SpringWorks Therapeutics Inc. (NASDAQ: SWTX) stock jumped 20% after the clinical-stage company focusing on life-changing treatment for cancer, and rare diseases announced encouraging results from its DeFi study. The trial was a placebo-controlled, double-blind third phase study assessing the safety, tolerability, and efficacy of the company’s experimental oral gamma-secretase inhibitor, Nirogacestat, in the treatment of progressing desmoid tumors.
Springworks releases data on the DeFi study.
CEO Saqib Islam said:
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Desmoid tumors are aggressive soft-tissue tumors that can lead to severe negative outcomes for patients, including long-lasting pain, disfigurement, and amputation. In rare cases, when vital organs are impacted, desmoid tumors can also be life-threatening.
The primary goal of the DeFi trial was improved progression-free survival (PFS), with nirogacestat showing a statistically meaningful improvement versus control, with a 71% reduction in the probability of disease progression. Furthermore, the trial achieved all critical secondary goals, with nirogacestat showing statistically meaningful improvements in patient-reported outcomes and objective response rate when compared to placebo.
Nirogacestat was better tolerated and had a good side effect profile. However, a more significant percentage of women of reproductive age experienced ovarian dysfunction-related symptoms. Other adverse occurrences were mostly in line with what had been reported initially.
More data from the DeFi study is expected in 2H 2022
More data is planned to be shared at a medical conference, and SpringWorks expects to file a New Drug Application (NDA) to the Food and Drug Administration in 2H 2022. Islam concluded:
Today’s announcement represents a significant milestone towards our goal of bringing the first approved therapy to the desmoid tumor community. We look forward to sharing the DeFi trial data with the FDA and to presenting detailed study results at a medical meeting later this year.
The FDA has designated Nirogacestat as an Orphan Drug for desmoid tumors treatment, while the European Commission has granted the drug Orphan Drug Designation for soft tissue sarcoma treatment. the drug was also issued with Fast Track and Breakthrough Therapy Designations for treatment of persistent, unresectable, refractory, or recurrent deep fibromatosis or desmoid tumors patients
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