Incyte and Syndax Announce U.S. Food and Drug Administration (FDA) Approval of Niktimvo™ (axatilimab-csfr) 9 mg and 22 mg Vial Sizes

Niktimvo Approval and Launch - Niktimvo, a first-in-class therapeutic agent, was approved by the FDA on August 14, 2024, for the treatment of chronic GVHD after failure of at least two prior lines of systemic therapy [1][26] - The approval was based on positive data from the global AGAVE-201 trial, which showed 75% of patients receiving 0.3 mg/kg every two weeks achieved a response at six months of treatment (N=79) [1] - Niktimvo is the first and only FDA-approved anti-CSF-1R antibody targeting the drivers of inflammation and fibrosis in chronic GVHD [3][26] - The U.S. launch of Niktimvo is expected in early February 2025, with product available in 9 mg and 22 mg vial sizes [26] Chronic GVHD and Market Need - Chronic GVHD is a serious condition affecting approximately 17,000 patients in the U.S., with nearly 50% of patients requiring at least three lines of treatment [5] - The condition develops in approximately 42% of allogeneic stem cell transplant recipients, highlighting the significant unmet medical need [5] AGAVE-201 Trial Results - The AGAVE-201 trial evaluated axatilimab in 241 patients with recurrent or refractory chronic GVHD, showing durable responses across all organs studied and patient subgroups [6][26] - The trial's primary endpoint was the proportion of patients achieving an objective response by cycle 7 day 1, with secondary endpoints including duration of response and quality-of-life assessments [6] Commercialization and Partnerships - Incyte and Syndax are co-commercializing Niktimvo in the U.S., with Incyte holding exclusive commercialization rights outside the U.S. [29] - Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB in 2016, and entered into a co-development and co-commercialization agreement with Incyte in September 2021 [9] Pipeline and Future Development - Axatilimab is being studied in frontline combination trials for chronic GVHD, including a Phase 2 trial with ruxolitinib and a Phase 3 trial with steroids [10] - Additional studies are underway for idiopathic pulmonary fibrosis, indicating potential expansion of axatilimab's therapeutic applications [10] Patient Support and Access - IncyteCARES provides personalized patient support, including financial assistance and education, to eligible U.S. patients prescribed Niktimvo [4]