Magrolimab Receives FDA Breakthrough Therapy Designation for Treatment of MDS
Former TAP Partner
September 15, 2020 - FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for magrolimab, a first-in-class, investigational anti-CD47 monoclonal antibody for the treatment of newly diagnosed myelodysplastic syndrome (MDS).
Breakthrough Therapy designation is designed to expedite the development and regulatory review of investigational treatments for serious or life-threatening conditions that, based on preliminary clinical evidence, have the potential to substantially improve clinical outcomes compared with available therapy.
The FDA granted Breakthrough Therapy designation for magrolimab based on positive results of an ongoing Phase 1b study, which evaluated magrolimab in combination with azacitidine in previously untreated intermediate, high and very high-risk MDS. In data presented at the 2020 European Hematology Society Congress, 91 percent of evaluable patients (n=33) treated with magrolimab plus azacitidine achieved an objective response, with 42 percent achieving a complete remission (CR). The combination of magrolimab plus azacitidine was generally well-tolerated. No maximum tolerated dose was reached and no MDS patients discontinued treatment due to a treatment-related adverse event.
Magrolimab is currently being studied in the double-blind, placebo-controlled, randomized Phase 3 ENHANCE trial in previously untreated higher risk MDS. The trial will evaluate the safety and efficacy of magrolimab, in combination with azacitidine, as measured by CR and duration of CR.
Prior to the acquisition of Forty Seven by Gilead Sciences, LLS funded Forty Seven’s magrolimab efforts in MDS and NHL through its Therapy Acceleration Program® (TAP), a strategic initiative to partner directly with innovative biotechnology companies and leading research institutions to accelerate the development of promising new therapies for blood cancers.