Nirogacestat Being Evaluated for Desmoid Tumors in Adults and Children

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07/04/23

James Cassidy, MD, PhD, Chief Medical Officer at SpringWorks Therapeutics, discusses the phase 3 DeFi trial and phase 2 pediatric study of nirogacestat as a treatment for Desmoid tumors.

Desmoid tumors are benign tumors that can grow anywhere on the body into surrounding healthy tissues, including joints, muscle, and viscera. These rare tumors can cause significant pain, internal bleeding, and reduced mobility. These benign tumors are notoriously recurrent, especially with surgical removal, and each recurrence tends to be more aggressive than the previous tumor.

As Dr. Cassidy explains, nirogacestat is an oral, selective, small molecule, gamma-secretase inhibitor. Gamma secretase cleaves multiple transmembrane protein complexes, including Notch, which may contribute to desmoid tumor growth.

There are currently two active clinical trials of nirogacestat as a treatment for Desmoid tumors. One is the phase 3 DeFi trial which is fully recruited; the data from this study will hopefully be presented by the end of 2021. The second trial is a phase 2 pediatric study which is currently still recruiting patients.

For more information about this and other rare tumors, visit checkrare.com/cancers/