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12:00 AM
Dec 08, 2014
 |  BioCentury  |  Strategy

BioMarin bulks up

Why BioMarin buying Prosensa despite drisapersen Phase III miss in DMD

Five years after discussing a licensing deal with Prosensa Holding N.V., BioMarin Pharmaceutical Inc. decided to acquire the Duchenne muscular dystrophy play to get what could become the California company's largest drug. BioMarin believes it can win accelerated approval for Prosensa's drisapersen despite the fact that the compound missed the primary endpoint in its lone Phase III trial.

On Nov. 24, BioMarin said it would acquire Prosensa for $17.75 per share in cash, or about $680 million. Prosensa shareholders also will receive a contingent value right (CVR) worth up to $4.14 per share, or $160 million.

In 2009, BioMarin lost out to GlaxoSmithKline plc, which licensed drisapersen and took options for three other compounds for £16 million ($25.2 million) up front and up to £412 million ($649 million) in milestones. GSK returned rights to Prosensa in January.

Drisapersen is likely to be the first product reviewed in the U.S. for DMD, for which it has breakthrough therapy and Orphan drug designations.

The antisense oligoribonucleotide, which induces exon 51 skipping on the dystrophin gene, could treat about 13% of DMD cases, or about 10,000 patients. BioMarin also will gain five follow-on DMD compounds that address another 35,000 DMD patients, plus two exon-skipping compounds for Huntington's disease and myotonic dystrophy.

Drisapersen will join a portfolio that comprises mostly ERTs for lysosomal storage disorders and includes five marketed ultra-Orphan drugs.

But first, BioMarin will have to persuade regulatory agencies that the totality of the data for drisapersen justify approval. The company believes it has identified a compelling reason for the Phase III miss, and that its experience gaining approval for rare disease compounds - including one that was approved despite missed Phase III endpoints - will be an asset.

"We have the experience of dealing with authorities when indeed the data need some interpretation," BioMarin CEO Jean-Jacques Bienaimé said last week at the Piper Jaffray Healthcare conference. "This asset is valued more in our hands than it is in theirs."

Explaining the miss

BioMarin could not provide an interview in time for publication and Prosensa declined to be interviewed, but on a Nov. 24 conference call to discuss the deal, EVP and CMO Henry Fuchs acknowledged BioMarin will have to explain why results from Phase III differed "substantially" from those in Phase II.

A 6 mg dose of drisapersen met the primary endpoint of improving 6MWD...

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