Tag Archives: rna

Why Muscular Dystrophy Is Biotech’s Favorite Orphan

With tax-sheltering deals off the table for now, drugmakers have turned back to their traditional prey: hot biotech startups. But what investors want to know is: What kind of biotechnology are the startups looking for? One possible sign came in November, when big-cap biotech BioMarin Pharmaceutical (BMRN) agreed to pay $680 million for Prosensa Holding (RNA). Prosensa was preparing to file for approval of drisapersen, a treatment for Duchenne

BioMarin CEO Sees Big Future For Prosensa Drug

BioMarin Pharmaceuticals raised a few eyebrows last month when it announced its latest buyout. BioMarin (BMRN) agreed to pay $680 million upfront for Prosensa Holding (RNA), a Dutch biotech developing a drug for Duchenne muscular dystrophy called drisapersen. Drisapersen succeeded in its phase-two trial but failed its phase three, causing partner GlaxoSmithKline (GSK) to end their relationship. At BioMarin’s investor day on Dec. 10, executives

BioMarin Buying Prosensa, Backing Up Troubled Drug

Biotech BioMarin Pharmaceutical (BMRN) agreed Monday to pay $680 million in cash for Prosensa Holding, developer of a muscular-dystrophy drug with a checkered history. Prosensa soared 63%, while BioMarin slipped a fraction in early trading in the stock market today. The deal also included two milestone payments of $80 million each if Prosensa’s (RNA) lead drug drisapersen is approved for Duchenne’s muscular dystrophy (DMD) in the U.S. no later