Wednesday, July 14, 2010

as reported by The Street 10 Firms Expecting Clinical Trial Results

10 Firms Expecting Clinical Trial Results
By Adam Feuerstein

BOSTON (TheStreet) -- I've put together an updated calendar of meaningful clinical trials with results expected through the end of the current year. The biotech and drug companies included here are listed in alphabetical order.

Unlike U.S. Food and Drug Administration drug approvals, which are more precisely timed, the release dates for data from clinical trials is a bit fuzzier, so the timelines here are culled from company guidance when possible.
Successful biotech traders and investors understand the importance of staying one step ahead of stock-moving catalysts in the biotech sector. Use this clinical trial list as a companion to my recently published FDA drug approval calendar.

This list was compiled using company reports and the BioMedTracker service from Sagient Research, a subscription-based tool for keeping track of biotech and drug catalysts.

Read on for the list.

Company: Ariad Pharmaceuticals (ARIA)
Drug/indication: Ridaforolimus for sarcoma
Estimated timing: Before year's end.
Recent stock performance: Closed Tuesday at $2.73, off its recent high of $4.25 reached in mid May.
The phase III "SUCCEED" study is designed to detect a 33% improvement in median progression-free survival between "rida" and placebo in patients with soft-tissue or bone sarcoma who were previously treated, successfully, with chemotherapy. Full enrollment in the study of 650 patients was reached in December 2009 and independent study monitors completed their last interim check in May, recommending the study proceed to a final analysis.
Ariad licensed worldwide commercial rights to rida to Merck(MRK). The two companies recently restructured the terms of the partnership.

Company: AstraZeneca (AZN)
Drug/indication: Zibotentan for prostate cancer
Estimated timing: Fourth quarter
Recent stock performance: A rebound in June and July has pushed the stock up 6% for the year.
Zibotentan represents the biggest, near-term competitive threat to Dendreon's(DNDN) prostate cancer immunotherapy Provenge. The phase III study of zibotentan targets the same patient population described in Provenge's FDA-approved label -- men with prostate cancer that no longer respond to hormone treatment but who have minimal bone pain and have not yet started chemotherapy.
The study of 580 men compares treatment with zibotentan against best supportive care with a primary endpoint of overall survival. If the study is positive and zibotentan gains FDA approval, doctors will have another treatment choice besides Provenge for this group of prostate cancer patients.

Company: BioMarin Pharmaceuticals (BMRN)
Drug/indication: Peg-Pal for phenylketonuria (PKU)
Estimated timing: Third quarter
Recent stock performance: At $19, the stock is flat for the year, off its recent high of $25 reached in May.
Peg-Pal is a follow-on therapy for PKU that is being developed as a treatment for patients who do not respond to Biomarin's currently marketed PKU drug Kuvan. This phase II study treats 35 patients with PKU for 16 weeks.

Company: Bristol-Myers Squibb (BMY)
Drug/indication: Ipilimumab for melanoma
Estimated timing: Fourth quarter
Recent stock performance: At almost $26, trading close to its year high
Bristol was the star of the most recent ASCO cancer conference where the company presented positive results from a phase III study of ipilimumab in previously treated melanoma patients. Bristol plans to seek FDA approval for ipilimumab based on these data despite some lingering questions about the strength of the results.
Bristol can put all the questions about ipilimumab to rest with boffo results from this next phase III study in 500 first-line, or newly treated melanoma patients. The study compares ipilimumab plus the chemotherapy DTIC against DTIC plus a placebo, with overall survival as the primary endpoint.

Company: Corcept Therapeutics (CORT)
Drug/indication: Corlux for Cushing's syndrome
Estimated timing: Fourth quarter
Recent stock performance: Up more than 20% for the year.
Cushing's syndrome is a rare disease in which patients suffer from high blood pressure, elevated blood sugar and/or diabetes and other symptoms because of exposure to high levels of the "stress hormone" cortisol.
Corcept enrolled 50 Cushing's patients in its pivotal, phase III study in which all patients are being treated with Corlux for 24 weeks. The primary endpoint of the study is improvement in glucose tolerance or a decrease in diastolic blood pressure relative to baseline. The active ingredient in Corlux is mifepristone, also known as the abortion drug RU-486

Company: Cyclacel Pharmaceuticals (CYCC)
Drug/indication: Seliciclib for non-small cell lung cancer
Expected timing: Before year's end
Recent stock performance: At around $1.50, the stock has been cut in half from the $3 level reached in January.
Last January, investors bid up shares of Cyclacel on the publication of research demonstrating that seliciclib was effective against certain lung cancer cells -- in a petri dish. Some of that investor excitement for the stock has ebbed in the subsequent seven months, but could be rekindled if Cyclacel posts positive results from this phase II study in non-small cell lung cancer patients. The phase II study is somewhat unique because it uses a follows a "randomized discontinuation" design that was most famously used to test the initial efficacy of Onyx Pharmaceutical's cancer drug Nexavar. In this case, all lung cancer patients are first treated with seliciclib for six weeks. Patients whose tumors remain stable at a minimum after this run-in treatment phase are then randomized to continue treatment with seliciclib or receive best supportive care. The study's primary endpoint is progression-free survival measured in the randomized portion of the study.

Company: Merck
Drug/indication: Boceprevir for hepatitis C
Estimated timing: Third quarter
Recent stock performance: Flat for the year
Vertex Pharmaceuticals (VRTX) set the efficacy bar for new hepatitis C drugs when the company's experimental drug telaprevir posted a 75% "cure" rate in a pivotal phase III study of newly diagnosed patients.
Investors await results from two pivotal studies of Merck's boceprevir in newly diagnosed hepatitis C patients as well as in patients who failed to respond to previous treatment. Likewise, Vertex is expected to announce results later this year from additional late-stage studies of telaprevir in previously treated patients.

Company: Onyx Pharmaceuticals (ONXX)
Drug/indication: Carfilzomib for multiple myeloma
Estimated timing: Third quarter
Recent stock performance: Down 28% and near its 52-week low.
Onyx acquired the next-generation proteasome inhibitor carfilzomib when the company bought the privately held drug developer Proteolix in October 2009. This "pivotal" phase IIb study enrolled 269 patients with multiple myeloma that continued to grow despite two or three prior therapies. If carfilzomib can shrink tumors in a significant portion of these patients, Onyx intends to seek accelerated approval with the FDA.

Company: Raptor Pharmaceuticals(RPTP)
Drug/indication: DR Cysteamine for cystinosis
Expected timing: Fourth quarter
Recent stock performance: At $2.60, up for the year but still below the spike to $4 which occurred last October.
DR Cysteamine is a proprietary formulation designed to require less frequent dosing (every 12 hours versus every six hours) and cause fewer gastrointestinal side effects than immediate-release cysteamine for patients with cystinosis. Cystinosis is a rare genetic disease caused by the buildup of the amino acid cystine within cells. Patients with cystinosis develop severe kidney problems and suffer from impaired growth. Raptor's phase III study compares DR Cysteamine to immediate release cysteamine.

Company: Seattle Genetics(SGEN)
Drug/indication: Lintuzumab (SGN-33) for acute myeloid leukemia
Estimated timing: Third quarter
Recent stock performance: Up 22% for the year.
Seattle Genetics scores points for designing this pivotal lintuzumab study in AML with a control arm and a survival benefit endpoint, unlike recent AML single-arm studies conducted by Genzyme(GENZ) and Vion Pharmaceuticals that failed to pass muster with FDA or the agency's panel of outside cancer experts.
If lintuzumab can score a win in this study by helping patients with AML live longer, FDA approval should be relatively straightforward. However, the patients enrolled in the lintuzumab study are elderly (60 years or older) and very sick with an aggressive form of blood cancer that has proven resistant to effective treatment with previous drugs. Data from an earlier and much smaller study of lintuzumab in elderly AML patients showed some promising hints of tumor remission.

-- Reported by Adam Feuerstein in Boston.

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