Deciphera's tumor-tackling oral med achieves 40% response rate, teeing up 2024 submission

More than three years after Deciphera Pharmaceuticals’ first approval, the company appears to be on the precipice of achieving a second commercial product. 

Oral tumor treatment vimseltinib passed the primary endpoint in a phase 3 trial of patients with tenosynovial giant cell tumors (TGCTs), according to an announcement Monday. In patients with TGCTs who were not amenable to surgery and had not received prior anti-CSF1/CSF1R therapy, vimseltinib spurred a 40% objective response rate at the 25-week mark while patients given placebo had a 0% ORR. 

A little more than 80 patients were randomized to receive 30 mg of vimseltinib twice weekly while 39 patients were given placebo. Eight patients experienced a grade 3 or 4 increase in creatine phosphokinase, one measure of potential muscle injury. A grade 3 event is considered severe, while grade 4 is life-threatening. Deciphera said 6% of treated patients discontinued treatment due to treatment-emergent side effects. The most common side effect was swelling around the eye. 

Deciphera also dropped additional phase 1/2 open-label data, with patients in two phase 2 cohorts treated with the 30-mg dose. The median duration of response has yet to be reached in those cohorts, and the company says those data show “clinically meaningful symptomatic benefit at week 25 across multiple secondary efficacy measures.” The discontinuation rate among patients in the phase 1/2 trial was 9%. 

The cumulative data, led by the new phase 3 figures, are set to be packed and sent off to the FDA’s desk in the second quarter of 2024. A marketing application for Europe is tentatively set for the third quarter of next year. 

“We are excited about the potential for vimseltinib to become our next approved medicine, supporting our continued evolution to a company with multiple marketed products,” said CEO Steve Hoerter in a release. The company’s first approval came in May 2020 when Qinlock was given the green light to treat fourth-line patients with advanced gastrointestinal stromal tumors. The tablet brought in $41.8 million in revenue in the third quarter this year, a 29% increase compared to the third quarter of 2022.

Deciphera sits on $376.9 million in cash and equivalents, enough to last into 2026. 

Behind vimseltinib is DCC-3116, a ULK inhibitor being tested in combination with a number of other drugs to treat cancers caused by RAD/RAF mutations. And by early 2024, the company could have a fourth clinical-stage product, with plans to apply for human testing of DCC-3084 by the end of the year.