SpringWorks Therapeutics Announces Presentation of Additional Data from Phase 3 DeFi Trial of Nirogacestat in Adults with Desmoid Tumors at the 2023 ASCO Annual Meeting - Seite 2

During the DeFi study, patients also completed three prespecified assessment tools that included pain measurements to characterize the impact of nirogacestat on this symptom. Changes from baseline in pain severity were compared between treatment arms at Cycle 10 per the prespecified exploratory endpoints. Statistically significant and clinically meaningful reductions in pain were observed with nirogacestat compared with placebo at Cycle 10 across all three assessment tools evaluated in DeFi: the Brief Pain Inventory-Short Form (BPI-SF), the GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom Scale (GODDESS-DTSS), and the European Organisation for Research and Treatment of Cancer Core Quality of Life Questionnaire (EORTC QLQ-C30).

The following results from the assessment tools will be presented at ASCO:

• Nirogacestat significantly reduced pain severity per the BPI-SF “worst pain” score (0–10 range) by 1.55 points, compared with 0.05 points with placebo (P<0.001) at Cycle 10.
• Nirogacestat significantly reduced pain per the GODDESS-DTSS pain score (0–10 range) by 1.78 points, compared with an increase in pain of 0.34 points with placebo (P<0.001) at Cycle 10. The GODDESS-DTSS pain score includes questions about worst pain, dull pain, and shooting pain.
• Nirogacestat significantly reduced pain per the EORTC QLQ-C30 pain subscale (0–100 range) by 22.36 points, compared with an increase in pain of 7.00 points with placebo (P<0.001) at Cycle 10. This pain sub-scale includes questions about pain and its interference with daily activities.
• A statistically significant greater proportion of patients achieved a clinically meaningful pain reduction with nirogacestat treatment compared with placebo at Cycle 10 per the BPI-SF “worst pain” score (P=0.001) and the GODDESS-DTSS pain score (P<0.001).
• Reductions in pain were rapid, becoming evident as early as Cycle 2 (the first post-treatment timepoint evaluated), and these reductions were sustained through to the end of the double-blind phase of the DeFi trial.
  

“Many patients with desmoid tumors live with severe, chronic pain that significantly impacts their quality of life,” said Winette T.A. van der Graaf, M.D., Ph.D., Group Leader and Medical Oncologist, Department of Medical Oncology, Netherlands Cancer Institute, Amsterdam, Netherlands and investigator in the DeFi trial. “It is very encouraging that nirogacestat demonstrated a rapid, sustained and consistent reduction in different aspects of pain during the trial, including worst pain, dull pain, shooting pain, and pain interference with people’s ability to perform daily activities.”