BioMarin ($BMRN) 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 $160 million more in near-term milestones if it can land an early approval for its late-stage drug for Duchenne muscular dystrophy.
The buyout, which immediately triggered a 53% spike in Prosensa's share price, gives BioMarin control of drisapersen, a drug that failed a pivotal Phase III for DMD and lost its Big Pharma chaperone, GlaxoSmithKline ($GSK). With the deal BioMarin, which has enjoyed commercial success in rare diseases, finds itself competing with Sarepta ($SRPT) and PTC Therapeutics ($PTCT) in the competition to hustle ahead new DMD drugs to regulators.
BioMarin is paying $17.75 a share for Prosensa and will add two milestones of $80 million each if it can land a U.S. approval by a May 15, 2016, deadline and a Europe approval no later than February 15, 2017.
Prosensa's drug failed to beat a placebo in significantly improving boys' ability to complete a 6-minute walk test--the classic measure of success in this field. But left to its own devices the biotech, a 2012 Fierce 15 winner, completed fresh analysis of new extension study data that backed a theory that providing the drug earlier while extending treatment could delay disease progression. The biotech held what it called "positive" reviews with the FDA and began a rolling submission of the application just weeks ago. Evidently BioMarin has high hopes of success where GlaxoSmithKline saw nothing but failure.
|BioMarin CEO Jean-Jacques Bienaime|
"We will leverage our experience at developing rare disease therapies to achieve regulatory approvals and bring drisapersen to market as quickly as possible," said BioMarin CEO Jean-Jacques Bienaime. "Further, if we are successful in advancing drisapersen to early regulatory approvals, we believe this transaction would be accretive to operating and GAAP profitability in 2017." If the transaction falls through, BioMarin is committed to acquire a $50 million equity stake in the biotech.
All three leading companies in DMD have been on an unending roller coaster ride. Sarepta's experienced a series of shocks and excitements, with the CEO advancing and then reeling back in his statements about the FDA's willingness to accept limited--though very encouraging--data sets on its DMD drug (which like drisapersen also relies on exon skipping to restore dystrophin and slow the rate of decline for boys with this lethal disease). And PTC, which has only reported failure in the clinic, was first slapped by European regulators after filing for early approval and then unexpectedly embraced.
Patient groups have played a big role in DMD. Boys afflicted with the disease have no current treatments to turn to, which has apparently persuaded regulators to take a more open-minded approach to the risk-benefit equation they use in evaluating new drug approvals. That may short-circuit the process in the U.S., which is why BioMarin may be making this gamble now after GlaxoSmithKline decided to walk.
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