An antibody therapeutic, magrolimab, being tested for myelodysplastic syndrome (MDS), a group of cancers in which the bone marrow does not produce enough healthy blood cells, was granted breakthrough therapy designation by the Food and Drug Administration (FDA). 

Breakthrough Therapy

FDA breakthrough therapy designation speeds development of new treatments. It requires early evidence that a therapy may outperform current options. CIRM funded a Phase 1b trial in MDS and AML that supplied the key data for this designation.

Cancer cells display CD47, which sends a “don’t eat me” signal to macrophages. Magrolimab blocks CD47, allowing immune cells to detect and destroy cancer cells.

Irv Weissman’s Stanford team developed magrolimab with CIRM support. Their work led to the creation of Forty Seven, Inc., which Gilead acquired in 2020 for $4.9 billion.

In CIRM’s 2019-2020 18-Month Report, Mark Chao credited CIRM for helping advance the treatment.

“CIRM’s support has been instrumental to our ability to rapidly progress Forty Seven’s CD47 antibody targeting approach.”

New approach for higher-risk MDS

Magrolimab is currently being studied as a combination therapy with azacitidine, a chemotherapy drug, in a Phase 3 clinical trial in previously untreated higher-risk MDS. This is one of the last steps before seeking FDA approval for widespread commercial use.

In a press release, Merdad Parsey, M.D., Ph.D., Chief Medical Officer at Gilead Sciences, discusses the significance of the FDA designation and the importance of the treatment.

“The Breakthrough Therapy designation recognizes the potential for magrolimab to help address a significant unmet medical need for people with MDS and underscores the transformative potential of Gilead’s immuno-oncology therapies in development.”