Syndax Pharmaceuticals, Inc., a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to axatilimab, its anti-CSF-1R monoclonal antibody, for the treatment of patients with chronic graft versus host disease (cGVHD).

"Receipt of Orphan Drug Designation underscores axatilimab's potential to serve as a safe and effective intervention for patients with cGVHD," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "Through its ability to inhibit monocyte derived macrophages, which play a key role in the fibrotic disease process, we believe axatilimab could represent a meaningful therapeutic approach for cGVHD, as well as other fibrotic diseases. As previously announced, our pivotal Phase 2 AGAVE-201 trial is now underway in patients with cGVHD, with topline results expected in 2023."

The FDA's Office of Orphan Drug Products grants Orphan Drug Designation to support drug candidates in development for underserved patient populations or rare disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation qualifies a candidate for various development incentives, including tax credits for eligible clinical trials, waiver of application fees, and market exclusivity for seven years upon FDA approval.

At the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition in December 2020, Syndax reported updated data from its Phase 1 trial of axatilimab in patients with cGVHD which demonstrated deep, durable responses and multiorgan clinical benefit in patients refractory to multiple therapeutic agents. The Company recently announced that the pivotal Phase 2 AGAVE-201 trial, which will evaluate the safety and efficacy of three doses and schedules of axatilimab in patients with cGVHD, is now underway. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for GVHD, with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score. The Company expects to report topline data in 2023.