The FDA altered its view of Sarepta Therapeutics Inc.’s data with exon-skipping therapy eteplirsen for Duchenne muscular dystrophy (DMD) after Prosensa Holding NV’s failure with exon-skipper drisapersen for the condition, but Sarepta CEO Chris Garabedian hopes the agency can still be brought around. Read More
China isn’t Asia. That’s certainly one take-away from the 2013 BIO Convention in China, or BIO China. The learning curve is steep for Western biopharmas seeking a toehold in Asia’s largest market, with 1 .4 billion people, and if companies accustomed to doing business in Japan and South Korea expect the same conditions in China, they’re doomed to disappointment. Read More
Briefing documents released ahead of Thursday’s FDA advisory committee meeting regarding Vanda Pharmaceuticals Inc.’s therapy for non-24-hour disorder, tasimelteon, revealed an ongoing disconnect between the sponsor and agency regarding clinical endpoints for the disorder, whose symptoms can be difficult to pin down. Read More
Recognizing that an initial public offering (IPO) isn’t the best route for all small companies in need of capital, the Equity Capital Formation Task Force is pushing the SEC to lift the barriers on Regulation A offerings immediately. Read More
Scenesse (afamelanotide), which Clinuvel Pharmaceuticals Ltd. is developing for the rare light intolerance disorder erythropoietic protoporphyria (EPP), failed to hit the endpoint of a U.S. Phase III trial. Read More
To be of any use at all, scientific experiments need to be reproducible. And it has become increasingly clear over the past few years that far too many of them aren’t. Read More
• Bioasis Technologies Inc., of Vancouver, British Columbia, said new research in animals conducted under its BT2111 program showed that the drug, a conjugate of Herceptin (trastuzumab, Roche AG), not only crosses the blood-brain barrier, but also penetrates the blood-tumor barrier up to 10 times better than Herceptin alone. Read More
• Galectin Therapeutics Inc., of Norcross, Ga., said five of the eight patients have been enrolled and infused in the first cohort of its blinded Phase 1 trial of GR-MD-02 for patients with nonalcoholic steatohepatitis (NASH or fatty liver disease) with advanced fibrosis. Read More
• Helsinn Group, of Lugano, Switzerland, said it granted Chugai Pharma Marketing Ltd., a wholly owned subsidiary of Chugai Pharmaceutical Co. Ltd., of Tokyo, exclusive commercialization rights to Phase III-stage ghrelin receptor agonist, anamorelin, in Germany, France, Benelux, UK and Ireland. Read More
