Reduced the Risk of Disease Progression by 71% Innovative Anti-cancer Therapy Reached Phase 3 Clinical Endpoint

Today, SpringWorks Therapeutics announced that its oral specific gamma-secretase small molecule inhibitor nirogacestat met the primary endpoint in a Phase 3 clinical trial in patients with progressive disease (desmoid tumors), reducing the risk of disease progression in patients 71%. Based on this positive result, the company plans to submit a New Drug Application (NDA) to the US FDA in the second half of this year.

Desmoid tumors are rare soft tissue tumors that can be disabling and disfiguring. It can rapidly invade surrounding healthy tissue, causing severe pain, internal bleeding, limited range of motion, and even death. Historically, the treatment for desmoid tumors was surgical resection, however, the recurrence rate of the tumor after surgical resection was very high, resulting in poor efficacy. There are currently no approved treatments for desmoid tumors.

Nirogacestat is an oral specific γ-secretase small molecule inhibitor. Gamma-secretase is capable of cleaving a variety of transmembrane protein complexes, including Notch proteins. The Notch protein is thought to activate the signaling pathway that leads to the growth of desmoid tumors. In addition to treating desmoid tumors, SpringWorks has research and development agreements with companies to test it in combination with B-cell maturation antigen (BCMA)-targeted therapy in patients with multiple myeloma.