Replies to post #90805 on Biotech Values

[DewDiligence]

Biogen Advances FVIII Program to Phase-3

[BIIB now has both its FVIII and FIX programs in phase-3; the latter entered phase-3 in Oct 2009 (#msg-42643715) before the partnership with Biovitrum was restructured to give BIIB control of development (#msg-46820177).

BIIB’s rFVIIIFc is a fusion protein consisting of FVIII fused to the Fc region of IgG. BIIB seeks to unseat BAX’s Advate, the market leader among FVIII recombinants, by showing that rFVIIIFc is just as safe and has a longer half-life.]

http://finance.yahoo.com/news/Biogen-Idec-and-Swedish-bw-659125348.html?x=0&.v=2

›Friday July 9, 2010, 9:39 am

WESTON, Mass. & STOCKHOLM--(BUSINESS WIRE)--Biogen Idec (NASDAQ: BIIB) and Swedish Orphan Biovitrum (STO: SOBI) today announced that they plan to advance the companies' long-lasting, fully-recombinant factor VIII Fc fusion protein (rFVIIIFc) into a registrational clinical trial in people with hemophilia A. The decision to advance the program is based on promising data from a Phase 1/2a open-label, cross-over, multi-center, dose-escalation study that evaluated the safety and pharmacokinetics of an intravenous injection of rFVIIIFc in 16 previously-treated patients with severe hemophilia A. In the study, rFVIIIFc was well tolerated and demonstrated a prolonged half-life compared to Advate (antihemophilic factor recombinant, plasma/albumin-free method, rFVIII), supporting advancement of the program.

The primary objective of the Phase 1/2a study was to assess the safety of rFVIIIFc at different doses; the secondary objective was to estimate the pharmacokinetic parameters of rFVIIIFc at doses ranging from 25 to 65 IU/kg. Preliminary results demonstrated that rFVIIIFc’s prolonged half-life compared to Advate was seen consistently across all patients and dose levels, and other measures of pharmacokinetics including mean residence time and area under the curve were also increased. There were no signs of injection site reactions, inhibitor development or anti-rFVIIIFc drug antibodies in the single-dose study, and there have been no reports of drug-related serious adverse events.

The treatment of severe hemophilia A requires frequent injections, creating a significant burden for individuals with the disorder. The potential of rFVIIIFc, which is based on Biogen Idec's novel and proprietary monomeric Fc-fusion technology, to prolong protection from bleeding and to reduce the frequency of injections needed for treatment will be evaluated in the registrational trial. The global trial, which will commence following communications with regulatory authorities, is being designed to assess the safety, pharmacokinetics and efficacy of rFVIIIFc in both the prevention and treatment of bleeding in hemophilia A patients.

"Biogen Idec is committed to transforming the lives of people with hemophilia by developing a portfolio of long-lasting therapies,” said Glenn Pierce, M.D., Ph.D., Vice President and Chief Medical Officer of Biogen Idec’s hemophilia therapeutic area. “We are excited to announce the advancement of rFVIIIFc into a registrational study. Like our long-lasting hemophilia B program, which entered a registrational trial earlier this year, rFVIIIFc offers the potential to make a significant difference in the lives of people with hemophilia.”

"The Phase 1/2a results are very encouraging, and the decision to initiate our second registrational program in hemophilia represents strong progress in our efforts to improve the lives of individuals with hemophilia,” said Peter Edman, Ph.D., Chief Scientific Officer of Swedish Orphan Biovitrum. “This is also an important milestone in the ongoing development of Swedish Orphan Biovitrum."

In June, the European Medicines Agency’s Committee for Orphan Medicinal Products issued a positive opinion regarding the orphan drug application for rFVIIIFc. A final decision is expected in the coming weeks.‹

[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.