Syndax Pharmaceuticals (SNDX) investor relations material

Business and Product Performance

• Revuforj and Niktimvo, both first- and best-in-class oncology drugs, have launched successfully and target multi-billion-dollar markets, with Niktimvo reaching $96 million and Revuforj $88 million in net revenue as of Q3.

• Both products are marketed synergistically to the same physician audience, enhancing commercial efficiency and supporting rapid market adoption.

• Niktimvo achieved annualized sales of $200 million and profitability in its first full quarter, with high patient persistency.

• Revuforj saw 25% prescription growth in Q3 and about 750 new patient starts, contributing to a sustainable growth engine.

• The company is on track for profitability in the next few years, supported by stable expenses and expanding product margins.

Clinical and Scientific Updates

• Revuforj is the first and only FDA-approved menin inhibitor for multiple acute leukemia subtypes, now indicated for both KMT2A and NPM1 mutations in adults and children ≥1 year.

• Niktimvo is the first and only CSF-1R blocking antibody FDA-approved for third-line chronic GVHD, showing rapid, durable responses and inclusion in NCCN Guidelines.

• Both drugs are supported by strong real-world and clinical trial data, showing high response rates, durable remissions, and favorable safety profiles.

• Extensive clinical development includes 23 presentations at ASH, with multiple oral and poster sessions for both products.

• Ongoing trials aim to expand indications for both drugs, including frontline settings and new disease areas such as idiopathic pulmonary fibrosis.

Clinical Efficacy and Safety Data

• Real-world evidence for revumenib shows 77% ORR, 31% CR/CRh, and 75% MRD negativity in acute leukemia, with favorable tolerability.

• Pediatric post-HSCT maintenance with revumenib resulted in 90% relapse-free survival at 19 months, with all patients alive and therapy well tolerated.

• Phase 2 SAVE trial in newly diagnosed AML showed 86% ORR, 76% CR, and 100% MRD negativity among responders.

• Phase 1 trial with intensive chemotherapy in newly diagnosed AML showed 96% ORR, 92% CRC, and 86% MRD negative CR, with rapid count recovery and manageable safety.

• Differentiation syndrome occurred in 25% of patients, with 12% Grade 3/4 and 2 fatal cases; QTc prolongation in 36% of patients, with 17% Grade 3/4.