Pfizer, Sangamo Report Positive Phase I/II Data For Severe Hemophilia A Gene Therapy

The current standard of care for severe hemophilia A requires regular infusions to replace missing Factor VIII.

Genomic medicines companies Pfizer and Sangamo have announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec (SB-525, or PF-07055480), an investigational gene therapy for patients with severe hemophilia A.

The five all received the 3e13 vg/kg dose of giroctocogene fitelparvovec and underwent follow-up assessments over periods that varied per patient, stretching up to 61 weeks for the longest-treated participant.

Their median activity level was 64.2% via chromogenic assay (patient-level geometric means after week 9 post-infusion)

The five were among 11 patients participating in the Alta study, an open-label, dose-ranging, multicenter clinical trial designed to assess the safety and tolerability of giroctocogene fitelparvovec in patients with severe hemophilia A.

The mean age of the 11 patients assessed across the four dose cohorts was 30 years, with their ages ranging from 18–47 years.

“These follow-up data indicate that treatment with giroctocogene fitelparvovec resulted in sustained factor levels up to 14 months following treatment and suggests the potential of this investigational gene therapy to alleviate the treatment burden of current hemophilia disease management,” Sangamo chief medical officer Bettina M. Cockroft, MD, said in a statement.

“We are excited that these data affirm previous findings from this Phase 1/2 study, and that all five patients have sustained levels of factor VIII activity with no bleeding events or use of factor replacement therapy. We are encouraged by the potential of giroctocogene fitelparvovec to demonstrate longer-term durability, an important element for patients living with severe hemophilia A,” said Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Disease Research Unit.

“The Phase 3 lead in study is ongoing, and we look forward to dosing patients with this investigational gene therapy in the pivotal Phase 3 trial later this year.”

“The current standard of care for severe hemophilia A requires regular infusions to replace missing Factor VIII. Gene therapy, on the other hand, offers a new approach with the potential to provide a one-time treatment that would enable patients to produce the missing factor on their own,” said Bettina M. Cockroft, M.D., M.B.A., Chief Medical Officer of Sangamo.

Facebook Comments