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Syndax Highlights Recent Accomplishments and Anticipated 2025 Milestones at the 43rd Annual J.P. Morgan Healthcare Conference
SNDXSyndax(SNDX) Prnewswire·2025-01-13 12:00

Company Achievements - Launched Revuforj® (revumenib), the first and only FDA-approved menin inhibitor, for the treatment of relapsed or refractory (R/R) acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older [2][6] - Revumenib was added to the NCCN Clinical Practice Guidelines for AML and ALL as a category 2A recommendation for R/R acute leukemia with a KMT2A rearrangement [2][6] - Received FDA approval for Niktimvo™ (axatilimab-csfr) for the treatment of chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg [2][10] - Announced a 350millionroyaltyfundingagreementwithRoyaltyPharmabasedonU.S.netsalesofNiktimvo,whichisexpectedtohelpthecompanyreachprofitability[7]ClinicalTrialResultsMettheprimaryendpointinthePhase2cohortoftheAUGMENT101trialofrevumenibin64adultswithR/RmNPM1AML[6]Reportedanoverallresponserate(ORR)of100350 million royalty funding agreement with Royalty Pharma based on U.S. net sales of Niktimvo, which is expected to help the company reach profitability [7] Clinical Trial Results - Met the primary endpoint in the Phase 2 cohort of the AUGMENT-101 trial of revumenib in 64 adults with R/R mNPM1 AML [6] - Reported an overall response rate (ORR) of 100% and a composite complete remission (CRc) rate of 95% in the BEAT AML trial evaluating revumenib with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar AML patients [6] - Presented an ORR of 82% and a CR/CRh rate of 48% in the SAVE trial evaluating revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with R/R AML or MPAL [6] - Published results from the pivotal Phase 2 AGAVE-201 trial of axatilimab in adult and pediatric patients with recurrent/refractory active chronic GVHD in the New England Journal of Medicine [10] 2025 Milestones - Submit a supplemental NDA (sNDA) filing for revumenib in R/R mNPM1 AML in the first half of 2025, with potential FDA approval around year-end 2025 [11] - Initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients unfit to receive intensive chemotherapy in Q1 2025 [11] - Launch Niktimvo in the U.S. in early Q1 2025, co-commercialized by Syndax and Incyte [11] - Complete enrollment in the MAXPIRe Phase 2 IPF trial in 2025, with topline data expected in 2026 [11] Industry Impact - Revumenib and axatilimab are positioned to address multi-billion-dollar markets, with potential for expansion into additional indications [3] - The company's robust clinical data and first-mover advantage with Revuforj are expected to drive U.S. adoption as the preferred menin inhibitor [11] - Syndax's strong cash position, including the 350 million royalty funding agreement, is expected to fund operations through profitability [7]