SpringWorks Targets Rare Tumors, But Myeloma Is The Mother Lode

The company aims to file for desmoid tumors in the second half of 2022, but it has more than half a dozen combination programs with BCMA-targeting therapies in multiple myeloma.

While SpringWorks is initially pursuing desmoid tumors, multiple myeloma represents a much bigger market • Source: Shutterstock

SpringWorks Therapeutics Inc. hopes to become the first company to win US Food and Drug Administration approval for a drug to treat desmoid tumors, a rare and potentially fatal cancer type, after a successful Phase III study of nirogacestat, and the company is already working on commercialization plans. But the drug may have a bigger opportunity as a combination treatment with cell therapy in multiple myeloma.

SpringWorks announced on 24 May that nirogacestat met the primary endpoint and was generally well tolerated in the Phase III DeFi trial in desmoid tumors.

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