Gilead's Anti-CD47 Monoclonal Antibody Magrolimab Receives FDA Breakthrough Therapy Designation for Myelodysplastic Syndromes

Gilead Sciences, Inc. today announced that the U.S. FDA has granted Breakthrough Therapy Designation to magrolimab, a first-in-class anti-CD47 monoclonal antibody, for the treatment of newly diagnosed myelodysplastic syndromes (MDS).

Myelodysplastic Syndromes is a cancer caused by poorly formed or dysfunctional blood cells in the bone marrow. No new treatments have been approved by the FDA in 14 years. The median survival for patients with low-risk MDS is 6 years, while that for patients with high-risk MDS is approximately 18 months.

CD47 is a "don't eat me" signal expressed on the cell surface, which prevents normal cells from being phagocytosed by macrophages through binding to the SIRPα receptor on the macrophage surface. However, in various cancer types, tumor cells also express CD47 to prevent their phagocytosis by macrophages.

Magrolimab is an anti-CD47 monoclonal antibody acquired by Gilead Sciences, Inc. through its acquisition of Forty Seven, Inc. It is designed to disrupt the “don’t eat me” signal by binding to CD47 and interfering with the recognition of CD47 by the SIRPα receptor on macrophages. The U.S. FDA has granted magrolimab Fast Track designation for the treatment of myelodysplastic syndromes (MDS), acute myeloid leukemia, diffuse large B-cell lymphoma, and follicular lymphoma.

▲ Mechanism of Action of Anti-CD47 Antibodies (Image source: Forty Seven Inc. official website)

The granting of this Breakthrough Therapy Designation is based on positive results from an ongoing Phase 1b study. Data presented at the 2020 European Hematology Association Congress showed that among 33 patients evaluable for efficacy, the combination therapy of magrolimab and azacitidine achieved an objective response rate of 91%, with 42% of patients achieving complete remission (CR).

Magrolimab is currently being evaluated in the randomized, double-blind, placebo-controlled Phase 3 ENHANCE trial, in combination with azacitidine, for the treatment of previously untreated high-risk MDS patients.

References:

[1] Gilead’s Magrolimab, an Investigational Anti-CD47 Monoclonal Antibody, Receives FDA Breakthrough Therapy Designation for Treatment of Myelodysplastic Syndrome. Retrieved September 15, 2020, from https://www.businesswire.com/news/home/20200915005434/en

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