SpringWorks Therapeutics is bouncing to the FDA. With a phase 3 desmoid tumor trial hitting its primary and secondary endpoints, the Pfizer spinout is planning to seek approval for nirogacestat in the second half of the year.
Investigators enrolled 142 adults with progressing desmoid tumors, soft tissue cancers that affect more than 1,000 new patients a year in the U.S. but lack approved treatments. Participants received 150 mg of the gamma secretase inhibitor nirogacestat or placebo twice daily.
SpringWorks saw a 71% reduction in the risk of disease progression versus placebo, causing the study to hit its primary endpoint with a p-value of less than 0.001. The trial also hit key secondary endpoints that looked at the objective response rate and patient-reported outcomes, although SpringWorks is yet to share the numbers on those measures.
Data on the safety profile seen in the study is similarly scant at this stage. SpringWorks said the drug candidate was generally well tolerated. The majority of women of childbearing potential “had adverse events consistent with ovarian dysfunction.” SpringWorks was aware of the risk. A clinical trial of the drug in children said patients and parents “should be counseled about and consider fertility preservation options before starting treatment.”
In the second half of the year, SpringWorks plans to share more data at a medical conference and file for FDA approval. Saqib Islam, CEO of the biotech, sees the potential for the drug candidate to address an unmet need if it comes to market.
“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,” Islam said in a statement.
Shares in SpringWorks climbed 25% in premarket trading in the wake of the news to above $47. The news sent shares in Ayala Pharmaceuticals, a small biotech running a phase 3 trial of a rival gamma secretase inhibitor in desmoid tumors, up more than 10% to around $2.50.