Dr Bernd Kasper speaks to ecancer on his study of the DeFi trial’ a phase 3, randomised controlled trial of nirogacestat versus placebo for progressing desmoid tumours (DT).

DeFi is a global, Phase III, randomised, double-blind, placebo (pbo)-controlled trial in adults with progressing DT per RECIST v1.1

The results demonstrated significant PFS improvement of Niro compared with pbo. ORR was significantly improved with niro versus pbo, with a median time to response of 5.6 versus 11.1 months.

Niro demonstrated statistically and clinically significant improvements in PFS, ORR, symptom burden, physical/role functioning, and health-related quality of life and had a manageable safety profile in adults with progressing DT.

September is Desmoid Tumor Awareness Month, and given that nearly 30% to 40% of desmoid tumors are initially misdiagnosed, partly due to a lack of awareness, it is important to raise awareness among both patient and doctor communities to better support those who may be living with these devastating tumors. Dr. James (Jim) Cassidy, MD, PhD, Chief Medical Officer at SpringWorks Therapeutics discusses desmoid tumors, also called aggressive fibromatosis or desmoid-type fibromatosis, which are rare and often debilitating and disfiguring soft-tissue tumors with no FDA-approved treatment. He also talks about how SpringWorks is working to advance a potential new treatment option for people with desmoid tumors.

#SpringWorksTherapeutics #DesmoidTumorAwareness

Dr. James (Jim) Cassidy is Chief Medical Officer of SpringWorks. He has over 30 years of experience in oncology as an academic physician-scientist and a drug development leader in both biotechnology and pharmaceutical companies, with experience spanning from early-stage research to translational and clinical development to post-marketing medical affairs strategy and lifecycle management. Prior to joining SpringWorks, he was Vice President of Oncology Strategic Program Direction at Regeneron Pharmaceuticals. Prior to this role, Jim was Corporate Vice President of Translational Development at Celgene, where he oversaw translational science efforts for the company’s entire portfolio of programs addressing both hematological malignancies and solid tumors. Before that, he was Vice President of Oncology at Bristol-Myers Squibb, where he was responsible for all oncology assets from development candidate nomination through clinical proof-of-concept studies, including biomarkers and translational research, and was closely involved with late-stage development, commercial, and business development efforts as well. Prior to Bristol-Myers Squibb, Jim held several roles of increasing responsibility at Hoffmann La-Roche, including Global Head of Translational Research for Oncology and Acting Head of the Oncology Therapy Area. Before joining Roche, Jim had been a leading academic physician-scientist, most recently having served as Professor of Oncology, Head of the Department of Cancer Research and Head of the Division of Cancer Sciences and Molecular Pathology at the University of Glasgow in Scotland. Jim received his medical degree and doctorate from the University of Glasgow.

Dr Jonathan Trent comments on the use of nirogacestat alone or in combination in desmoid tumor at the 2022 ESMO Florida conference

Fariba Navid, MD, Associate Professor of Clinical Pediatrics, Keck School of Medicine of USC, announces her involvement in a trial for nirogacestat as a treatment for Desmoid tumors in pediatric patients.

Desmoid tumors are benign tumors that can grow anywhere on the body into surrounding healthy tissues, including joints, muscle, and viscera. These rare tumors can cause significant pain, internal bleeding, and reduced mobility.

The most common course of action for Desmoid tumors - particularly those in non-lethal areas - is to “wait and watch” as these tumors can shrink on their own. Surgery to remove the tumors may also be used. However, as Dr. Navid says, surgery is not always ideal as it has a high recurrence rate. As Dr. Navid explains, the recommended treatment for patients with Desmoid tumors who cannot undergo surgery is the use of systemic therapy.

Dr. Navid explains in this video that a phase 3 clinical trial of nirogacestat for the treatment of adult patients with Desmoid tumors was recently completed. Official results of that trial are to come. Given the success of nirogacestat in adults, a separate trial of the drug for the treatment of children and adolescents was started. Dr. Navid serves as the Study Chair for this pediatric study, which is currently recruiting patients.

For more information about Desmoid tumors and other rare tumors, visit checkrare.com/cancers/