Shots:

• The P-III (DeFi) trial evaluated the efficacy, safety & tolerability of nirogacestat (150mg) vs PBO in a ratio (1:1) in 142 adult patients with progressing desmoid tumors
• The 1EPs of the study are 1EPs as assessed by BICRs while 2EPs & exploratory EPs include safety & tolerability measures, ORR, DoR, changes in tumor volume as assessed by MRI & changes in PROs
• The company plans to file an NDA for nirogacestat to the US FDA before the end of 2022 which will be reviewed under the FDA’s RTOR program. Nirogacestat has received ODD from the US FDA for desmoid tumors and from the EC for soft tissue sarcoma while FTD & BTD for progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis

Ref: GlobeNewswire | Image: SpringWorks Therapeutics