Sarepta needs to raise money. Who cares? At its current valuation, Sarepta is a great, speculative biotech story. Selling stock to raise much-needed money doesn't make Sarepta any less interesting. In fact, an offering may actually spur more investors to take a look at the stock. Having more money on hand will definitely help Sarepta if the management decides it wants to partner eteplirsen.
Eteplirsen has the potential to treat about 15% of the 12,000 DMD patients in the U.S. Let's do some simple math: 1,800 patients times $300,000 (a guesstimate of eteplirsen's cost but certainly not outrageous for a therapy serving a rare, genetic disease) yields a potential treatment market valued at $540 million. That's just the U.S., I'm not including Europe or the rest of the world.
The 48-week eteplirsen data are expected in October.
Paul A. emails, "I read your tweet where you said the Biocryst Pharmaceuticals (BCRX) results from its gout drug sucked. I don't understand why you feel this way because everything seems very positive to me. Care to explain?"BioCryst's ulodesine (BCX4208) may compare favorably against placebo in its own phase II studies, but the drug's efficacy and safety profile is weaker than Ardea BioSciences' competing gout drug lesinurad, which is already well into phase III studies. AstraZeneca bought Ardea for $1.23 billion last April. BioCryst needs to find a partner for ulodesine but who will want to acquire or license a gout drug that is behind and inferior to a competitor? Lastly, a voice from the choir of stupid. What's funny about the following email is that it was sent Thursday night, after the Amarin approval, which I predicted correctly. In fact, I went a perfect 5-0 in my Summer FDA Drug Approval Contest. Bernard A. writes: "Dear bald head -- I have never seen anybody in my entire life who makes more bad calls on stock approval than you. Aren't you the same moron who know somebody who can guess the outcome of the advisory committee, aren't you the one who guess Arna will not get approved, or maybe you are the one who talked an investor a few weeks ago she told you Amrn will not get approved. You are a moron!!" Thanks for sharing, Bernie. --Written by Adam Feuerstein in Boston.
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