Pfizer spinout SpringWorks will ship its first drug to the FDA before year’s end with PhIII win
SpringWorks Therapeutics thinks it has cemented the backbone for its first “pipeline-in-a-product” oncology treatment and will send it to the FDA before the clock strikes 2023 with a Phase III win on Tuesday.
The oral gamma secretase inhibitor, dubbed nirogacestat, beat placebo on the primary goal of progression-free survival in adults with progressing desmoid tumors.
The soft-tissue tumors can lead to long-lasting pain, disfigurement and amputation, and there are currently no approved meds for the rare oncology indication. The tumors typically impact patients aged 20 to 44 years old and disproportionately affect women at rates 2 to 3 times higher, with up to a total of 1,650 new cases diagnosed in the US annually, according to SpringWorks.
The news sent the Stamford, CT biotech’s stock $SWTX soaring 18% at one point before the opening bell Tuesday. In the investor call and presentation, CEO Saqib Islam noted patent protection on the drug lasts until 2039.
In the 142-patient DeFi trial, the treatment showed a 71% reduction in the risk of disease progression, the biotech said. The drug hit secondary goals of statistically significant improvements over placebo in objective response rate and patient-reported outcomes, according to SpringWorks. The randomized, double-blind study took place at sites in North America and Europe.
SpringWorks said the drug, which was given twice daily, was “generally well tolerated with a manageable safety profile.”
But multiple questions surrounding reproductive adverse events went largely unanswered during the company’s investor call.
“The majority of women of childbearing potential had adverse events consistent with ovarian dysfunction,” the company said in its press release.
On the investor call, Islam was asked multiple times about this safety point, and he repeatedly said it was a “class effect as related to ovarian dysfunction” and noted a few times that more specific data will be released at a medical conference later this year. Executives declined to disclose what percentage of the trial enrollees were women.
Asked if it was “fair to assume you haven’t reached a median progression free survival in the treatment arm,” the CEO responded by saying it is “premature to reach that conclusion.” The drug did not meet that measure in either the Phase I or Phase II studies.
The drug is in nearly a dozen clinical trials, flush with multiple combo therapy studies testing nirogacestat alongside BCMA drugs from the who’s who of Big Pharma and biotech alike: GSK, Johnson & Johnson, Pfizer, AbbVie, Regeneron, Seagen, Precision BioSciences and Allogene Therapeutics. All those studies are in relapsed/refractory multiple myeloma. SpringWorks is teamed up with Children’s Oncology Group for a mid-stage pediatric desmoid tumor monotherapy study.
Nirogacestat comes from the coffers of Pfizer. The Big Pharma spun out the drug and other assets into SpringWorks in 2017. The biotech hit the ground running at the time with an initial $103 million from investors like Bain. Islam, who joined the leadership team at the beginning, was previously Moderna’s CBO.
SpringWorks leadership reiterated on the call that it had more than $380 million to spend at the end of last quarter, which gives runway “comfortably” into 2024 for its 18 R&D programs.
