Third Quarter 2026 Earnings Call - SYNDAX PHARMACEUTICALS INC

Financial Performance:

• Total revenue for Q3 2026 reached $83.3 million, representing an 82% year-over-year increase.
• Revuforge (Revumenib) net revenue grew to $61 million (up 90% YoY, 11% QoQ), driven by a 20% increase in new patient starts and growing post-transplant therapy resumption.
• Nictimbo net revenue reached $67 million (up 47% YoY, 12% QoQ); the company holds approximately a 33% share of the third-line-plus chronic GVHD market.
• Collaboration revenue from Nictimbo totaled $22.6 million, contributing a margin rate of approximately 34% (guided to stabilize in the low-to-mid 30% range); this revenue stream is annualizing at over $90 million.
• Ended the quarter with a strong cash position of $551 million in cash, equivalents, and investments.
• R&D and SG&A expenses for 2026 are guided at approximately $400 million, excluding $50 million in non-cash stock compensation.
• The company remains fully funded to execute commercial priorities and the clinical pipeline, including frontline trials.

Guidance and Future Outlook:

• Management expects Revuforge to account for over 80% of total Menvin inhibitor sales in the class this quarter.
• Long-term growth is projected to be driven by post-transplant maintenance therapy resumption, with expectations that 70-80% of KMT2A patients will eventually resume therapy for 1-2 years.
• Revuforge is positioned to potentially exceed $2 billion in peak annual net revenue in the U.S. if approved for frontline AML indications.
• New data supporting Revuforge's use in NUP98 rearranged AML (an area with high unmet need) is expected to be published in Q4 2026.
• Axotilimab Phase 2 trial top-line data with ruxolitinib is expected in Q1 2027; Phase 3 top-line data with corticosteroids is anticipated in early 2028.
• Two new pipeline assets, SNDX-4321 (EGFR inhibitor for NSCLC) and SNDX-62122 (next-gen menin inhibitor for myelofibrosis), are on track to enter clinical trials in 2027.

Business Segments and Product Lines:

• Revuforge (Revumenib): Market leader in relapsed/refractory NPM1 and KMT2A AML. Commercial momentum is driven by expansion into NPM1 (now ~35% of net revenue) and KMT2A indications. Over 620 prescribing accounts active, with formulary coverage for nearly 100% of covered lives.
• Nictimbo: Gaining traction in fourth-line chronic GVHD with strong adoption; tracking toward a launch success profile similar to Resiroc (which reached $500M annual sales in 4 years).
• Pipeline Advancements: Advancing a lifecycle program for Revuforge to include frontline combination therapy; advancing Axotilimab for newly diagnosed chronic GVHD; initiating proof-of-principle trial for menin inhibition in myelofibrosis in late 2027.
• Key Milestone: Revuforge received the "Best Product for Rare Orphan Diseases" award and was named one of Time's best innovations of 2026.

Market and Competitive Landscape:

• Revuforge maintains a dominant market share of all new menin inhibitor starts across all age groups, with Medicare data showing majority share in the >65 demographic.
• Management asserts a "best-in-class" profile, citing a 50% overall response rate in pivotal trials and extensive clinical/commercial experience compared to competitors.
• Anticipated competitor data (from J&J, Sumitomo) at ASH is viewed with confidence; management believes the bar for competitors is high given Revuforge's 2+ year track record and broad evidence base.
• Competitive dynamics are expected to shift as competitors target specific subsets, whereas Revuforge's broad efficacy allows use across NPM1, KMT2A, and NUP98.

Risks and Challenges:

• Transplant Headwinds: The increasing rate of patients proceeding to stem cell transplant creates a short-term revenue headwind (treatment pause) before becoming a long-term tailwind (post-transplant maintenance).
• Competitive Entry: While confident, the company acknowledges potential registration-enabling data from competitors at ASH could impact market dynamics.
• Frontline Trial Execution: Advancing to frontline indications requires successful execution of large pivotal trials (EVOLVE 2, REVEAL) and achieving statistical significance on dual primary endpoints (CR8/OS or MRD-neg CR).

Management Commentary and Tone:

• Tone is highly confident and optimistic, emphasizing "robust demand," "decisive movement towards profitability," and "durable growth."
• CEO Michael Metzger highlighted the company's "first-to-frontline" ambition and the validation of their clinical strategy.
• CFO transition announced: Keith Goldan retired after 4 years; Aaron Andre appointed as new CFO effective November 9, 2026.
• Management expressed strong belief in the "transplant-to-maintenance" paradigm as a key differentiator for Revuforge.

Analyst Questions and Answers:

• Transplant Rates: Management clarified that 40% of NPM1 patients are now proceeding to transplant (up from ~11% in trials), with 50% resuming therapy, driven by earlier treatment and higher response rates; expected to grow to 60-80% resumption over time.
• Frontline Data: Nick Botwood (CMO) noted upcoming Q4 data from BEAT AML and SAVE trials will show longer follow-up, improved survival data, and confirm tolerability, supporting pivotal trial designs.
• Axotilimab Dosing: Agave 201 data supports transitioning patients from bi-weekly to monthly infusions without loss of efficacy, offering a patient-centric benefit with reduced healthcare burden.
• Growth Drivers: Analysts questioned the sustainability of new patient growth; management attributed the 20% QoQ increase to broad-based account growth (Tier 1-2 and community practices) and improved patient identification, dismissing Q2 as an anomaly.
• Maintenance Duration: Physician feedback indicates a trend toward keeping patients on maintenance for 1-2 years, with some discussions about indefinite maintenance for highly responsive patients.

Other Key Points:

• NUP98 Rearrangement: Revuforge is the only menin inhibitor with reported clinical activity in NUP98 rearranged AML, a subgroup with poor prognosis and no targeted treatments.
• Real-World Evidence (RWE): Syndex is the first to publish RWE confirming Revuforge's safety and efficacy in monotherapy and combination settings for NPM1, KMT2A, and NUP98.
• ASH 2026: The company plans a significant data presentation at the American Society of Hematology meeting in December, covering the full acute leukemia treatment continuum.
• Axotilimab Labeling: Reduced frequency dosing data may eventually inform labeling changes to improve patient convenience.