FDA Approves Incyte and Syndax’s GvHD Drug Niktimvo

The US Food and Drug Administration (FDA) has granted approval to Incyte and Syndax Pharmaceuticals for their antibody therapy, Niktimvo (axatilimab-csfr). This therapy is indicated as a third-line treatment for chronic graft versus host disease (GvHD) in both adults and children who weigh at least 40kg.

Niktimvo marks the second FDA-approved treatment for chronic GvHD within Incyte's portfolio, the other being Jakafi (ruxolitinib), which generated $2.6 billion in sales last year. Market projections by GlobalData suggest that Niktimvo could achieve sales exceeding $305 million by 2030.

Incyte and Syndax plan to seek approval for two smaller vial sizes of Niktimvo before its US launch, anticipated in the first quarter of 2025. Niktimvo is a monoclonal antibody targeting the colony-stimulating factor 1 receptor (CSF1R). The therapy is co-marketed by Incyte and Syndax in the US, with Incyte holding exclusive commercialization rights outside the US. Syndax obtained the worldwide rights for Niktimvo from UCB in 2016.

The FDA's approval of Niktimvo was primarily based on positive data from the Phase II AGAVE-201 trial (NCT04710576). This study achieved its primary endpoint, showing a 75% overall response rate within six months of Niktimvo administration. The median time to response was approximately 1.5 months.

Common side effects observed in the Phase II study included elevated levels of aspartate aminotransferase, blood creatine phosphokinase, lipase, blood lactate dehydrogenase, and alanine aminotransferase, all indicative of tissue injuries. Serious adverse events occurred in 42.3% of the participants, and 37 participants discontinued treatment due to these adverse effects.

Several other therapies are currently in development for treating GvHD. Pfizer's sirolimus, a macrolide lactam derivative, and Eli Lily's Olumiant (baricitinib) are both under investigation in Phase II trials. Equillium's IgG1 monoclonal antibody, itolizumab, is in a Phase III clinical trial (NCT05263999). Mesoblast is also developing a cell therapy for steroid-refractory acute GvHD, although the company has faced two FDA rejections for its product remestemcel-L, with additional data requested by the agency for approval.

Incyte is further evaluating Niktimvo as a first-line combination treatment with Jakafi for chronic GvHD. This Phase II trial (NCT06388564) is anticipated to start recruiting by the end of this year. Additionally, a Phase III trial investigating Niktimvo in combination with steroids for the same indication is expected to commence by the end of 2024.

Syndax is also studying Niktimvo in patients with idiopathic pulmonary fibrosis. This Phase II trial (NCT06132256) aims to enroll about 135 participants, with the study expected to be completed by the second quarter of 2025.

The FDA approval for Incyte and Syndax’s Nikitimvo was based on the positive data from the Phase II AGAVE-201 trial. 

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