Sarepta, jockeying with rival BioMarin to commercialize a treatment for Duchenne muscular dystrophy, is looking to buy up complementary assets to fortify its position. But the company's better-funded competitor believes its long history in rare disease R&D will give it a leg up as the pair move toward make-or-break FDA decisions.
BioMarin just bought itself a pricey ticket into a high-profile Phase III drug development race. The rare-disease specialist has stepped in to buy the Dutch biotech Prosensa for $680 million in cash while offering up to $160 million more in near-term milestones if it can land an early approval for its late-stage drug for Duchenne muscular dystrophy.
Prosensa has begun the FDA submission process for its treatment for Duchenne muscular dystrophy, planning to fully file by year's end with hopes of finally winning approval for a drug that has survived a few brushes with failure.
Prosensa, one of a handful of biotechs racing to develop treatments for Duchenne muscular dystrophy, has secured $7 million from a nonprofit to accelerate its R&D efforts.
After a late-stage failure and a kiss-off from partner GlaxoSmithKline, Prosensa and its innovative treatment for Duchenne muscular dystrophy seemed to be on a downward slope. But now, after a series of "positive" meetings with the FDA, the company says it plans to submit its breakthrough-designated drug for approval this year, taking advantage of the agency's accelerated pathway.
After the markets closed last night, Prosensa issued an upbeat self-assessment of its 48-week extension data from a Phase II study of its lead drug drisapersen for Duchenne muscular dystrophy.
Prosensa's Duchenne muscular dystrophy treatment endured a Phase III setback that led partner GlaxoSmithKline to head for the exit, but the Dutch biotech is back with a new analysis of drisapersen's data that could spell a path forward, sending its shares up 25%.
GlaxoSmithKline has shut the door on its hopes in Duchenne muscular dystrophy, handing back rare-disease drug drisapersen to Prosensa, its former partner.
A Danish biotech company with a foothold in the U.S. is making a bid to cash in on the big IPO boom on Nasdaq. Egalet, which has been developing abuse-resistant opioid pain pills, has outlined plans to raise $69 million from an initial offering. That cash would primarily be used to develop a couple of late-stage programs nearing the clinic.
GlaxoSmithKline and Prosensa today conceded defeat in the Phase III study for drisapersen, one of two closely-watched therapies for Duchenne muscular dystrophy which had been vying for the lead in the field. Investigators reported that the therapy failed to significantly improve walking distance in patients.