January 12, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor
There might not have been a turf war between BioMarin and Sarepta before today but there sure is now, after BioMarin CEO Jean-Jacques Bienaime told the J.P. Morgan Healthcare Conference that the company’s Duchenne muscular dystrophy drug, drisapersen, is clearly more effective than its closest competitor, Sarepta‘s drug eteplirsen—and he had the slides to prove it.
Bienaime made the comparison at the J.P. Morgan Healthcare Conference which begins Monday in San Francisco and the oldest and largest conference of its type. It will include 300 of the largest biotech, healthcare and biopharma companies presenting their top-line data and estimates to a sea of eager bankers, analysts, institutional investors, hedge funds and journalists.
Bienaime drew the comparison between the two drugs despite no prior testing pitting them head-to-head. He said the BioMarin data was on a more solid footing because it includes patients who lost ambulation, while Sarepta’s eteplirsen excludes non-ambulatory patients. But when that data is similarly censored, Bienaime said drisapersen compares more favorably—and will achieve a market edge because of it.
That type of direct, full-contact product tackling is unusual in the biotech world, though analysts have questioned before whether the two will be able to co-exist in the relatively small Duchenne’s market.
BioMarin illustrated the difference with two slides during its presentation (see images).
BioMarin has a lot riding on the success of drisapersen. The company bought Dutch biotech Prosensa in November for $680 million in cash, plus an additional $160 million more in near-term milestones drisapersen can receive approval from the U.S. Food and Drug Administration when presented later this year.
At the time, the deal was considered a lemon, primarily because drisapersen had already failed a pivotal Phase III for DMD, which cost it its previous patron, GlaxoSmithKline. Now, however, Bienaime feels confident that the drug can go toe-to-toe with Sarepta—a charge the company will have a chance to answer later in the week when it presents at J.P. Morgan on Thursday.
Some analysts did think it was a solid deal for investors as long as ProSensa’s drug candidates complete crucial trials, said Jeremiah Shepherd, a biotech analyst for Credit Suisse, said at the time.
“Contingent Value Rights agreements appear achievable to us: We arrive at a premium to the offer price by probability adjusting the NPV of the anticipated CVRs by 80 percent,” wrote Shepherd in a note to investors. “We believe that both CVRs are achievable as RNA should have additional data to supplement the expected NDA and MAA filings ahead of the potential approvals.”
Under the terms of the deal, BioMarin will buy all of Prosensa’s issued and outstanding shares and all ordinary share equivalents with cash for $17.75 per share. There are then two built-in regulatory milestones that will have to be met before the Prosensa can cash in: The first for U.S. approval of drisapersen in the U.S. no later than May 15, 2016, and the second for approval in Europe no later than Feb. 15, 2017.
Those milestones theoretically appear achievable, said Shepherd in his note. “Rolling NDA still on track for completion in first quarter of 2015. The final module is still expected to be submitted in the first quarter of 2015 and is likely to be evaluated under a priority review,” he said. “The CVR for U.S. accelerated approval has a deadline of May 15, 2016 which should provide sufficient breathing room to address any issues the FDA may have with the NDA filing.”
