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jq1234

04/27/13 2:05 PM

#160393 RE: genisi #160392

HAE is one orphan disease that has changed from almost no effective treatment option to very crowded in very short period of time. In addition to those already approved, SNTS just resubmitted BLA this month for Ruconest, recombinant C1 esterase, for acute HAE, approved in EU already. VPHM is developing subQ version of Cinryze. As they noted in cc, FDA no longer considers HAE as unmet medical need, thus need large controlled trial from now on.
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mcbio

04/27/13 3:16 PM

#160396 RE: genisi #160392

DYAX

Well, with self-administered subQ, less frequent dosing, better efficacy than C1 inhibitors and without black-box it would be a winner in the prophylaxis indication, but as you've noted - it is not even in the clinic yet.

Thanks. It will be in the clinic shortly, though. Just trying to get a handle on risk-reward of DYAX here at ~$275M market cap. Seems reasonable to me given cash flow from Kalbitor, partnered pipeline potential, and potential for wholly-owned DX-2930, but I need to do more DD on their financials (someone mentioned before their poor financials so need to look closer there).