SpringWorks Therapeutics Announces Collaboration with Children’s Oncology Group to Conduct a Phase 2 Clinical Trial of Nirogacestat in Pediatric Patients with Desmoid Tumors and Reports Publication of Nirogacestat Case Series in Pediatric/Young Adult Desm

- Expansion of Nirogacestat Clinical Program to Include Pediatric Patients Supported by Recent Publication of Nirogacestat Case Series in Four Children/Young Adults with Desmoid Tumors Treated under the SpringWorks Expanded Access Program -

- Investigators Reported Clinical Responses in All Four Patients Treated with Nirogacestat, including One Complete Response, Two Partial Responses, and One Stable Disease, with No Grade 3 or 4 Adverse Events Reported - 

STAMFORD, Conn., Sept. 16, 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 a collaboration with the Children’s Oncology Group (COG) to evaluate nirogacestat, an investigational gamma secretase inhibitor, in a Phase 2 clinical trial for the treatment of children and adolescents with progressive, surgically unresectable desmoid tumors. The study, which will be sponsored by COG, a National Cancer Institute-supported clinical trials group, and supported by SpringWorks, will aim to enroll approximately 30 patients with a primary endpoint of 2-year progression-free survival. Fariba Navid, M.D., Associate Professor of Clinical Pediatrics, Keck School of Medicine of USC at the Children’s Hospital Los Angeles’ Cancer and Blood Disease Institute, will serve as the Study Chair for the trial, which is expected to begin recruiting patients this month.

“For many children diagnosed with a desmoid tumor, surgery is not an option due to high tumor recurrence rates and the potential for other morbidities and disabilities that can occur following surgery,” said Dr. Navid. “These patients often require medical treatment and we look forward to working with SpringWorks to study nirogacestat in a pediatric population.”

In addition, SpringWorks also reported a recent publication by investigators at the University of Minnesota and the Dana-Farber Cancer Institute in the Pediatric Cancer & Blood journal reporting data from four pediatric and young adult desmoid tumor patients who received nirogacestat under the SpringWorks Expanded Access Program. Following treatment with nirogacestat, the investigators reported a clinical response in all four patients. Specifically, after a median of 13.5 months on treatment, three patients experienced a durable benefit (one complete response, one partial response, and one stable disease) and one patient had an initial partial response followed by disease progression. These four patients had received between one and eight prior lines of therapy, including surgery, before being treated with nirogacestat. Furthermore, the investigators reported that no patient experienced a grade 3 or 4 adverse event while receiving nirogacestat.¹