SpringWorks Therapeutics Announces Data from Phase 3 DeFi Trial Evaluating Nirogacestat in Adult Patients with Progressing Desmoid Tumors at the European Society for Medical Oncology (ESMO) Congress 2022

– Nirogacestat Treatment Resulted in Rapid, Sustained and Statistically Significant Improvements in Primary and All Key Secondary Efficacy Endpoints –

– NDA Submission to the U.S. FDA Planned for Second Half of 2022; Application to be Submitted for Review Under the FDA’s Real-Time Oncology Review Program (RTOR) –

– Company to Host Virtual Investor Event on Sunday, September 11 at 10:00 a.m. ET (4:00 p.m. CEST) –

STAMFORD, Conn., Sept. 10, 2022 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, announced that data from the Phase 3 DeFi trial of nirogacestat, an investigational oral gamma secretase inhibitor, in adult patients with progressing desmoid tumors, are being presented today as a late-breaking oral presentation during a Presidential Symposium at the European Society for Medical Oncology (ESMO) Congress 2022.

“Desmoid tumors can have a debilitating impact on patients’ lives and there is an urgent need for a new standard-of-care treatment,” said Saqib Islam, Chief Executive Officer of SpringWorks. “DeFi is the largest and most robust Phase 3 trial conducted to date in patients with desmoid tumors and we believe these positive data bring us a step closer toward potentially introducing the first approved therapy for this underserved community. We look forward to completing our full NDA filing package by the end of the year, which will be submitted for review under FDA’s RTOR program.”

The DeFi trial met its primary endpoint of improving progression-free survival (PFS), as assessed by blinded independent central review, demonstrating a statistically significant improvement for nirogacestat over placebo, with a 71% reduction in the risk of disease progression (hazard ratio (HR) = 0.29 (95% CI: 0.15, 0.55); p< 0.001). The median Kaplan-Meier estimate of PFS was not reached in the nirogacestat arm and was 15.1 months in the placebo arm. A PFS benefit was observed across all prespecified subgroups, including gender, tumor location, prior treatment or surgery, and mutational status. Confirmed objective response rate (complete response + partial response) based on RECIST v1.1 was 41% with nirogacestat versus 8% with placebo (p<0.001). The complete response rate was 7% in the nirogacestat arm and 0% in the placebo arm. Nirogacestat demonstrated statistically significant and clinically meaningful improvements in patient-reported outcomes (PRO), which were key secondary endpoints of the study. Specifically, nirogacestat significantly reduced pain (p<0.001) and other DT-specific symptoms (p<0.001) and also significantly improved physical/role functioning (p<0.001) and overall health-related quality of life (p=0.007). Most PRO benefits were observed as early as Cycle 2, which was the first timepoint for post-treatment evaluation, and were sustained over the duration of the study.