Incyte's GvHD treatment approved by FDA

Three years after acquiring the anti-CSF-1R antibody from Syndax Pharmaceuticals, Incyte has achieved FDA approval for Niktimvo (axatilimab-csfr) to treat third-line chronic graft-versus-host disease (GvHD) in adults and children weighing at least 40 kg. In 2021, Incyte paid $117 million upfront for global rights to this antibody and made a $35-million equity investment in Syndax, which retains the option to co-promote the drug in the US. Syndax stands to gain up to $450 million in milestones related to Niktimvo.

The FDA approval for treating GvHD comes shortly after Incyte decided to discontinue five early-stage cancer programs, choosing instead to concentrate on its autoimmune disease portfolio and three selected oncology candidates.

The approval on Wednesday was supported by data from the Phase II AGAVE-201 study. This study revealed a 75% overall response rate among the 79 patients with GvHD who received the recommended intravenous dosage of 0.3 mg/kg of Niktimvo every two weeks. Of those who responded, 60% were still alive after 12 months without the need for new systemic therapy. The treatment proved to be generally well-tolerated, with the most common side effects including elevated levels of AST and ALT, musculoskeletal pain, fatigue, nausea, headache, diarrhea, and cough.

Incyte has ambitious plans for Niktimvo beyond this initial third-line approval. Speaking with FirstWord when the company licensed the antibody, Peter Langmuir, Incyte’s group VP of oncology targeted therapeutics, expressed hope to expand the drug’s use into earlier lines of therapy. The company is also exploring various therapeutic combinations involving Niktimvo for treating GvHD. A Phase II study that combines Niktimvo with another approved GvHD drug, Jakafi (ruxolitinib), is scheduled to start recruiting first-line patients in September, according to ClinicalTrials.org.

In addition, Syndax is conducting an ongoing Phase II trial of the antibody in patients with idiopathic pulmonary fibrosis. Incyte has the option to co-fund late-stage testing for this indication, further expanding the potential applications of Niktimvo.

This recent FDA approval marks a significant milestone for Incyte and opens new avenues for both companies involved. The success of Niktimvo not only highlights its efficacy in managing chronic GvHD but also showcases the strategic importance of the collaboration between Incyte and Syndax. The positive response data from the AGAVE-201 study underscores the potential impact of Niktimvo in providing relief to patients suffering from this challenging condition.

Looking ahead, Incyte’s strategic focus on expanding the therapeutic applications of Niktimvo and exploring its use in combination therapies could set new standards in the treatment of GvHD and other related conditions. The collaboration with Syndax, coupled with ongoing trials and future studies, will be crucial in determining the full therapeutic potential of Niktimvo.