FDA Approves Incyte/Syndax Partnered Drug For Chronic Graft-Versus-Host Disease, A Post-Transplant Complication

On Wednesday, the FDA approved Incyte Corporation INCY and Syndax Pharmaceuticals Inc’s SNDX Niktimvo (axatilimab-csfr), an anti-CSF-1R antibody for chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients.

“Niktimvo is Incyte’s second approved treatment for chronic GVHD,” said Hervé Hoppenot, CEO, Incyte.

Chronic GVHD is a serious condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor). The donated cells initiate an immune response and attack the transplant recipient’s organs.

Also Read: Incyte’s Q2 Earnings: Wider Than Expected Loss, Pipeline Review, Lifts Jakafi Sales Guidance.

Chronic GVHD is estimated to develop in approximately 42% of transplant recipients, affecting approximately 17,000 patients in the U.S.

The FDA approval was based on data from the global AGAVE-201 study of Niktimvo in 241 adult and pediatric patients with refractory chronic GVHD who received at least two prior lines of systemic therapy.

The trial met the primary endpoint across all cohorts receiving Niktimvo. The study showed durable responses across all organs studied and patient subgroups.

Among patients who received Niktimvo at the approved dose of 0.3 mg/kg every two weeks (N=79), 75% achieved an overall response rate (ORR) within the first six months of treatment, with a median time to response of 1.5 months.

In the U.S., Niktimvo will be co-commercialized by Incyte and Syndax Pharmaceuticals.

Incyte has exclusive commercialization rights for Niktimvo outside of the U.S.

To facilitate patient dosing and limit product waste, Incyte and Syndax will seek approval to launch two smaller vial sizes following the FDA’s approval of Niktimvo (as a 50mg vial).

Following FDA approval of the new vial sizes, the companies anticipate launching Niktimvo in the U.S. no later than the early first quarter of 2025.

Price Action: INCY stock is trading higher by 1.26% to $61.95, and SNDX stock is up 1.84% at $19.94 at the last check on Thursday.