Syndax Pharmaceuticals (SNDX) 44th Annual J.P. Morgan Healthcare Conference summary

Key Achievements and Financial Performance

• Transitioned to a commercial-stage oncology company with three FDA approvals in 14 months and strong launches for Revuforj and Niktimvo.

• Revuforj generated $44M in Q4 net revenue (up 38% QoQ) and $125M for the full year, surpassing benchmarks for AML therapies, with 1,150 prescriptions in Q4.

• Niktimvo achieved $56M in Q4 net revenue (up 22% QoQ) and $152M for the full year, annualizing at over $200M.

• Company maintains a year-end 2025 cash position of $394M, supporting ongoing growth and development plans.

• On track for profitability, driven by strong product launches and expanding market opportunities.

Product Highlights and Market Opportunities

• Revuforj is the first and only menin inhibitor approved for multiple acute leukemia subtypes, unlocking a $5B+ total addressable market.

• Covers all AML subtypes, including adult and pediatric, with unmatched efficacy and strong tolerability, enabling use as monotherapy or in combination.

• Achieved 50% market penetration in KMT2A and expects to reach up to 90% over time.

• Niktimvo is the first CSF1R blocking antibody approved for third-line+ chronic GVHD, with a $2B market and expansion potential to front line and IPF.

• 90% of U.S. bone marrow transplant centers are prescribing Niktimvo, with patients often staying on therapy for years.

Clinical Development and Future Plans

• Advancing broad clinical programs for Revuforj, including pivotal trials (EVOLVE, REVEAL, RAVEN) targeting front-line AML and maintenance settings.

• Ongoing trials for Niktimvo in combination with other agents for GVHD and in phase 2 for IPF, with key data readouts expected in 2026–2028.

• Planning to initiate a proof-of-principle trial for Revuforj in myelofibrosis based on promising early data.

• Focused on generating practice-changing evidence and supporting global registrations for both products.

• 2026 priorities include advancing pivotal trials, publishing new data, and expanding into new indications.