Incyte and partner Syndax Pharmaceuticals on Tuesday secured regulatory clearance to market a third-line chronic graft-versus-host disease treatment, or cGVHD.
The agency greenlit the duo’s axatilimab, a colony stimulating factor-1 receptor-blocking antibody that will be marketed as Niktimvo. The approval came 14 days ahead of the FDA’s decision deadline. The companies plan to launch the antibody during the fourth quarter of this year, Syndax recently said during a second-quarter update.
Incyte already markets a cGVHD treatment known as Jakafi. Novartis sells it in other markets as Jakavi. Before the end of this year, Incyte plans to test Niktimvo in combination with Jakafi for earlier lines of cGVHD treatment in a Phase 2 trial. It also plans to run a Phase 3 of Niktimvo in combination with steroids.
Wilmington, DE-based Incyte will lead the commercialization. It will comprise 70% of the sales effort, and Syndax will contribute the remainder, according to a recent corporate presentation from the biotech. Syndax estimates there are about 17,000 people in the US and more than 35,000 worldwide with cGVHD “at any one time.” It plans to target the approximately 6,500 who have failed two lines of treatment.
Spokespeople for Incyte and Syndax didn’t immediately respond to Endpoints News inquiries about Niktimvo’s wholesale acquisition cost. Earlier this summer, TD Cowen analyst Phil Nadeau estimated that axatilimab would reach peak worldwide sales of about $500 million.
Steve Closter“We estimate that the total addressable market for third line treatment in the US is between $1.5 billion to $2 billion, which assumes that patients will remain on therapy for over 12 months, and assuming axatilimab is priced at a premium to approved agents for chronic GVHD based on its product profile and Part B reimbursement,” Steve Closter, Syndax’s recently appointed commercial chief, said on the company’s second-quarter earnings call on Aug. 1, according to a transcript from AlphaSense.
In an open-label study known as AGAVE-201, researchers tested three doses of axatilimab in adults and pediatric patients. The approval is for people weighing at least 40 kg, and the recommendation is an IV infusion over the course of 30 minutes every two weeks until the disease worsens or toxicity reaches an unacceptable level. The drug had orphan and fast track designation.
The FDA said the median time to first response was 1.5 months. The median duration of response, meaning time before progression, death or use of new systemic therapies, was 1.9 months. In the clinical trial, adverse reactions included aspartate aminotransferase, infection (pathogen unspecified), increased alanine aminotransferase, decreased phosphate, decreased hemoglobin, viral infection, fatigue and diarrhea, among a list of other side effects.
Axatilimab is also being tested as a treatment for idiopathic pulmonary fibrosis. A Phase 2 is underway for the condition, which is currently treated by Roche’s Esbriet (and generic versions) and Boehringer Ingelheim’s Ofev. Multiple drugmakers are also exploring new medicines for the chronic disease, which causes shortness of breath and can lead to death.
Waltham, MA-based Syndax expects another FDA drug approval later this year for its menin inhibitor for certain forms of acute leukemia. The drug, revumenib, has a decision deadline of Dec. 26 after the agency added a three-month delay.
Incyte, meanwhile, recently culled four early-stage programs and bought immune and neuro startup Escient Pharmaceuticals for $750 million.