Nirogacestat success for desmoid tumours

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06/28/23

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.