectrum) is a commercial stage biopharmaceutical company, engaged in developing and commercializing therapies with a focus primarily in the areas of hematology-oncology and urology. The Company has a fully developed commercial infrastructure that markets and sells two drugs in the United States, Zevalin and Fusilev. The Company has several drug candidates in development, which include apaziquone (EOquin), which is being studied in two large Phase III clinical trials for non-muscle invasive bladder cancer (NMIBC) under a strategic collaboration with Allergan; and belinostat, a drug partnered with TopoTarget A/S to jointly develop. Belinostat is being studied in multiple indications, including a Phase II registrational trial for relapsed or refractory Peripheral T-Cell Lymphoma (PTCL).
Spectrum Pharma (NASDAQ: SPPI) traded down 6.45% during mid-day trading on Friday. The stock has a 52 week low of $3.67 and a 52 week high of $7.19. Its 50-day moving average is $5.70 and its 200-day moving average is $4.51. The company has a market cap of $322.1 million and a price-to-earnings ratio of N/A.
Related articles
Main Category: Colorectal Cancer
Also Included In: Cancer / Oncology
Article Date: 06 Jan 2011 - 1:00 PST
Spectrum Pharmaceuticals (Nasdaq: SPPI), a biotechnology company with fully integrated commercial and drug development operations with a primary focus in oncology, announced it submitted a supplemental New Drug Application with the U.S. Food and Drug Administration (FDA) for a Ready-to-Use formulation of FUSILEV (levoleucovorin) for Injection. This submission is in support of FUSILEV's use in colorectal cancer, which is currently under review by the FDA with a Prescription Drug User Fee Act (PDUFA) action date of April 29 2011. FDA has up to 60 days to formally accept the submission.
"If approved, we believe a RTU formulation of FUSILEV, as the name implies, will be more convenient for the staff and easy to use. RTU formulation requires no reconstitution, and since it will also be of a higher strength than the currently available lyophilized formulation, the healthcare provider's time to administer FUSILEV to colorectal cancer patients will be considerably reduced," said Rajesh C. Shrotriya, MD, Chairman of the Board of Directors, Chief Executive Officer, and President of Spectrum Pharmaceuticals. Continued
FUSILEV is currently FDA approved and marketed by Spectrum for rescue after high-dose methotrexate therapy in osteosarcoma. FUSILEV is also indicated to diminish the toxicity and counteract the effects of impaired methotrexate elimination, and of inadvertent overdosage of folic acid antagonists.
About FUSILEV® (levoleucovorin) for Injection
FUSILEV, a novel folate analog, is available in vials as freeze-dried powder. FUSILEV rescue is indicated after high-dose methotrexate therapy in osteosarcoma. FUSILEV is also indicated to diminish the toxicity and counteract the effects of impaired methotrexate elimination and of inadvertent overdosage of folic acid antagonists. FUSILEV has been marketed outside the United States by Wyeth, Sanofi-Aventis, Takeda, and others for more than 10 years.
Important FUSILEV® (levoleucovorin) for Injection Safety Considerations
FUSILEV is contraindicated for patients who have had previous allergic reactions attributed to folic acid or folinic acid. Due to calcium content, no more than 16-mL (160-mg) of levoleucovorin solution should be injected intravenously per minute. FUSILEV enhances the cyto-toxicity of fluorouracil. Concomitant use of d,l-leucovorin with trimethoprim-sulfamethoxazole for pneumocystis carinii pneumonia in HIV patients was associated with increased rates of treatment failure in a placebo-controlled study. Allergic reactions were reported in patients receiving FUSILEV.
Vomiting (38%), stomatitis (38%) and nausea (19%) were reported in patients receiving FUSILEV as rescue after high dose methotrexate therapy. FUSILEV may counteract the antiepileptic effect of phenobarbital, phenytoin and primidone, and increase the frequency of seizures in susceptible patients.
Source:
Spectrum Pharmaceuticals: Website http://www.spectrumpharm.com/
Product Pipeline for Spectrum Pharmaceuticals
View drug information on Fusilev.
UPDATE: Spectrum Pharmaceuticals In Deal With Viropro For Cancer Drug
DOW JONES NEWSWIRES
Spectrum Pharmaceuticals Inc. (SPPI) signed a deal with Viropro Inc. (VPRO) to develop a biosimilar version of Swiss pharmaceutical giant Roche Holding AG's (RHHBY, ROG.VX) cancer drug rituximab ahead of its U.S. patent expiration in 2015.
The agreement comes as many patent expirations loom over large pharmaceutical companies. Rituximab, used in the treatment of many lymphomas, leukemias, transplant rejection and some autoimmune disorders, saw worldwide sales of about $5.6 billion in 2009.
Financial terms weren't disclosed.
Viropro, a biotech drug maker, said Spectrum will license the rights to certain technology and it should get milestone payments over 36 months and royalty payments from ensuing sales.
Biosimilar drugs are officially approved versions of biopharmaceutical products made by a different company as patent and exclusivity expire on the product.
Biotech company Spectrum, which has a focus on cancer treatment, has been focused on making another push to improve the weak sales of its leading drug Zevalin. In its most recent quarterly results, Spectrum swung to a loss as it spent more to market Zevalin and another drug, Fusilev, which treats a vitamin deficiency in patients who have been treated with chemotherapy for colorectal cancer.
Meanwhile, Roche, the world's biggest maker of cancer drugs, is facing pressure following the U.S. Food and Drug Administration December decision to revoke approval of blockbuster drug Avastin for the treatment of breast cancer. The company has said it will appeal.
Spectrum shares were up 4 cents at $6.92. The stock is up 49% over 12 months.
Spectrum Pharma (NASDAQ: SPPI) Posts Large Volume Increase, Hits $6.34
January 7th, 2011
Shares of Spectrum Pharma (NASDAQ: SPPI) saw unusually high trading volume on Friday. Approximately 1.492 million shares changed hands during mid-day trading. During the most recent quarter, the stock had an average daily volume of 540,149 shares. The stock last traded at $6.34.
On a related note, analysts at Zacks Investment Research reiterated a “neutral” rating on shares of Spectrum Pharma in a research note to investors on Wednesday, December 29th. Separately, analysts at Morgan Joseph raised their price target on shares of Spectrum Pharma from $8.00 to $10.00 in a research note to investors on Wednesday, December 8th. They now have a “buy” rating on the stock.
Spectrum Pharmaceuticals, Inc. ( Spectrum) is a commercial stage biopharmaceutical company, engaged in developing and commercializing therapies with a focus primarily in the areas of hematology-oncology and urology. The Company has a fully developed commercial infrastructure that markets and sells two drugs in the United States, Zevalin and Fusilev. The Company has several drug candidates in development, which include apaziquone (EOquin), which is being studied in two large Phase III clinical trials for non-muscle invasive bladder cancer (NMIBC) under a strategic collaboration with Allergan; and belinostat, a drug partnered with TopoTarget A/S to jointly develop. Belinostat is being studied in multiple indications, including a Phase II registrational trial for relapsed or refractory Peripheral T-Cell Lymphoma (PTCL).
Spectrum Pharma (NASDAQ: SPPI) traded down 6.45% during mid-day trading on Friday. The stock has a 52 week low of $3.67 and a 52 week high of $7.19. Its 50-day moving average is $5.70 and its 200-day moving average is $4.51. The company has a market cap of $322.1 million and a price-to-earnings ratio of N/A.
Related articles
- Inside Wall Street: Tiny Spectrum Pharmaceuticals Is Getting Plenty of Attention (dailyfinance.com)
- Spectrum Pharma Jumps in With the Big Dogs (fool.com)
- UPDATE 2-Spectrum Pharma to make biosimilar of Roche's rituximab (reuters.com)
- Spectrum sues Cangene for breach of contract (fiercebiotech.com)
- Drug Pipeline Pulse Check: Spectrum, QRxPharma, CytRx and Impax - Seeking Alpha (news.google.com)
- Advances in Colorectal Cancer: Improving Your Quality of Life (everydayhealth.com)
- Top Small-cap Biotech Spectrum Announces Promising Future for Cancer Drug - Beacon Equity Research (news.google.com)
- UPDATE 1-Spectrum Pharma says cancer drug shows promise; shrs up (reuters.com)
Raptor Pharmaceuticals in 2011
To contact us Click HERE
Cysteamine Image via Wikipedia
Interest for Raptor Pharmaceuticals is really picking up in 2011. Here are the recent headlines for this up and coming Small Cap Pharmaceutical company. It has a Pipeline for Rare Diseases and I expect 2011 to be a strong year for Raptor Pharmaceuticals if Clinical Trial results are successful. Results for DR Cysteamine clinical trials is expected in 1Q 2011. DR Cysteamine has clinical development programs including:
◦Nephropathic Cystinosis, a rare genetic disorder (Phase 3)
◦Non-alcoholic Steatohepatitis (NASH), a metabolic disorder of the liver (Phase 2)
◦Huntington's Disease, an inherited neurodegenerative disease (Phase 2)
Raptor Pharmaceuticals Stock Quote: RTTP Google Finance
Read more from their previous 10-K filings for the company's Strategic Development Plan here.
Find out more about their Clinical Pipeline here.
Continued
Raptor Pharmaceutical Hires Patrick Reichenberger to Lead Commercial Development of DR Cysteamine for Nephropathic Cystinosis
Contracts in Place for Cambrex Corporation and Patheon, Inc. to Manufacture Active Pharmaceutical Ingredient and Commercial Product, Respectively
January 10, 2011 6:30 AM EST
NOVATO, Calif., Jan. 10, 2011 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTP) announced today the appointment of Patrick Reichenberger to the newly created position of Vice President, Commercial Operations. Additionally, Raptor has engaged Cambrex Corporation (NYSE: CMB) for the manufacturing of the active pharmaceutical ingredient ("API"), cysteamine bitartrate, and Patheon, Inc. (TSX: PTI) to produce the commercial product, DR Cysteamine.
A 20-year industry veteran with significant expertise in commercial operations and product-launch management, Mr. Reichenberger will lead the development and management of Raptor's sales and marketing efforts along with its commercial manufacturing, supply and distribution programs. Prior to joining Raptor, Mr. Reichenberger served as Senior Director of Commercial Development at XOMA, LLC. In this role, he led XOMA's commercial development department with particular emphasis on XOMA 052, an anti-IL1 antibody for diabetes, cardiovascular disease, and Behcet's Uveits, an orphan disease.
Prior to XOMA, Mr. Reichenberger was in charge of marketing, sales and distribution at Questcor Pharmaceuticals where he developed patient access and reimbursement programs supporting the successful commercial re-launch of H.P. Acthar® Gel for the treatment of infantile spasms, an ultra-orphan, pediatric disease. Prior to Questcor, he led commercial and strategic marketing for brands at Genentech, Athena Neurosciences, and Parke-Davis, where he assisted in the launch of Lipitor®.
"The addition of Patrick is particularly appropriate at this time as we work towards a potential commercial launch of DR Cysteamine for nephropathic cystinosis in 2012. His hands-on commercial development expertise will be central to our efforts to build the necessary commercial infrastructure and capability for the near term and as the Company grows in later years." stated Christopher M. Starr, Ph.D., Chief Executive Officer of Raptor.
Mr. Reichenberger stated "My experience in building commercial infrastructures around orphan therapeutics, especially Acthar® Gel, is particularly applicable to Raptor's nephropathic cystinosis program. I am encouraged by the enthusiastic patient and foundation support for Raptor's DR Cysteamine program, and I look forward to working to create a sound patient access program ready for commercial launch. While developing and implementing such a program for nephropathic cystinosis is my top priority at Raptor, I am also very excited about the entire Raptor pipeline. I am impressed with the commercial opportunities at Raptor with a near term program in an orphan indication for nephropathic cystinosis and mid-stage programs in other indications, including Huntington's Disease and NASH. I'm pleased to have the opportunity to use all of my capabilities, from long-term strategic planning to practical marketing and sales, in this position."
Ted Daley, President of Raptor stated "We are very pleased to welcome Patrick to lead our commercial efforts. The addition of Patrick to our leadership team, along with our recently established long-term manufacturing agreements with Cambrex and Patheon, are significant milestones in the continued advancement of our DR Cysteamine programs. Both Cambrex and Patheon have significant expertise working with cysteamine bitartrate. Cambrex has provided us with cysteamine bitartrate for all our clinical studies and, for over 10 years, has been one of the few API manufacturers worldwide that is capable of providing a commercial source of pharmaceutical-grade cysteamine bitartrate. Patheon developed Raptor's DR Cysteamine microbead formulation and currently manufactures our clinical drug supply. We look forward to employing their combined expertise and capabilities in our commercial manufacturer."
About Nephropathic Cystinosis
Nephropathic cystinosis is an inborn metabolic error characterized by the abnormal transport of cystine, an amino acid, out of the lysosomes. Failure to treat nephropathic cystinosis can cause serious health consequences, including renal failure and resultant kidney transplant, growth failure, rickets, photophobia and blindness. Symptom onset typically occurs within the first year of life, when cystine crystals accumulate in various tissues and organs, including the kidneys, brain, liver, thyroid, pancreas, muscles and eyes.
About Cysteamine and DR Cysteamine
DR Cysteamine is Raptor's proprietary enteric-coated, microbead oral formulation of cysteamine bitartrate designed to potentially reduce dosing frequency and gastrointestinal side effects associated with immediate-release cysteamine bitartrate, which is approved for sale by the FDA and EMA to treat nephropathic cystinosis, a rare, genetic lysosomal storage disease.
In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego for the development DR Cysteamine for nephropathic cystinosis and cysteamine for other potential indications including Huntington's Disease, NASH and Batten Disease.
About Raptor Pharmaceutical Corp.
Raptor Pharmaceutical Corp. (Nasdaq: RPTP) ("Raptor") is dedicated to speeding the delivery of new treatment options to patients by working to improve existing therapeutics through the application of highly specialized drug targeting platforms and formulation expertise. Raptor focuses on underserved patient populations where it can have the greatest potential impact. Raptor currently has product candidates in clinical development designed to potentially treat nephropathic cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase ("ALDH2") deficiency, and thrombotic disorder.
Raptor's preclinical programs are based upon bioengineered novel drug candidates and drug-targeting platforms derived from the human receptor-associated protein ("RAP") and related proteins that are designed to target cancer, neurodegenerative disorders and infectious diseases.
For additional information, please visit http://www.raptorpharma.com/.
Wedbush Starts Raptor Pharmaceuticals (RPTP) at Outperform
January 6, 2011 5:05 PM EST
Wedbush initiates coverage on Raptor Pharmaceuticals (NASDAQ: RPTP) with a Outperform rating and $7 price target.
The firm said, "We expect that Phase III data from RPTP’s lead DR Cysteamine program, anticipated in Q1:11, to be an important positive catalyst for the stock. Mature results from the Phase III results, in our opinion, will demonstrate non-inferiority to Cystagon as suggested by previous Phase IIa and IIb studies. Additionally, the drug, a delayed-release formulation of the approved active ingredient cysteamine bitartrate (Cystagon), should offer a significant improvement in quality of life and should demonstrate a more favorable safety profile with fewer side effects, in our opinion."
For more ratings news on Raptor Pharmaceuticals click here and for the rating history of Raptor Pharmaceuticals click here.
Shares of Raptor Pharmaceuticals closed at $3.39 yesterday, with a 52 week range of $1.41-$4.00.
Raptor Pharmaceuticals: A Pipeline With Potential
October 07, 2010
By: Tony Pelz-Seeking Alpha
In my continuing search for “off the beaten track” small cap biotechs with impending catalysts, I came across Raptor Pharmaceuticals (Ticker: RPTP). Despite its checkered past, RTPT possesses an interesting pipeline with numerous near-to-medium term catalysts. According to the Company,
Raptor currently has product candidates in clinical development designed to potentially treat nephropathic cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's Disease, aldehyde dehydrogenase deficiency and a non-opioid solution designed to potentially treat chronic pain.
The Company also has a small roster of pre-clinical products.
Here are the catalysts and associated estimated market values:
•DR Cysteamine (Cystinosis): Phase 3 started June 2010; data expected to be reported 4Q’10/1Q’11; est. market value: $100 million
•DR Cysteamine (NASH): Phase 2a data released 5/2010; IND filing for Phase 2b 1H’11; est. market value: $1.8 billion
•DR Cysteamine (Huntington’s Disease): Phase 2 planned initiation Q3’10; est. market value: $2.1 billion
•Convivia: Out-licensing discussions; est. market value: $1.8 billion
•Tezampanel/NGX-426: Phase 2/3 ready, out-licensing discussions; est. market value: $700 million
The core medium term value drivers of the business going forward are clearly the candidates targeting NASH and Huntington’s. For the NASH indication alone, the Company claims there are currently 7.5 million adults (25 million by 2025) suffering from the disease with no current approved treatments. Investors are obviously cognizant of this market potential as positive results from Phase 2a data released in May 2010 caused the shares to rocket from around $1.50 to over $3.85. In addition, the stock regained all of its losses in just three days following a highly dilutive stock offering back in August; there appears to be a floor to the share price – quite rare. The near term Phase 3 data (Cystinosis) expected in 4Q’10/1Q’11, although targeting a smaller market, should at the very least (if positive) provide some price stability given the expectations that the Company should eventually be positioned to generate organic cash flows in the near to medium term with an approved product – i.e. to survive long enough to get the other “big ticket” products approved.
On the financial side, RPTP maintains a cash balance of around $17.2 million with no debt. Raptor management believes existing cash levels, at current monthly cash burn, will last into December 2011 – this is a decent amount of leeway. The Company currently has around 30.1 million shares (41.9 million fully diluted) outstanding – if the shares begin to run, look out for additional equity raisings and associated dilution.
I am approaching this trade as more of a 1 year + horizon investment. I attempted to build a position with options for about 4 weeks but to no avail; far too illiquid with ridiculous spreads (4) – I would expect spreads to tighten up as word gets out about this Company and its potential. As such, I will be slowly building a common stock position. On that front, I don’t like the technical/chart for the stock. After the May 2010 price spike, the shares have flat lined into a tight range between $2.70 and $3.00 – also, this range has tighten significantly over the past several weeks. Usually, this tells me that the stock is about to make a big move in either direction (i.e. up or down). Thus, I intend to slowly build this position and not put on the entire trade in one lot. As options become more liquid, I may either trade out of the shares into a long synthetic, sell Calls against the position or sell Puts.
Looking forward, I don’t expect the stock to make a huge move on the near term Phase 3 data (if positive). That said, the Company’s other products and associated potential upside appear quite substantial. If these products begin to rack up decent data in the trials, not only will the market take notice (and hence the shares rise) but Raptor could become a decent takeover target.
Raptor Pharma (RPTP) Updates on DR Cysteamine Phase 3, Financials
August 24, 2010 6:49 AM EDT
Raptor Pharmaceutical Corp. (Nasdaq: RPTP), provides updates on timelines related to its clinical programs including the pivotal Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").
Financial Updates and Guidance
As of August 24, 2010, the Company has approximately $17.2 million in cash and cash equivalents taking into account approximately $14 million of proceeds, net of placement agent fees and expenses, the Company received in a private placement financing which closed on August 12, 2010. The Company anticipates that its cash and cash equivalents will support planned operations and its planned clinical development programs, as described below, into December 2011.
Cystinosis
Raptor's cystinosis program remains the highest priority for the Company. "Over the next 18 months, the majority of the Company's resources will be devoted to completing what we believe will be the final development phase of this program," said Christopher M. Starr, Ph.D., CEO of Raptor. "Anticipating the successful completion of our ongoing Phase 3 clinical trial in 2010 and NDA submission in 2011, we are building the commercial infrastructure in our anticipation of a commercial launch in 2012."
In November 2009, Raptor completed its Phase 2b clinical trial of DR Cysteamine in cystinosis. DR Cysteamine demonstrated improved tolerability and the potential to reduce total daily dosage and administration frequency compared to immediate-release cysteamine bitartrate.
On June 28, 2010, Raptor enrolled the first patient in its pivotal Phase 3 clinical trial in cystinosis. This trial is designed as a randomized, crossover, outpatient study of the safety, tolerability, pharmacokinetics ("PK") and pharmacodynamics ("PD") of DR Cysteamine compared to immediate-release cysteamine bitartrate in cystinosis patients. The study is being conducted at nine centers: four in the U.S., including Emory University, Children's Memorial Hospital at Northwestern University, Stanford University, and Texas Children's Hospital at Baylor University; and five in the EU including in Hopital Necker-Enfants Malades and Hopital Robert Debré in Paris, Hospices Civils de Lyon in Lyon, France, Centre Hospitalier Universitaire in Montpellier, France and Radboud University Medical Center in Nijmegen, The Netherlands.
"We are very excited to have begun this study and are pleased with the patient support and initial rate of enrollment we are seeing at our sites," said Craig Langman, M.D., the Isaac A. Abt, M.D., Professor of Kidney Diseases at Feinberg School of Medicine, Northwestern University in Chicago and Principal Investigator of the Phase 3 clinical trial. "We currently have three U.S. sites enrolling patients and a fourth to commence early next month. Our European sites will begin enrolling patients by mid-September".
The Company expects to complete patient enrollment in the next few months and anticipates that all patients will have completed the treatment period by the end of the year. The treatment period for each of the anticipated 30 patients in the clinical trial is a total of 9 weeks and all patients will continue on DR Cysteamine in an extension study following the treatment period.
If the results from this trial are successful, Raptor anticipates filing an NDA for DR Cysteamine in cystinosis in mid-2011 and is planning a concurrent filing with the EMA for registration in the EU.
Huntington's Disease
Raptor anticipates that the Phase 2 study using DR Cysteamine in 96 Huntington's Disease patients will start in the third quarter of 2010. The Phase 2 clinical trial will be conducted under a previously announced collaboration agreement with The Centre Hospitalier Universitaire d'Angers ("CHU d'Angers") of Angers, France. Clinical expenses of the study will be covered by a grant from the French government.
Under the collaboration agreement between the Company and CHU d'Angers, Raptor will supply the DR Cysteamine study drug and will retain commercial rights to the clinical trial results. This Phase 2 trial was contemplated based on encouraging preclinical results reported by Institut Curie, CNRS and Inserm scientists that cysteamine prevents death of neurons and increases levels of a brain growth factor called brain-derived neurotrophic factor ("BDNF") in Huntington's Disease models. Reduced levels of BDNF in the brain have been widely reported to be important in the clinical development of Huntington's Disease.
In addition to intellectual property licensed from University of California, San Diego ("UCSD") related to its DR Cysteamine programs, Raptor holds exclusive worldwide licenses to Huntington's Disease related intellectual property from the Weizmann Institute of Science in Israel and Japan's Niigata University.
Non-Alcoholic Steatohepatitis ("NASH")
Raptor's management views its DR Cysteamine development program in NASH as an exciting opportunity based on both its potential market size and the possibility of creating partnering relationships. Following encouraging results from its Phase 2a clinical trial of DR Cysteamine in juvenile NASH patients, reported earlier this year, Raptor is moving its NASH program forward in anticipation of a future Phase 2b clinical trial.
In the next six months, the Company will be developing a commercial-ready formulation for this potential indication, designed as a delayed-release, coated compressed tablet dosage form of cysteamine bitartrate, which will be appropriate for the expected dose levels for NASH patients, anticipated to be significantly lower than the DR Cysteamine dosage for cystinosis patients. The Company plans to file an IND with the FDA by mid-2011 for a Phase 2b clinical trial to start when funding for this trial becomes available. In this regard, the Company is exploring partnering opportunities and potential grant support for the Phase 2b clinical trial.
ConviviaTM
The Company completed its first out-licensing agreement of ConviviaTM with Uni Pharma Co., Ltd in Taiwan in June 2010. Raptor has ongoing discussions with other Asian companies to potentially develop ConviviaTM further in various Asian markets.
Cysteamine Image via WikipediaInterest for Raptor Pharmaceuticals is really picking up in 2011. Here are the recent headlines for this up and coming Small Cap Pharmaceutical company. It has a Pipeline for Rare Diseases and I expect 2011 to be a strong year for Raptor Pharmaceuticals if Clinical Trial results are successful. Results for DR Cysteamine clinical trials is expected in 1Q 2011. DR Cysteamine has clinical development programs including:
◦Nephropathic Cystinosis, a rare genetic disorder (Phase 3)
◦Non-alcoholic Steatohepatitis (NASH), a metabolic disorder of the liver (Phase 2)
◦Huntington's Disease, an inherited neurodegenerative disease (Phase 2)
Raptor Pharmaceuticals Stock Quote: RTTP Google Finance
Read more from their previous 10-K filings for the company's Strategic Development Plan here.
Find out more about their Clinical Pipeline here.
Continued
Raptor Pharmaceutical Hires Patrick Reichenberger to Lead Commercial Development of DR Cysteamine for Nephropathic Cystinosis
Contracts in Place for Cambrex Corporation and Patheon, Inc. to Manufacture Active Pharmaceutical Ingredient and Commercial Product, Respectively
January 10, 2011 6:30 AM EST
NOVATO, Calif., Jan. 10, 2011 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTP) announced today the appointment of Patrick Reichenberger to the newly created position of Vice President, Commercial Operations. Additionally, Raptor has engaged Cambrex Corporation (NYSE: CMB) for the manufacturing of the active pharmaceutical ingredient ("API"), cysteamine bitartrate, and Patheon, Inc. (TSX: PTI) to produce the commercial product, DR Cysteamine.
A 20-year industry veteran with significant expertise in commercial operations and product-launch management, Mr. Reichenberger will lead the development and management of Raptor's sales and marketing efforts along with its commercial manufacturing, supply and distribution programs. Prior to joining Raptor, Mr. Reichenberger served as Senior Director of Commercial Development at XOMA, LLC. In this role, he led XOMA's commercial development department with particular emphasis on XOMA 052, an anti-IL1 antibody for diabetes, cardiovascular disease, and Behcet's Uveits, an orphan disease.
Prior to XOMA, Mr. Reichenberger was in charge of marketing, sales and distribution at Questcor Pharmaceuticals where he developed patient access and reimbursement programs supporting the successful commercial re-launch of H.P. Acthar® Gel for the treatment of infantile spasms, an ultra-orphan, pediatric disease. Prior to Questcor, he led commercial and strategic marketing for brands at Genentech, Athena Neurosciences, and Parke-Davis, where he assisted in the launch of Lipitor®.
"The addition of Patrick is particularly appropriate at this time as we work towards a potential commercial launch of DR Cysteamine for nephropathic cystinosis in 2012. His hands-on commercial development expertise will be central to our efforts to build the necessary commercial infrastructure and capability for the near term and as the Company grows in later years." stated Christopher M. Starr, Ph.D., Chief Executive Officer of Raptor.
Mr. Reichenberger stated "My experience in building commercial infrastructures around orphan therapeutics, especially Acthar® Gel, is particularly applicable to Raptor's nephropathic cystinosis program. I am encouraged by the enthusiastic patient and foundation support for Raptor's DR Cysteamine program, and I look forward to working to create a sound patient access program ready for commercial launch. While developing and implementing such a program for nephropathic cystinosis is my top priority at Raptor, I am also very excited about the entire Raptor pipeline. I am impressed with the commercial opportunities at Raptor with a near term program in an orphan indication for nephropathic cystinosis and mid-stage programs in other indications, including Huntington's Disease and NASH. I'm pleased to have the opportunity to use all of my capabilities, from long-term strategic planning to practical marketing and sales, in this position."
Ted Daley, President of Raptor stated "We are very pleased to welcome Patrick to lead our commercial efforts. The addition of Patrick to our leadership team, along with our recently established long-term manufacturing agreements with Cambrex and Patheon, are significant milestones in the continued advancement of our DR Cysteamine programs. Both Cambrex and Patheon have significant expertise working with cysteamine bitartrate. Cambrex has provided us with cysteamine bitartrate for all our clinical studies and, for over 10 years, has been one of the few API manufacturers worldwide that is capable of providing a commercial source of pharmaceutical-grade cysteamine bitartrate. Patheon developed Raptor's DR Cysteamine microbead formulation and currently manufactures our clinical drug supply. We look forward to employing their combined expertise and capabilities in our commercial manufacturer."
About Nephropathic Cystinosis
Nephropathic cystinosis is an inborn metabolic error characterized by the abnormal transport of cystine, an amino acid, out of the lysosomes. Failure to treat nephropathic cystinosis can cause serious health consequences, including renal failure and resultant kidney transplant, growth failure, rickets, photophobia and blindness. Symptom onset typically occurs within the first year of life, when cystine crystals accumulate in various tissues and organs, including the kidneys, brain, liver, thyroid, pancreas, muscles and eyes.
About Cysteamine and DR Cysteamine
DR Cysteamine is Raptor's proprietary enteric-coated, microbead oral formulation of cysteamine bitartrate designed to potentially reduce dosing frequency and gastrointestinal side effects associated with immediate-release cysteamine bitartrate, which is approved for sale by the FDA and EMA to treat nephropathic cystinosis, a rare, genetic lysosomal storage disease.
In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego for the development DR Cysteamine for nephropathic cystinosis and cysteamine for other potential indications including Huntington's Disease, NASH and Batten Disease.
About Raptor Pharmaceutical Corp.
Raptor Pharmaceutical Corp. (Nasdaq: RPTP) ("Raptor") is dedicated to speeding the delivery of new treatment options to patients by working to improve existing therapeutics through the application of highly specialized drug targeting platforms and formulation expertise. Raptor focuses on underserved patient populations where it can have the greatest potential impact. Raptor currently has product candidates in clinical development designed to potentially treat nephropathic cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase ("ALDH2") deficiency, and thrombotic disorder.
Raptor's preclinical programs are based upon bioengineered novel drug candidates and drug-targeting platforms derived from the human receptor-associated protein ("RAP") and related proteins that are designed to target cancer, neurodegenerative disorders and infectious diseases.
For additional information, please visit http://www.raptorpharma.com/.
Wedbush Starts Raptor Pharmaceuticals (RPTP) at Outperform
January 6, 2011 5:05 PM EST
Wedbush initiates coverage on Raptor Pharmaceuticals (NASDAQ: RPTP) with a Outperform rating and $7 price target.
The firm said, "We expect that Phase III data from RPTP’s lead DR Cysteamine program, anticipated in Q1:11, to be an important positive catalyst for the stock. Mature results from the Phase III results, in our opinion, will demonstrate non-inferiority to Cystagon as suggested by previous Phase IIa and IIb studies. Additionally, the drug, a delayed-release formulation of the approved active ingredient cysteamine bitartrate (Cystagon), should offer a significant improvement in quality of life and should demonstrate a more favorable safety profile with fewer side effects, in our opinion."
For more ratings news on Raptor Pharmaceuticals click here and for the rating history of Raptor Pharmaceuticals click here.
Shares of Raptor Pharmaceuticals closed at $3.39 yesterday, with a 52 week range of $1.41-$4.00.
Raptor Pharmaceuticals: A Pipeline With Potential
October 07, 2010
By: Tony Pelz-Seeking Alpha
In my continuing search for “off the beaten track” small cap biotechs with impending catalysts, I came across Raptor Pharmaceuticals (Ticker: RPTP). Despite its checkered past, RTPT possesses an interesting pipeline with numerous near-to-medium term catalysts. According to the Company,
Raptor currently has product candidates in clinical development designed to potentially treat nephropathic cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's Disease, aldehyde dehydrogenase deficiency and a non-opioid solution designed to potentially treat chronic pain.
The Company also has a small roster of pre-clinical products.
Here are the catalysts and associated estimated market values:
•DR Cysteamine (Cystinosis): Phase 3 started June 2010; data expected to be reported 4Q’10/1Q’11; est. market value: $100 million
•DR Cysteamine (NASH): Phase 2a data released 5/2010; IND filing for Phase 2b 1H’11; est. market value: $1.8 billion
•DR Cysteamine (Huntington’s Disease): Phase 2 planned initiation Q3’10; est. market value: $2.1 billion
•Convivia: Out-licensing discussions; est. market value: $1.8 billion
•Tezampanel/NGX-426: Phase 2/3 ready, out-licensing discussions; est. market value: $700 million
The core medium term value drivers of the business going forward are clearly the candidates targeting NASH and Huntington’s. For the NASH indication alone, the Company claims there are currently 7.5 million adults (25 million by 2025) suffering from the disease with no current approved treatments. Investors are obviously cognizant of this market potential as positive results from Phase 2a data released in May 2010 caused the shares to rocket from around $1.50 to over $3.85. In addition, the stock regained all of its losses in just three days following a highly dilutive stock offering back in August; there appears to be a floor to the share price – quite rare. The near term Phase 3 data (Cystinosis) expected in 4Q’10/1Q’11, although targeting a smaller market, should at the very least (if positive) provide some price stability given the expectations that the Company should eventually be positioned to generate organic cash flows in the near to medium term with an approved product – i.e. to survive long enough to get the other “big ticket” products approved.
On the financial side, RPTP maintains a cash balance of around $17.2 million with no debt. Raptor management believes existing cash levels, at current monthly cash burn, will last into December 2011 – this is a decent amount of leeway. The Company currently has around 30.1 million shares (41.9 million fully diluted) outstanding – if the shares begin to run, look out for additional equity raisings and associated dilution.
I am approaching this trade as more of a 1 year + horizon investment. I attempted to build a position with options for about 4 weeks but to no avail; far too illiquid with ridiculous spreads (4) – I would expect spreads to tighten up as word gets out about this Company and its potential. As such, I will be slowly building a common stock position. On that front, I don’t like the technical/chart for the stock. After the May 2010 price spike, the shares have flat lined into a tight range between $2.70 and $3.00 – also, this range has tighten significantly over the past several weeks. Usually, this tells me that the stock is about to make a big move in either direction (i.e. up or down). Thus, I intend to slowly build this position and not put on the entire trade in one lot. As options become more liquid, I may either trade out of the shares into a long synthetic, sell Calls against the position or sell Puts.
Looking forward, I don’t expect the stock to make a huge move on the near term Phase 3 data (if positive). That said, the Company’s other products and associated potential upside appear quite substantial. If these products begin to rack up decent data in the trials, not only will the market take notice (and hence the shares rise) but Raptor could become a decent takeover target.
Raptor Pharma (RPTP) Updates on DR Cysteamine Phase 3, Financials
August 24, 2010 6:49 AM EDT
Raptor Pharmaceutical Corp. (Nasdaq: RPTP), provides updates on timelines related to its clinical programs including the pivotal Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").
Financial Updates and Guidance
As of August 24, 2010, the Company has approximately $17.2 million in cash and cash equivalents taking into account approximately $14 million of proceeds, net of placement agent fees and expenses, the Company received in a private placement financing which closed on August 12, 2010. The Company anticipates that its cash and cash equivalents will support planned operations and its planned clinical development programs, as described below, into December 2011.
Cystinosis
Raptor's cystinosis program remains the highest priority for the Company. "Over the next 18 months, the majority of the Company's resources will be devoted to completing what we believe will be the final development phase of this program," said Christopher M. Starr, Ph.D., CEO of Raptor. "Anticipating the successful completion of our ongoing Phase 3 clinical trial in 2010 and NDA submission in 2011, we are building the commercial infrastructure in our anticipation of a commercial launch in 2012."
In November 2009, Raptor completed its Phase 2b clinical trial of DR Cysteamine in cystinosis. DR Cysteamine demonstrated improved tolerability and the potential to reduce total daily dosage and administration frequency compared to immediate-release cysteamine bitartrate.
On June 28, 2010, Raptor enrolled the first patient in its pivotal Phase 3 clinical trial in cystinosis. This trial is designed as a randomized, crossover, outpatient study of the safety, tolerability, pharmacokinetics ("PK") and pharmacodynamics ("PD") of DR Cysteamine compared to immediate-release cysteamine bitartrate in cystinosis patients. The study is being conducted at nine centers: four in the U.S., including Emory University, Children's Memorial Hospital at Northwestern University, Stanford University, and Texas Children's Hospital at Baylor University; and five in the EU including in Hopital Necker-Enfants Malades and Hopital Robert Debré in Paris, Hospices Civils de Lyon in Lyon, France, Centre Hospitalier Universitaire in Montpellier, France and Radboud University Medical Center in Nijmegen, The Netherlands.
"We are very excited to have begun this study and are pleased with the patient support and initial rate of enrollment we are seeing at our sites," said Craig Langman, M.D., the Isaac A. Abt, M.D., Professor of Kidney Diseases at Feinberg School of Medicine, Northwestern University in Chicago and Principal Investigator of the Phase 3 clinical trial. "We currently have three U.S. sites enrolling patients and a fourth to commence early next month. Our European sites will begin enrolling patients by mid-September".
The Company expects to complete patient enrollment in the next few months and anticipates that all patients will have completed the treatment period by the end of the year. The treatment period for each of the anticipated 30 patients in the clinical trial is a total of 9 weeks and all patients will continue on DR Cysteamine in an extension study following the treatment period.
If the results from this trial are successful, Raptor anticipates filing an NDA for DR Cysteamine in cystinosis in mid-2011 and is planning a concurrent filing with the EMA for registration in the EU.
Huntington's Disease
Raptor anticipates that the Phase 2 study using DR Cysteamine in 96 Huntington's Disease patients will start in the third quarter of 2010. The Phase 2 clinical trial will be conducted under a previously announced collaboration agreement with The Centre Hospitalier Universitaire d'Angers ("CHU d'Angers") of Angers, France. Clinical expenses of the study will be covered by a grant from the French government.
Under the collaboration agreement between the Company and CHU d'Angers, Raptor will supply the DR Cysteamine study drug and will retain commercial rights to the clinical trial results. This Phase 2 trial was contemplated based on encouraging preclinical results reported by Institut Curie, CNRS and Inserm scientists that cysteamine prevents death of neurons and increases levels of a brain growth factor called brain-derived neurotrophic factor ("BDNF") in Huntington's Disease models. Reduced levels of BDNF in the brain have been widely reported to be important in the clinical development of Huntington's Disease.
In addition to intellectual property licensed from University of California, San Diego ("UCSD") related to its DR Cysteamine programs, Raptor holds exclusive worldwide licenses to Huntington's Disease related intellectual property from the Weizmann Institute of Science in Israel and Japan's Niigata University.
Non-Alcoholic Steatohepatitis ("NASH")
Raptor's management views its DR Cysteamine development program in NASH as an exciting opportunity based on both its potential market size and the possibility of creating partnering relationships. Following encouraging results from its Phase 2a clinical trial of DR Cysteamine in juvenile NASH patients, reported earlier this year, Raptor is moving its NASH program forward in anticipation of a future Phase 2b clinical trial.
In the next six months, the Company will be developing a commercial-ready formulation for this potential indication, designed as a delayed-release, coated compressed tablet dosage form of cysteamine bitartrate, which will be appropriate for the expected dose levels for NASH patients, anticipated to be significantly lower than the DR Cysteamine dosage for cystinosis patients. The Company plans to file an IND with the FDA by mid-2011 for a Phase 2b clinical trial to start when funding for this trial becomes available. In this regard, the Company is exploring partnering opportunities and potential grant support for the Phase 2b clinical trial.
ConviviaTM
The Company completed its first out-licensing agreement of ConviviaTM with Uni Pharma Co., Ltd in Taiwan in June 2010. Raptor has ongoing discussions with other Asian companies to potentially develop ConviviaTM further in various Asian markets.
Related articles
- Xoma snags $505 million pact on its top drug prospect (fiercebiotech.com)
- Small-cap Biotech Stock of the Day; XOMA Limited - Beacon Equity Research (news.google.com)
- Biotech. Stocks To Keep An Eye On (XOMA, GILD, STEM, CTIC) - EFPR (press release) (news.google.com)
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