ESMO is directly attacking the new drug for rare tumor hard fibromas

The 2022 European Society for Internal Oncology (ESMO) Annual Meeting will be held
in Paris from September 9 to 13 local time.

Progressive hard fibroma (DT) is a rare locally invasive soft-tissue tumor that has not been approved as a systemic therapy
.

DeFi is a global, phase III, randomized, double-blind, placebo (pbo)controlled trial (NCT03785964)
in adults with progressive DT.

The results showed that PFS was significantly improved in the niro group compared with the pbo group (risk ratio [HR], 0.

ORR also improved significantly in the niro group (41% vs 8%; P<0.

Statistically and clinically significant and sustained improvements
of statistically and clinically significant significance were observed early in the NIRO group compared to the PBO group.

In terms of safety, 95% of adverse events (AEs) in the niro group were grade 1/2, the most common being diarrhea (84%), nausea (54%), fatigue (51%), hypophosphatemia (42%), and maculopapular rash (32%)
.
75% (27/36) of women of childbearing age developed ovarian dysfunction after receiving niro treatment, and 20 cases (74%) were in remission, of which 11 cases were discontinued for any reason
.

The study showed that Nirogacestat showed statistically and clinically significant improvements in PFS, ORR, disease-specific symptom burden, body/role function, and health-related quality of life, and had a controlled safety profile
in adults with progressive DT.
DeFi is the largest and most rigorous randomized controlled trial conducted in DT to date and the first phase III trial
to achieve a positive result for γ-secretase inhibitors.

References:

B.
Kasper, et al.
DeFi: A phase III, randomized controlled trial of nirogacestat versus placebo for progressing desmoid tumors (DT).
2022 ESMO.
Abstract LBA2.