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.
Prosensa picked the right day to price its IPO. The Dutch biotech rang up the offering at $13 a share, at the top of the range, raising $78 million after bumping the number of shares on offer to 6 million. The stock will trade on Nasdaq as "RNA."
GlaxoSmithKline has landed the FDA's coveted breakthrough therapy designation for drisapersen, a late-stage treatment for Duchenne muscular dystrophy that is engaged in a fierce clinical race with Sarepta's closely watched DMD drug eteplirsen.
The former Genzyme CEO and chairman has joined the biotech Prosensa as a strategic adviser, bringing his experience in building Genzyme into a world leader in rare diseases.
Now, more than ever, the life sciences industry is all about innovative and disruptive technologies. Every year for the past decade, FierceBiotech has made its picks on which companies hold the best odds for success in our Fierce 15 report. This year, though, we've added another Fierce 15 to focus on medical devices and diagnostics. I'd like to encourage readers to consider the differences by comparing the companies in each report.