On November 24th rare disease drug maker BioMarin Pharmaceuticals announced it was buying Prosensa for $840MM plus approval milestones. Prosensa has no marketed products but is in the late stages of obtaining approval for its lead drug candidate, drisapersen, for a rare form of muscular dystrophy. BioMarin will pay Prosensa shareholders an additional $80MM if drisapersen receives U.S. regulatory approval before May 2016, and another $80MM if the drug is approved in Europe before February 2017.
Drisapersen failed a late stage trial in 2013 but FDA agreed to look at new analysis of the data. Some analysts are calling BioMarin’s move a “call option” but this would not be unusual given activity in the RNAi space. Prosensa’s previous partner for drisapersen, GlaxoSmithKline, ended the relationship when the clinical trial data was first revealed, but Glaxo also ended a relationship with another RNAi company – Amicus Therapeutics – which eventually succeeded.
BioMarin is now in a race with Sarepta Therapeutics, which has a competing muscular dystrophy drug that uses a similar mechanism of action. Given these challenges, spending potentially $840MM dollars seems like an expensive call option indeed. So what’s going on here?
Lets take a quick look in Collective IP and try to get a better understanding of what BioMarin may be thinking. Collective IP contextualizes patents with inventors, universities, public filings and clinical trials. For now we won’t dive into the inventors and universities but rather focus simply on patents and public filings.
By comparing the profile and patents we learn that, specifically, skipping dystropin exons 46 and 51 is one of the keys to the patent estate. By searching Collective IP for exon skipping specifically we discover:
Mapping this patent against public filings we discover an important IP piece of the puzzle explaining why BioMarin bought Prosensa. This material event document, surfaced in Collective IP, shows that in 2011, Avi Biopharma (the previous name of Sarepta), challenged Prosensa’s European Patent #1619249 at the Opposition Division of the European Patent Office and lost. Accordingly, Sarepta’s muscular dystrophy drug could potentially be infringing on Prosensa’s drug. And regardless, Sarepta does not, at least based upon this patent, have a claim against Prosensa in this important jurisdiction.
This (and other patent conflicts both resolved and pending) shine a light on Biomarin’s “call option” on Prosensa, suggesting that it makes a lot more sense than might appear at first blush.
This is just one example of the patent office battles, in the US and Europe, happening between Prosensa and its competitors, in particular Sarepta, rushing to get a muscular dystrophy drug to market. Interference proceedings on other patents are occurring at the USPTO and appeals are pending before the EPO.
With Collective IP users can drill deep down into this information, but also contextualize this data via the ability to connect the dots across previously uncorrelated document cohorts.