SWTX - SpringWorks Therapeutics Announces Presentation of Additional Data from Phase 3 DeFi Trial of Nirogacestat in Adults with Desmoid Tumors at the 2023 CTOS Annual Meeting | Benzinga
- Nirogacestat Treatment Demonstrated Rapid and Sustained Improvements in Functional Status Compared to Placebo Across Multiple Assessment Tools -
STAMFORD, Conn., Nov. 01, 2023 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (NASDAQ:SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today announced the presentation of additional data from the Phase 3 DeFi trial at the 2023 Connective Tissue Oncology Society (CTOS) Annual Meeting, being held November 1-4, 2023. These data demonstrate the impact of nirogacestat, an investigational gamma secretase inhibitor, on physical and role functioning in adults with desmoid tumors. Data from the DeFi trial demonstrated statistically significant and clinically meaningful improvements across all primary and key secondary endpoints and were previously presented at leading medical congresses and published in the March 9, 2023 edition of the New England Journal of Medicine.1-3
"Desmoid tumors are locally aggressive and invasive soft tissue tumors that can cause severe pain and functional impairment. These impairments can be physical, such as difficulty walking or carrying out daily tasks, or role-related, such as difficulty caring for children or working, and they severely impact the day-to-day lives of patients," said Jim Cassidy, M.D., Ph.D., Chief Medical Officer of SpringWorks. "We are pleased that the robust data from our Phase 3 DeFi trial demonstrated that nirogacestat provided clinically meaningful improvements in key patient-reported outcomes, including pain as well as both physical and role functioning. We look forward to the opportunity to bring this important new medicine to the desmoid tumor community following anticipated U.S. regulatory approval."
Poster Presentation at the 2023 CTOS Meeting
Impact of Nirogacestat on Functional Status in Patients with Desmoid Tumors: Results from the Phase 3 DeFi Study
Abstract #: 1571083
Poster #: P 188
Poster Session Date and Time: Thursday, November 2, 5:30-6:30 p.m. CEST
As previously reported in the DeFi trial (NCT03785964), nirogacestat met its primary endpoint of significantly improving progression-free survival compared to placebo in adult patients with progressing desmoid tumors (hazard ratio: 0.29 [95% CI, 0.15–0.55]; P<0.001). Nirogacestat also achieved a significant and clinically meaningful improvement in physical and role functioning status, a key secondary endpoint, compared with placebo at Cycle 10 (p<0.001). The most frequently reported treatment-emergent adverse events that occurred in participants receiving nirogacestat were diarrhea (84%), ovarian dysfunction (75% of women of childbearing potential), nausea (54%), fatigue (51%), hypophosphatemia (42%), and maculopapular rash (32%).
During the DeFi study, the impact of nirogacestat on functional status was evaluated. Changes from baseline in physical and role functioning were compared between nirogacestat and placebo at Cycle 10, the prespecified time point for key secondary endpoints. Statistically significant and clinically meaningful improvements in physical and role functioning were observed with nirogacestat compared with placebo at Cycle 10 across all three assessment tools used: the GOunder/Desmoid Tumor Research Foundation DEsmoid Impact Scale (GODDESS DTIS) Physical Functioning (PF) subscale, the European Organisation for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30) PF and Role Functioning (RF) subscales, and the Patient-Reported Outcomes Measurement Information System Physical Function Short Form 10a (PROMIS PF10a) tool. Improvements in functional status were rapid, becoming evident as early as Cycle 2 (the first post-treatment time point evaluated) and these improvements were maintained through Cycle 24 across multiple tools measuring physical and role functioning.
The following results are being presented at CTOS:
- Nirogacestat significantly improved mean physical functioning score from baseline per the GODDESS DTIS PF domain compared with placebo at the pre-specified time point. The GODDESS DTIS PF domain captures varying degrees of vigorous and moderate daily activity, including moving and reaching.
- Nirogacestat significantly improved mean physical functioning score from baseline per the EORTC QLQ-C30 PF subscale compared with placebo at the pre-specified time point. The EORTC QLQ-C30 PF subscale captures the concepts of strenuous activities, taking a long walk, taking a short walk, need to stay in a bed or chair, and help with eating, dressing, washing, and using the toilet.
- Nirogacestat significantly improved mean role functioning score from baseline per the EORTC QLQ-C30 RF subscale compared with placebo at the pre-specified time point. The EORTC QLQ-C30 RF subscale captures the concepts of work or other daily activities and hobbies or leisure activities.
- At Cycle 10, patients receiving nirogacestat were five times more likely to have a clinically meaningful improvement in physical functioning (GODDESS DTIS Physical Functioning and EORTC QLQ-C30 Physical Functioning), and twice as likely to have a clinically meaningful improvement in role functioning ...