Dr Bernd Kasper presents his study, at ESMO 2022, of the DeFi trial a phase 3, randomised controlled trial of nirogacestat versus placebo for progressing desmoid tumours (DT).

DT are rare, locally aggressive soft-tissue tumours without approved systemic therapy. Nirogacestat, a novel oral gamma secretase inhibitor has shown antitumour activity in patients with DT.

Nirogacestat 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.