Drug/indication: Blisibimod for lupus (phase II trial)
Top-line results expected at the end of the second quarter
The "PEARL-SC" trial randomizes lupus patients to treatment with blisibimod or placebo with a primary endpoint of clinical improvement at 24 weeks in the SLE responder index, the same endpoint that Human Genome Sciences used to demonstrate the clinical efficacy of Benylsta.
Drug/indication: Tivantinib in non-small cell lung cancer (phase III)
Top-line results expected in second half of 2012
The "MARQUEE" trial compares tivantinib plus Takeda's Tarceva against Tarceva alone in previously treated non-small cell lung cancer patients. The study's primary endpoint is overall survival.
Arqule and partner Daiichi Sankyo will conduct an interim analysis of the tivantinib study after 50% of the planned patient deaths have occurred. This is the top-line result that will be ready in the second half of 2012. The final analysis of the trial isn't expected until next year.
Drug/indication: Xiaflex for Peyronie's disease (Phase III trial)
Top-line results expected before end of June
Peyronie's is a buildup of scar tissue that causes extreme (and uncomfortable) curvature of the penis during an erection. Auxilium's "IMPRESS" study compares Xiaflex to placebo injections with co-primary endpoints of improvement in curvature and quality of life measure.
Xiaflex is currently approved for Dupuytren's Contracture, a similar disease that causes involuntary curling of the fingers. Expansion into Peyronies could be a $300-500 million market opportunity for Auxilium.
Drug/indication: Long-lasting rFactor VIII for hemophilia A; long-lasting rFactor IX for hemophilia B; dexpramipexole for amyotrophic lateral sclerosis (ALS) or Lou Gehrig's disease. (All phase III trials)
Top-line results expected in the second half 2012
With its hemophilia and ALS programs, Biogen has the opportunity to diversify outside of its dominant position in multiple sclerosis. The dexpramipexole trial carries the biggest risk but also the most potential upside given a lack of effective therapies for ALS patients. Data from the phase III "EMPOWER" study are expected in the fourth quarter.
Biomarin Pharmaceuticals (BMRN)
Drug/indication: GALNS for MPS IV also known as Morquio Syndrome (Phase III trial)
Top-line results expected in the fourth quarter 2012
Positive data from orphan disease drug studies have a jumbo-sized effect on stock prices. (See Alexion Pharmaceuticals and Vertex Pharmaceuticals.) The phase III study of GALNS in MPS IV patients, if successful, could double Biomarin's revenue over the next five years and be the trigger for a takeout by a large pharmaceutical company eager to profit from the booming business of developing new therapies for rare, genetic diseases.
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