Biotech Values

[DewDiligence]

BIIB reports phase-3 data for rFIXFc in hemophilia-B:

http://finance.yahoo.com/news/biogen-idec-sobi-announce-positive-113000726.html

93.5 percent of patients completed the study. Recombinant FIXFc was generally well-tolerated. No inhibitors to rFIXFc were detected and no cases of anaphylaxis were reported in any patients, all of whom switched from commercially-available Factor IX products.

One serious adverse event was assessed to be possibly related to drug by the investigator. The patient experienced obstructive uropathy in the setting of hematuria; he continued rFIXFc treatment and the event resolved with medical management.

…The overall median annualized bleeding rates (including spontaneous and traumatic bleeds) were 2.95 in the weekly prophylaxis arm, 1.38 in the individualized interval prophylaxis arm, and 17.69 in the episodic [i.e. no prophylaxis] treatment arm. In the individualized interval prophylaxis arm, the median dosing interval during the last 6 months on study was 14 days.

Bottom line: This has the potential to be the new standard of care for hemophilia-B. It appears to be as safe and effective as other recombinant Factor IX products such as PFE’s Benefix, and has less frequent prophylactic dosing.