Syndax Pharmaceuticals’ Niktimvo failed to significantly preserve lung function in a mid-stage study, prompting the company to discontinue the drug’s development in a chronic lung scarring disease.
In a bid to expand Niktimvo, which was approved in 2024 for chronic graft-versus-host disease (cGVHD), into idiopathic pulmonary fibrosis (IPF), Syndax ran the Phase 2 MAXPIRe study, enrolling 145 patients with the lung condition.
Without providing specific data, the biotech on Monday said that the monoclonal antibody, also called axatilimab, failed to meet the trial’s primary endpoint of slowing the annualized rate of decline in forced vital capacity—a measure of how much air a person can exhale—at 26 weeks.
Given the findings, Syndax is discontinuing development of axatilimab for IPF.
Syndax stocks have dipped from $16.55 at market close yesterday to $14.89 apiece in premarket trading Tuesday—a 10% drop.
MAXPIRe “was always a high risk, high-reward trial,” Mizuho Securities wrote in a Monday note, adding that Wall Street’s “confidence going into this readout was never really that high” since the trial was trying to unlock a novel therapeutic mechanism to potentially access a multi-billion-dollar market.
No new safety signals were observed in the trial, so Mizuho sees no readthrough to the GVHD program.
Treatment-emergent adverse events that led to discontinuation remained low in patients treated with axatilimab. Niktimvo is currently approved for patients who have undergone at least two prior lines of cGVHD treatment.
Syndax will continue two ongoing studies in this indication, testing combinations of Niktimvo with standard-of-care medicines as a potential first-line intervention. Results from one of these studies are expected later this year, while the other is set to read out in early 2028, according to the Monday announcement.
Syndax is developing Niktimvo in GVHD in partnership with Incyte under a September 2021 partnership that involved a $152 million upfront payment, plus up to $450 million in milestones. This alliance doesn’t extend to IPF, Mizuho said on Monday.
IPF is a chronic respiratory condition that scars the lungs, making it difficult for patients to breathe. Syndax proposed Niktimvo as a treatment for this condition based on the asset’s ability to target and block CSF-1R, a protein that regulates the survival and activity of immune cells—underlying players in the formation of scar tissue in the lungs. The FDA in April 2021 granted Niktimvo orphan drug status for IPF.
Elsewhere in the IPF arena, Bristol Myers Squibb reported on Monday that its LPA1 blocker admilparant significantly improved lung function at 52 weeks—an effect that was observed only with the asset’s 60-mg dose and not at the 120-mg dose. The lack of a dose-response effect prompted Leerink analysts to call the readout “mixed,” noting that while admilparant’s data could be enough to support an approval application, the high-dose miss “raises questions about its profile.”
A low-dose win could derisk a regulatory pathway for BMS’ LPA1 antagonist in idiopathic pulmonary fibrosis after a recently reported patient death, but a miss at the higher dose leaves analysts awaiting full data.
October 12, 2026
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4 min read
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