RARE Daily

Deciphera Reports Positive Top-line Results from Phase 3 Study of Vimseltinib in Rare Cancer

October 30, 2023

Rare Daily Staff

Deciphera Pharmaceuticals reported positive top-line results from the MOTION pivotal phase 3 study of vimseltinib in patients with the rare cancer tenosynovial giant cell tumor, or TGCT, not amenable to surgery.

Vimseltinib is the company’s investigational, orally administered, potent, and highly selective switch-control kinase inhibitor of CSF1R.

The MOTION study met the primary endpoint with objective response rate (ORR) at Week 25 of 40 percent compared to 0 percent for placebo. It also met all key secondary endpoints with statistically significant and clinically meaningful improvements at Week 25 compared to placebo, including ORR by Tumor Volume Score of 67 percent compared to 0 percent for placebo. Treatment with vimseltinib was well-tolerated with no Evidence of cholestatic hepatotoxicity.

“The totality of data shown today demonstrate the potential for vimseltinib to offer a new and differentiated treatment option for patients with TGCT,” said Steve Hoerter, president and CEO of Deciphera Pharmaceuticals.

Deciphera expects to submit a New Drug Application to the U.S. Food and Drug Administration in the second quarter of 2024 and a Marketing Authorisation Application to the European Medicines Agency in third quarter of 2024.

Photo: Steve Hoerter, president and CEO of Deciphera Pharmaceuticals

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