Core Insights - Syndax Pharmaceuticals presented new data on Revuforj (revumenib) at the 67th ASH Annual Meeting, highlighting its efficacy and safety in treating acute leukemia, particularly in patients with NPM1m, KMT2Ar, and NUP98r mutations [1][2][3] Efficacy and Safety Data - The overall response rate (ORR) for revumenib was reported at 77% (10/13) in a retrospective review, with a complete remission (CR) rate of 31% (4/13) and 75% (9/12) achieving measurable residual disease (MRD) negativity [1][4][5] - In a Phase 2 SAVE trial, the ORR was 86% (18/21) and the CR rate was 76% (16/21), with 100% MRD negativity among responders [6][11] - Revumenib demonstrated a favorable safety profile, with only 24% (4/17) experiencing Grade 3 or 4 non-hematological adverse events, and low rates of dose reductions and discontinuations at 6% each [5][13] Clinical Trials and Future Directions - Syndax has initiated pivotal frontline trials for revumenib in patients unfit for intensive chemotherapy and in fit patients, aiming to expand clinical data supporting its use [2][23] - Multiple ongoing and planned trials are evaluating revumenib in combination with standard therapies for newly diagnosed patients with specific genetic mutations [22][23] Pediatric Applications - A retrospective review of revumenib as post-HSCT maintenance in children showed that all ten patients were alive at a median follow-up of 19 months, with a 90% relapse-free survival rate [7][8] Company Overview - Syndax Pharmaceuticals is focused on advancing innovative cancer therapies, with Revuforj being a first-in-class menin inhibitor approved for treating relapsed or refractory acute leukemia [22][40]

Financial Data and Key Metrics Changes - In Q2 2025, Syndax Pharmaceuticals reported net product sales of nearly $100 million for Revuforge and Nyktymbo, significantly exceeding expectations [5] - Revuforge net revenue increased by 43% quarter over quarter to $28.6 million, despite one-third of patients pausing treatment for stem cell transplants [5][39] - Nyktymbo generated $36.2 million in net revenue in its first full quarter, up from $13.6 million in the first two months of launch [13][39] Business Line Data and Key Metrics Changes - Revuforge has treated over 500 patients, with approximately 90% of usage in KMT2A patients, and has penetrated 25% of the annual 2,000 patient incidents [8][18] - Nyktymbo is already profitable for Syndax, contributing $9.4 million in collaboration revenue in Q2 2025 [14][40] - The average treatment duration for Revuforge is expected to increase to 6-12 months as treatment patterns mature [21][66] Market Data and Key Metrics Changes - The market opportunity for Revuforge is projected to exceed $5 billion across relapsed refractory and frontline settings [24] - The addressable market for Revuforge in the U.S. is expected to increase to $2 billion with the anticipated approval of the supplemental new drug application (sNDA) for relapsed-refractory mutant NPM1 AML [12] - Nyktymbo targets a $2 billion total addressable market in chronic GVHD, with a current indication for 6,500 patients requiring three or more lines of therapy [26] Company Strategy and Development Direction - Syndax aims to expand Revuforge into earlier lines of therapy and additional patient populations, enhancing its market position [6][12] - The company is focused on maintaining a stable operating expense base while fully funding strategic priorities [39][42] - Syndax is advancing its leadership in menin inhibition and exploring additional indications for Nyktymbo, including idiopathic pulmonary fibrosis [37][48] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in reaching profitability with current resources, citing strong performance from both product launches [43][45] - The company anticipates continued growth driven by strong physician enthusiasm and robust adoption of Revuforge and Nyktymbo [45][48] - Management highlighted the importance of clinical data and physician feedback in driving future growth and market penetration [29][31] Other Important Information - Syndax has a strong financial position with $518 million in cash and equivalents as of June 30, 2025 [43] - The company has strengthened its leadership team with the addition of Dr. Nick Botwood as Head of R&D and Chief Medical Officer [15] Q&A Session Summary Question: Path to profitability and assumptions for REVIVORGE and Nyktymbo - Management indicated that profitability is expected with existing resources, and both product launches are outperforming expectations [51][52] Question: Patients proceeding to transplant and maintenance therapy - Management noted that patients typically proceed to transplant after 2-3 cycles of treatment, and they expect a high percentage of patients to return for maintenance therapy [55][60] Question: Treatment duration observations in real-world practice - Management confirmed that the average treatment duration is expected to be 4-6 months in the first year, potentially increasing to 6-12 months as treatment patterns mature [62][66] Question: KMT-2A patient penetration and revenue growth - Management stated that they expect to reach 50% penetration of the identified KMT-2A population by the end of the year, with significant growth potential remaining [76][80] Question: FDA approval confidence and remaining open items - Management expressed confidence in the FDA approval process, indicating good progress and consistent dialogue with the agency [92][96]

Financial Performance - Revuforj generated $20.0 million in net revenue in 1Q25[3] - Niktimvo generated $13.6 million in net revenue in 1Q25 (reported by Incyte)[3] - The company held $602.1 million in cash and cash equivalents as of March 31, 2025[3] - Total revenue for 1Q25 was $20.0 million[24] - Net loss for 1Q25 was $84.8 million[24] Product Development and Milestones - Syndax initiated a pivotal frontline trial of revumenib with ven/aza in mNPM1 and KMT2Ar patients unfit for intensive chemo in 1Q25[3] - The company completed the submission of Revuforj sNDA for R/R mNPM1 AML in April 2025[3] - Syndax anticipates reporting Phase 1 data from a frontline trial evaluating revumenib with I C (7+3) in 4Q25[27] - Over 1,250 Niktimvo infusions have been ordered YTD[18] Market Access and Adoption - Two-thirds of accounts ordering Revuforj have ordered multiple times[10] - Approximately 95% of all Bone Marrow Transplants (BMTs) ordered are from top accounts[18]