THIRD QUARTER 2026 EARNINGS CALL - SYNDAX PHARMACEUTICALS INC

Financial Performance:

• Total revenue reached $83.3 million in Q3 2026, representing an 82% year-over-year increase.
• Revuforge net revenue grew to $61 million (90% YoY, 11% QoQ), accounting for the vast majority of the Menin inhibitor market share.
• Nictinvo net revenue totaled $67 million (47% YoY, 12% QoQ), annualizing at over $260 million.
• Collaboration revenue attributed to Nictinvo reached $22.6 million, with a margin contribution of approximately 34% (expected to stabilize in the low-to-mid 30% range).
• The company ended Q3 with $551 million in cash, equivalents, and investments, maintaining a fully funded position to execute commercial and R&D priorities.
• R&D plus SG&A expenses for 2026 are guided at approximately $400 million, excluding $50 million in non-cash stock compensation.

Guidance and Future Outlook:

• Management targets profitability, noting that current revenue growth from two medicines is driving the company toward this goal.
• Peak annual net revenue for Revuforge is projected to exceed $2 billion in the U.S. alone, driven by potential frontline AML indications.
• Expectation to publish Revuforge data for relapsed refractory NUP98 rearranged acute leukemia in Q4 2026, potentially expanding the treated population.
• Nictinvo is expected to see continued growth through further penetration in third and fourth-line chronic GVHD and potential paradigm shifts with upcoming combination data.
• New clinical assets (SNDX-4321 and SNDX-62122) are on track to enter the clinic in 2027, with IND submissions expected by end of 2026 for 4321 and early 2027 for 62122.
• Top-line results for Axotilimab combination trials in newly diagnosed chronic GVHD are anticipated in Q1 2027 (Phase 2) and early 2028 (Phase 3).

Business Segments and Product Lines:

• Revuforge (Menin Inhibitor): Dominant market leader in relapsed/refractory NPM1 and KMT2A acute leukemia. Prescription volume doubled YoY, with 1,700 total prescriptions (13% QoQ growth).
• Transplant Integration: 50% of KMT2A and 40% of NPM1 patients are proceeding to stem cell transplant (up from 11% in pivotal trials), with 50-60% resuming therapy post-transplant, creating a long-term revenue tailwind.
• Nictinvo (Chronic GVHD): Captured ~33% of the 3rd-line-plus market with 300+ new patient starts and 6,350 infusions in Q3.
• Pipeline Updates: Axotilimab reduced frequency dosing data (Agave 201) published, showing feasibility of transitioning from Q2 to Q4 weekly infusions with sustained response.
• New Assets: SNDX-4321 (EGFR-mutated NSCLC) and SNDX-62122 (next-gen Menin inhibitor for myelofibrosis) are advancing toward clinical initiation.

Market and Competitive Landscape:

• Revuforge holds a dominant share of new Menin inhibitor starts across all age groups, with formulary coverage for nearly 100% of covered lives.
• Management anticipates future competitors (e.g., J&J, Sumitomo) but views Revuforge as best-in-class due to two years of commercial experience, deep clinician trust, and a robust real-world evidence base.
• Revuforge is positioned to be the first Menin inhibitor in the frontline AML setting, aiming to capture the "first-to-market" advantage with upcoming ASH data.
• Nictinvo faces a benchmark of ~40% response rate at six months with steroids alone; the company targets a 60% response rate and improved event-free survival.

Risks and Challenges:

• Transplant Headwind: The short-term pause in treatment for stem cell transplants creates a temporary revenue headwind, though expected to become a durable long-term tailwind as patients resume therapy.
• Competitive Entry: Potential registration-enabling data from competitors at ASH could challenge market dynamics, though management remains confident in their differentiation.
• Clinical Trial Execution: Success depends on the execution of pivotal frontline trials (EVOLVE 2 and REVEAL) and achieving required endpoints for FDA approval.

Management Commentary and Tone:

• Tone is highly confident and optimistic, emphasizing strong commercial momentum, robust clinical data, and a clear path to profitability.
• CEO Michael Metzger highlighted the company's "bold R&D vision" and "world-class capabilities" as key differentiators.
• CFO transition noted: Keith Goldan retired after 4 years, replaced by Aaron Andre effective November 9, 2026, bringing extensive biopharma financial experience.
• Management expressed confidence in the "stacking" effect of post-transplant patients driving significant revenue growth in 2027.

Analyst Questions and Answers:

• Transplant Rates: Management confirmed the 40% (NPM1) and 50% (KMT2A) transplant rates are durable trends driven by treating patients earlier and in combination, not just cohort maturity.
• Frontline Data: Updates on BEAT AML and SAVE trials will provide longer follow-up, focusing on tolerability, CR rates, MRD eradication, and survival data to support pivotal trials.
• Duration of Therapy: Average treatment duration for NPM1 patients not transplanting is >7 months; post-transplant maintenance is expected to reach 1-2 years, significantly extending the franchise life.
• Growth Drivers: New patient growth was broad-based across Tier 1, Tier 2, and community practices, with high repeat prescribing rates.
• Axotilimab Trials: Phase 2 top-line data moved to Q1 2027 to ensure maturity, including event-free survival; Phase 3 data expected early 2028.
• Q4 Outlook: No specific Q4 revenue breakdown provided, but management expressed hope for continued momentum based on Q3 performance.

Other Key Points:

• Revuforge received the "Best Product for Rare Orphan Diseases" award from the Galen Foundation and was named a top innovation by Time in 2026.
• Real-world evidence indicates Revuforge's efficacy in the commercial setting exceeds pivotal trial results, particularly regarding transplant eligibility.
• The company is actively pursuing a guideline listing for Revuforge in the NUP98 rearranged AML population (2-5% of AML cases).
• Management confirmed no formal crossover is allowed in frontline maintenance studies (REVEAL) as Event-Free Survival is the primary endpoint.