Syndax Pharmaceuticals (SNDX) BofA Securities 2025 Healthcare Conference summary

Commercial launches and product differentiation

• Two new agents, Revuforj and Niktimvo, have launched, targeting rare leukemia and chronic GVHD, respectively, with first-in-class mechanisms and strong early uptake in both efficacy and safety profiles.

• Revuforj is the first approved menin inhibitor for KMT2A acute leukemia, with expansion into NPM1 pending regulatory review.

• Niktimvo, a CSF-1R antibody, is partnered with Incyte and is showing promising results in third-line chronic GVHD.

• Early launch data for both products indicate outperformance versus internal expectations, with rapid adoption and positive patient outcomes.

• Broad and deep prescriber base and strong payer coverage are driving rapid market penetration for Revuforj.

Clinical data and upcoming milestones

• Revuforj demonstrated a 67% complete response rate and 81% composite CR rate in the BEAT AML study, significantly outperforming historical standards.

• All evaluable patients in the BEAT AML study achieved MRD negativity, suggesting deep and durable responses.

• An update to the AUGMENT-101 study showed a 26% CR/CRh rate in NPM1-mutant patients, with regulatory submission under RTOR and potential approval expected soon.

• Ten abstracts, including pivotal studies, will be presented at EHA, highlighting both monotherapy and combination data.

• Two randomized phase III studies in frontline AML are planned, focusing on KMT2A and NPM1 populations, with accelerated approval endpoints.

Market opportunity and growth strategy

• Revuforj is expected to penetrate up to 50% of the KMT2A population in its first year, with NPM1 as a significant upcoming driver.

• Rapid payer adoption and nearly complete formulary coverage have been achieved, with 80% of patients starting therapy within a week.

• Niktimvo is seeing strong uptake in chronic GVHD, with potential to expand into earlier lines and other indications such as IPF.

• Combination strategies with standard of care agents are prioritized, with key partnerships (e.g., HOVON) supporting pivotal trials.

• Regulatory support for accelerated approval pathways is influencing trial design and market entry strategies.