Niktimvo (axatilimab-csfr) - (MAXPIRe)

October 12, 2026: Syndax Pharmaceuticals (NASDAQ: SNDX) said the Phase 2 MAXPIRe trial of axatilimab in idiopathic pulmonary fibrosis (IPF) failed its primary endpoint, the annualized rate of decline in forced vital capacity (FVC). The 26-week, randomized, double-blind, placebo-controlled study enrolled 145 patients across 62 sites in 14 countries and tested axatilimab 0.3 mg/kg versus placebo every two weeks, stratified by background antifibrotic therapy.

Syndax said axatilimab was well tolerated, with a safety profile comparable to placebo, low treatment-emergent adverse events leading to discontinuation in both arms, and no new safety signals or treatment-related fatal adverse events. Based on these results, the company does not plan further development of axatilimab in IPF.

The company said the MAXPIRe outcome does not affect ongoing axatilimab programs in chronic graft-versus-host disease (cGVHD). Axatilimab, marketed as Niktimvo, is already FDA approved in the U.S. for cGVHD after failure of at least two prior systemic therapies in adults and pediatric patients weighing at least 40 kg. Ongoing frontline cGVHD studies include a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774), with readouts expected later in 2026 and in early 2028.