July 8 Clinical Quick Takes: Hemophilia data from Sangamo-Pfizer, Novo; plus GamaMabs, Xarelto and more

Sustained Factor VIII levels for Sangamo, Pfizer hemophilia A gene therapy
Sangamo Therapeutics Inc. (NASDAQ:SGMO) and partner Pfizer Inc. (NYSE:PFE) presented data at the International Society on Thrombosis and Haemostasis (ISTH) meeting in Melbourne showing that the first two hemophilia A patients in the Phase I/II ALTA trial to receive the fourth SB-525 dose level, 3x10¹³ vector genomes per kg (vg/kg), reported no bleeding events and had sustained Factor VIII levels for up to 24 weeks. The next two patients receiving the dose exhibited similar Factor VIII activity kinetics as the first two patients. The partners reported in April that the first two patients receiving the fourth dose level of the gene therapy had achieved normal Factor VIII activity levels at week 6 following infusion (see "Sangamo Gains on Phase I/II Readout for Hemophilia A Gene Therapy").

Novo planning Phase III for mAb treating hemophilia A and B
Novo Nordisk A/S (CSE:NOVO B; NYSE:NVO) presented data at ISTH showing that concizumab (mAb2021, NN7415) was well tolerated and prevented bleeding episodes in hemophilia A patients in the Phase II explorer5 trial and in hemophilia A and B patients in the Phase II explorer4 trial. Novo said it plans to begin Phase III testing of the mAb against TFPI this year...