SpringWorks Therapeutics Announces Full Enrollment of Phase 3 DeFi Trial Evaluating Nirogacestat in Adult Patients with Desmoid Tumors

STAMFORD, Conn., July 22, 2020 (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, today announced that it has achieved full enrollment in its Phase 3 DeFi trial evaluating nirogacestat, an investigational gamma secretase inhibitor, in adult patients with progressing desmoid tumors. As previously disclosed, SpringWorks expects to report topline results from the DeFi trial in the second or third quarter of 2021.

“Desmoid tumors are debilitating and disfiguring soft-tissue tumors and there is a significant need for an FDA-approved therapy for patients living with this devastating disease. We are delighted to have reached another milestone for the nirogacestat program by completing enrollment of the DeFi trial,” said Saqib Islam, Chief Executive Officer of SpringWorks. “This achievement represents an important step toward our goal of bringing this new potential therapy to patients with desmoid tumors. We look forward to completing the trial and reporting data from the study next year.”

The DeFi trial is a global, randomized, double-blind, placebo-controlled Phase 3 trial to evaluate the efficacy, safety and tolerability of nirogacestat in adult patients with progressing desmoid tumors. The study was designed to enroll 118 patients across approximately 50 sites in the United States and Europe. Patients have been randomized 1:1 to receive 150 mg of nirogacestat or placebo twice daily. Key eligibility criteria included tumor progression by >20% as measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1) within 12 months prior to the first dose of study treatment. The primary endpoint is progression-free survival and secondary endpoints include safety and tolerability measures, as well as objective response rate, changes in tumor volume assessed by MRI, and changes in patient-reported outcomes. More information about the DeFi trial is available at www.clinicaltrials.gov under the identifier NCT03785964.

Nirogacestat has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of desmoid tumors (June 2018) and from the European Commission for the treatment of soft tissue sarcoma (September 2019). The FDA also granted Fast Track and Breakthrough Therapy Designations for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis (November 2018 and August 2019).