Biotech Values

[DewDiligence]

Truncated FVIII from Ipsen and partner, Inspiration, gets EU orphan-drug designation:

http://finance.yahoo.com/news/Ipsen-OBI1-Receives-Orphan-bw-1461578384.html?x=0&.v=1

Approximately one-third of individuals with hemophilia A develop an immune reaction (inhibitors) to hFVIII and can no longer respond to treatment with the coagulation factor. Current therapies, specifically FVIIa [NVO’s NovoSeven] and FEIBA, work by bypassing the natural hemostatic pathway and driving coagulation by raising FVIIa and other activated coagulation factors to higher levels than normal. In contrast, OBI-1, a recombinant B-domain deleted FVIII bioengineered based on the porcine amino acid sequence that typically possesses low cross reactivity to anti-hFVIII antibodies, is a physiologic replacement therapy, activating the natural hemostatic pathway. This would allow clinicians to correlate activity and efficacy with a biomarker and therefore guide dosing and better predict treatment outcomes.

BIIB is a direct competitor in the hemophilia arena (#msg-52146755).