Syndax Pharmaceuticals (NASDAQ:SNDX) executives told investors at a Guggenheim biotech conference that the company entered 2026 with what CEO Michael Metzger called a “fantastic start,” pointing to strong commercial performance from its two marketed products and a slate of upcoming clinical and lifecycle milestones.
Metzger said the company recently pre-announced fourth-quarter results and described 2025 as a standout year from a revenue perspective. He cited $125 million in annual net sales for Revuforj and $152 million in net sales for Niktimvo in its first 11 months on the market in chronic graft-versus-host disease (GVHD), with both products showing quarter-over-quarter growth.
Revuforj growth tied to label expansion and transplant-related use
Metzger emphasized Revuforj’s role in helping eligible AML patients reach transplant earlier in treatment, noting physicians are increasingly using it in second- and third-line settings rather than later lines. He also highlighted the ability to restart patients on therapy post-transplant as maintenance, which he said has become an important growth driver.
Asked about early 2026 trends, Metzger said momentum has “continued to go very well,” adding the company planned to provide additional detail on its earnings call.
On market share in NPM1, Metzger said it was too early to quantify but stated the company expects to be “dominant,” drawing a parallel to its position in KMT2A.
Maintenance adoption expected to rise over time
Metzger said that, as of the company’s third-quarter reporting period, about 35% to 40% of transplanted KMT2A patients were returning to Revuforj for maintenance in the commercial setting. He suggested the maintenance rate could eventually reach 70% to 80% at steady state, though he cautioned that the increase would occur over multiple quarters, not immediately.
CMO and Head of R&D Nick Botwood said maintenance initiation typically occurs 60 to 90 days after transplant to allow for engraftment and patient recovery. He cited factors that influence physician decisions, including genetic subtype, relapse risk, measurable residual disease (MRD) status, and patient choice. Botwood also said the company expects to present additional data this year intended to support and educate physicians on post-transplant dosing and management.
Botwood said physicians’ intent is often to keep patients on maintenance for one to two years, depending on tolerability and management of post-transplant fragility such as cytopenias. He referenced data presented at ASH from a pediatric series at MD Anderson in which 90% of children were alive at one year, calling it different from historical expectations.
Metzger added that in Revuforj’s first year on the market, average duration of therapy across the overall KMT2A population has been about four to six months, influenced by transplant and maintenance patterns. Over time, he said the company expects average duration to move toward six to 12 months, depending on how many patients go on maintenance and for how long.
Revumenib expansion: myelofibrosis plans follow “Best of ASH” preclinical work
Botwood described a collaboration with John Crispino’s lab that received “Best of ASH,” focused on myeloproliferative neoplasms (MPNs), including myelofibrosis. He outlined a hypothesis linking menin biology to HOX gene upregulation and downstream disease features such as fibrotic megakaryocytes, abnormal platelets, and splenomegaly.
In mouse models, Botwood said revumenib showed activity both alone and in combination with Jakafi, with effects on underlying pathology. He said Syndax is working with academic centers to move quickly into clinical studies initially in relapsed/refractory myelofibrosis, with an intent to move into frontline combination approaches over time.
Niktimvo: chronic GVHD opportunity and Incyte economics
Turning to Niktimvo (axatilimab) in chronic GVHD, Metzger estimated the U.S. third-line-plus setting at about 6,500 patients, and said the frontline opportunity could expand to about 17,000 patients. He said the company believes the drug could “approach” $1 billion in third-line-plus, with larger potential as earlier-line indications are pursued.
CFO Keith Goldan reviewed the collaboration structure with Incyte in the U.S., stating Syndax and Incyte share a 50/50 commercial profit split and co-promote the product with both companies fielding teams. Goldan said the split is based on net revenue reported by Incyte minus costs including royalties payable to UCB, cost of goods sold, and commercial expenses. He also noted that in the first full quarter of sales, the collaboration already produced net commercial profit.
Goldan said Syndax has guided that it expects about 25% to 30% of the net sales Incyte reports to flow to Syndax’s P&L as collaboration revenue, describing that as a current steady state. He added that over the long term, that percentage could rise as sales grow against a relatively fixed commercial expense base.
Metzger said Incyte is advancing earlier-line development in GVHD with two trials:
- Phase II trial combining Niktimvo with Jakafi, expected to read out in the early part of 2027 (Metzger said it could be guideline-informing and potentially supportive of approval).
- Phase III trial combining Niktimvo with steroids, expected to read out in the early part of 2028.
Metzger also noted Incyte holds rights outside the U.S. and is focused on ex-U.S. approvals.
IPF phase II readout planned for the second half of 2026
Botwood said Syndax is running a phase II trial in idiopathic pulmonary fibrosis (IPF) with a second-half 2026 readout. He described supportive rationale across preclinical models, biomarker and cytokine effects, and clinical observations from AGAVE-201 in bronchiolitis obliterans syndrome, a pulmonary manifestation of GVHD. Botwood said in AGAVE-201, patients receiving the 0.3 mg/kg dose (the approved axatilimab dose) showed nearly a 50% response rate under standard NIH criteria, and about a fifth had a 10% improvement in FEV1.
He said the IPF study is fully enrolled and designed as a double-blind, placebo-controlled trial with 2-to-1 randomization, 135 patients, and a primary endpoint of forced vital capacity (FVC) at 26 weeks. Botwood said the company has no insight into the data yet and will apply rigorous statistics, modeling annualized FVC.
Metzger added that the drug was originally in-licensed from UCB, which had intended it for IPF before shifting focus and licensing it to Syndax. Botwood also said the antibody has molecular configuration features that may contribute to potency, noting efficacy in dose-finding work appeared greatest at the lowest assessed dose.
On funding, management said the current IPF trial burden was taken on by Syndax, and that future development would be cost-shared under the collaboration framework, described as 55% Incyte / 45% Syndax, if the phase II results are positive.
Goldan said Syndax sees a path to profitability, pointing to strong launches for both products and guidance to keep operating expenses flat at $400 million in 2026 (excluding non-cash compensation). He said the company is “fully funded” and does not need additional cash, while declining to specify a precise timing for profitability.
About Syndax Pharmaceuticals (NASDAQ:SNDX)
Syndax Pharmaceuticals is a clinical-stage biopharmaceutical company dedicated to developing novel therapies for the treatment of cancer. Headquartered in Waltham, Massachusetts, the company focuses on small-molecule inhibitors that target key epigenetic and protein interaction pathways. Syndax’s research platform aims to enhance the effectiveness of existing therapies and address high unmet medical needs in oncology.
The company’s lead investigational candidate, entinostat, is a selective class I histone deacetylase (HDAC) inhibitor being evaluated for multiple solid tumor and hematologic indications.
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