- SpringWorks Therapeutics plans to soon seek Food and Drug Administration approval for an experimental tumor drug it's developing after results from a late-stage clinical trial showed treatment reduced the risk of disease progression.
- But the positive results, released by the biotech company via press release Tuesday, weren't enough to lift Springworks shares, which fell by as much as 10% in morning trading on the news.
- Springworks' drug, called nirogacestat, is meant to treat adults with progressive desmoid tumors, which, while benign, can be painful and frequently recur despite surgery. Fewer than 2,000 cases are diagnosed in the U.S. each year, according to the company.
Springworks launched nearly five years ago as a spinoff company from Pfizer, taking with it four clinical-stage drugs, including nirogacestat.
Tuesday's study results give Springworks a path to regulators for what would be its first approved medicine. But the announcement, which typically would be expected to buoy shares, instead led to them tumbling in value.
The biotech's disclosure gave few specifics on the study findings, but did note that nirogacestat met the study's primary goal, showing treatment reduced the risk of disease progression by 71% over placebo. Additionally, Springworks said its drug also achieved all key secondary study goals, although it did not share details.
Full data from the trial will be disclosed at a medical conference in the second half of the year, according to the company, which also plans to file for approval with the FDA in the same timeframe.
Called DeFi, the trial enrolled 142 adults with progressive desmoid tumors and randomized them to receive either nirogacestat or a placebo.
Treatment was "generally well tolerated," according to Springworks, though the majority of women of childbearing potential had adverse events that were "consistent with ovarian dysfunction." The company's statement did not describe the breakdown of those adverse events between drug and placebo groups.
No treatments are currently approved for desmoid tumors, which are sometimes called aggressive fibromatosis or desmoid fibromatosis. They can grow quickly, but also can shrink and go away on their own. Surgery can be used to remove them, though they can recur again in the same spot.
The FDA previously granted an Orphan Drug designation to nirogacestat, which could be useful to Springworks as patents on the drug's chemical structure expire in 2025. Approved orphan drugs are eligible to receive seven years of market exclusivity in the U.S.
In addition to developing nirogacestat for desmoid tumors, Springworks is collaborating with other drugmakers testing cancer treatments aimed at a protein called BCMA. Preclinical testing has suggested Springworks' drug could help make those treatments more effective, and some combination trials are now ongoing.