UPDATED: BioMarin sells its PhIII cancer drug to Medivation for $570M

Biomarin CMO Hank Fuchs

Setting its sights squarely on a future focused on rare diseases, BioMarin ($BMRN) has opted to out-license its Phase III PARP inhibitor to Medivation for $410 million upfront with another $160 million in milestones.

Medivation ($MDVN) is getting talazoparib (BMN673) in the deal, which headed into Phase III back in 2013 for advanced breast cancer among patients with BRCA 1 or BRCA 2 mutations.

Medivation has enjoyed considerable success with its prostate cancer pill Xtandi, though it has paled occasionally in comparison to the rival Zytiga marketed by J&J ($JNJ). Now Medivation has a shot at a near-term add-on to the portfolio--but faces a small gang of competitors.

The price, though, is likely to raise a few eyebrows in the cancer field. Late-stage cancer assets in particular can often snag a bigger set of milestones than this these days, with a number of big players competing for programs. Medivation CEO David Hung has a rep as an effective dealmaker, though, and there are still plenty of unanswered questions about the market potential for PARP inhibitors. 

Neither company came out ahead on the news this morning. BioMarin shares are down 5% in a rocky market with Medivation shares sliding 6%.

Barclays' Geoff Meacham concluded that the deal is a distinct plus for Medivation, which can leverage the drug in combination with Xtandi and look for broader use outside of an initially limited patient population. Talazoparib is slated for a 2017 top-line readout for Phase III data, though Meacham sees a chance for that timeline to advance into 2016.

The PARP spotlight has been centered on AstraZeneca's ($AZN) Lynparza and Clovis Oncology's ($CLVS) rucaparib, which is also targeted on BRCA mutations and which won the FDA's breakthrough drug designation last spring. AstraZeneca was the first to market, though, winning an approval for Lynparza based on Phase II data among patients with defective BRCA genes. And Tesaro ($TSRO) is advancing niraparib (formerly MK-4827) for ovarian and breast cancer. Late-stage data for all of these drugs will ultimately determine how they fare on the market.

BioMarin, meanwhile, has been building its reputation in the rare disease arena. The late-stage focus at the biotech has centered on an experimental therapy for Duchenne muscular dystrophy as well as vosoritide (BMN 111), which is designed to correct dwarfism.

"Medivation's expertise and track record in oncology clinical development and commercialization has been well demonstrated by the Company's success to date," says BioMarin CMO Hank Fuchs. "Placing talazoparib in their capable hands allows us to optimize our portfolio and focus our resources on established areas of expertise--developing novel products to treat rare and ultra-rare genetic diseases."

- here's the release

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