ESMO: SpringWorks' Phase III DeFi trial of nirogacestat meets goal

US clinical-stage biotech SpringWorks Therapeutics (Nasdaq: SWTX) yesterday announced positive data from the Phase III DeFi trial of nirogacestat, an investigational oral gamma secretase inhibitor, in adult patients with progressing desmoid tumors, during the 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 of SpringWorks, addng: “DeFi is the largest and most robust Phase III 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 US Food and Drug Administration granted Breakthrough Therapy designation for nirogacestat in 2019. While there is currently no approved therapy for desmoid tumors, Bayer’s (BAYN: DE) Nexavar (sorafenib) has shown some benefit in this indication in a small trial, presenting an off-label option for physicians. It is estimated that 1,000 to 1,500 new desmoid tumor patients are diagnosed each year in the USA.