The event: Phase IIb study of SB-509 in diabetic neuropathy.
The timeline: Top-line results to be announced in the second half of 2011.
The stakes: The legitimacy of zinc-finger drug technology, which Sangamo has been developing for years on its own. SB-509 is an engineered protein, or "zinc finger," specifically designed to turn on a gene to restart nerve growth. Previously reported data from earlier studies of SB-509 in diabetic neuropathy have produced mixed results and a significant level of investor skepticism about the technology. Sangamo has the opportunity with the looming results from the phase IIb study in diabetic neuropathy to silence the critics and push SB-509 and the entire zinc-finger drug technology platform forward into pivotal late-stage studies and perhaps lucrative partnership deals. To get there, the data from the study need to demonstrate that SB-509 can improve the health and function of damaged nerves.
The event: FDA approval decision for Adcetris in two cancers -- relapsed or refractory Hodgkin's lymphoma, and relapsed or refractory systemic anaplastic large cell lymphoma (ALCL).
The timeline: The FDA approval decision is expected Aug. 30. Before that, on July 14, the FDA is convening an FDA advisory panel to review Adcetris.
The stakes: Seattle Genetics carries a market value of $2.4 billion, which means investors have not only banked on Adcetris' approval but are also expecting the drug to perform well commercially. Two important firsts hinge on Adcetris: It will be the first approved drug to emerge from Seattle Genetics' lab; and the first drug to validate the company's antibody drug conjugate technology. Adcetris combines the tumor-targeting precision of a monoclonal antibody with the tumor-killing power of chemotherapy.
The event: Phase III efficacy studies of Libigel for the treatment of female sexual dysfunction.
The timeline: Top-line results expected in the fall.
The stakes: A female Viagra, sort of. BioSante is trying to succeed where other drug companies have failed -- to be the first company to receive U.S. approval for a drug to boost the sexual "desire" of post-menopausal women.
The event: Phase II study of CYT387 in myelofibrosis.
The timeline: Data at the American Society of Hematology (ASH) annual meeting, Dec. 10-13, 2011. An early peek at the CYT387 data could come in early November when ASH releases research abstracts for its annual meeting.
The stakes: YM BioSciences suffers from a credibility deficit. The company believes CYT387 is a best-in-class drug for myelofibrosis, capable of shrinking enlarged spleens, and most importantly reducing anemia without causing too many harmful side effects. Investors and potential partners want to see more data on CYT387 before they buy into YM Bioscience's story, which makes the ongoing phase II study critical for the company's future.
The event: Phase III study of cabozantinib in medullary thyroid cancer.
The timeline: Top-line results in the third quarter.
The stakes: Investors have been most focused on the development of cabozantinib in prostate cancer (phase III studies in the design stage) but don't forget that Exelixis' first shot at getting the drug approved will come in medullary thyroid cancer (MTC) if results from a soon-to-be-completed phase III study come out positive. Medullary thyroid cancer is rare so Exelixis' revenue-generating opportunity will be small. Nonetheless, as a proving ground for the much larger and more lucrative prostate cancer opportunity, the cabozantinib data in MTC are very important to Exelixis and is likely to have significant impact on the company's stock price.
The event: Phase III study of Stimuvax in non-small cell lung cancer.
The timeline: Results of an interim analysis in the second half of the year.
The stakes: A chance for a coveted place atop the cancer immunotherapy mountaintop (joining Dendreon and Bristol Myers-Squibb.) Stimuvax is designed to work by stimulating a patient's immune system to seek out and destroy cancer cells that express a protein known as MUC1. The phase III study is enrolling 1,400 patients with unresectable stage III non-small cell lung cancer, randomized to treatment with Stimuvax or a placebo. An interim analysis will be conducted after 75% of deaths in the study have taken place, which the company estimates will happen in the second half of the year. Three scenarios are possible: 1) the study continues to a final analysis in 2012; 2) the Stimuvax survival benefit is so great that the study is stopped early with a big win; or 3) Stimuvax treatment isn't working at all and won't help patients live longer so the study is halted for futility.
The event: FDA approval decision for Intermezzo, a sleeping pill designed for people who wake up in the middle of the night.
The timeline: The FDA approval decision is expected on or before July 14.
The stakes: Intermezzo's active ingredient is the same as what's found in the popular sleeping pill Ambien although at a lower dose and formulated to dissolve easily under the tongue. What makes Intermezzo unique and somewhat risky is the proposed indication as the first-of-its-kind prescription sleep aid for people who wake in the middle of the night but cannot fall back to sleep. To get Intermezzo approved, Transcept must convince the FDA that the drug can be packaged and used correctly to reduce the risk of dosing errors or impaired next-day driving ability.
The event: Phase III data on the Quad, a new four-in-one combination pill for HIV.
The timeline: Data from two phase III studies (Quad vs. Atripla and Quad vs. boosted Reyataz and Truvada) are due in the second half of the year.
The stakes: The long-term growth of Gilead's market-leading HIV drug franchise. The Quad is a single pill made up four Gilead drugs: Currently approved Viread and Emtriva plus two experimental drugs elvitegravir and cobicistat. If approved, the Quad could extend Gilead's competitive advantage in HIV therapy especially as generic competition looms in the 2017-2018 time frame. The Quad will also be more profitable for Gilead since it will likely replace Atripla, a three-in-one combination pill that includes one drug from Bristol-Myers Squibb.
The event: Interim analysis of the phase III prostate cancer study of MDV3100.
The timeline: Fourth quarter of the year although the timeline may slip into 2012.
The stakes: The AFFIRM trial in advanced prostate cancer, if positive, will help keep Medivation's MDV3100 on pace with Johnson & Johnson's Zytiga, approved earlier this year for the same patient population. Both companies are also testing their drugs in patients with less advanced prostate cancer, a bigger commercial market.
The event: U.S. approval filing and acceptance of Lymphoseek for the detection of lymph nodes following cancer diagnosis.
The timeline: Third-quarter filing, followed 60 days later by FDA acceptance or rejection of the filing.
Neoprobe is adamant that the completed Lymphoseek clinical trials were designed with FDA input and are sufficient for an approval filing and subsequent FDA review. Neoprobe short-sellers believe shortcuts were taken with the Lymphoseek clinical trials, which could force the FDA to issue a refuse-to-file letter and cause a significant delay in the approval process.
Who's right? We'll know if or when the FDA accepts the Lymphoseek approval filing for review. Acceptance of the new drug application by the FDA doesn't guarantee Lymphoseek's eventual approval, but it will put a dent in the shorts' thesis and buoy the confidence of Neoprobe and its shareholders. >>To see these stocks in action, visit the 10 Biotech Trades for Second Half of 2011 portfolio on Stockpickr. Sources: BioMedTracker.com, company and analyst reports, TheStreet research. --Written by Adam Feuerstein in Boston. >To contact the writer of this article, click here: Adam Feuerstein. >To follow the writer on Twitter, go to http://twitter.com/adamfeuerstein. >To submit a news tip, send an email to: firstname.lastname@example.org.