Syndax Highlights FDA-Approved Therapies and Anticipated Milestones at J.P. Morgan Healthcare Conference
核心洞察
Syndax launched Revuforj (revumenib) for relapsed/refractory acute leukemia (搜索) with KMT2A (搜索) translocation, receiving NCCN guideline inclusion for AML (搜索) and ALL (搜索).
Niktimvo (axatilimab-csfr) gained FDA approval for chronic GVHD (搜索) treatment after two prior systemic therapies in patients weighing at least 40 kg.
A supplemental NDA filing for revumenib in R/R mNPM1 AML (搜索) is expected in the first half of 2025, based on positive data from the AUGMENT-101 trial.
Syndax Pharmaceuticals presented at the 43rd Annual J.P. Morgan Healthcare Conference, highlighting its recent achievements and outlining key milestones anticipated for 2025. The company is building on a transformative 2024 marked by FDA approvals of Revuforj and Niktimvo, focusing on the U.S. launches of these medicines.
Revuforj (Revumenib) Launch and Clinical Advancements
Syndax launched Revuforj (revumenib) in late November 2024, the first FDA-approved menin inhibitor for relapsed or refractory acute leukemia (搜索) with a KMT2A (搜索) translocation in adult and pediatric patients one year and older. The FDA granted approval under the Real-Time Oncology Review (RTOR) program. Revumenib has also been included in the NCCN Clinical Practice Guidelines in Oncology for acute myeloid leukemia (AML (搜索)) and acute lymphoblastic leukemia (ALL (搜索)) as a category 2A recommendation for R/R acute leukemia with a KMT2A rearrangement.
Data from the Phase 2 AUGMENT-101 trial demonstrated that the primary endpoint was met in the protocol-defined efficacy population of 64 adults with R/R mNPM1 AML (搜索). A post-hoc efficacy analysis of all (搜索) 77 R/R mNPM1 AML patients meeting efficacy evaluable criteria in the Phase 2 cohort of AUGMENT-101 also yielded positive results. The pivotal Phase 2 data from AUGMENT-101 in R/R KMT2Ar acute leukemia (搜索) were published in the Journal of Clinical Oncology.
Further data presented at the 66th American Society of Hematology (ASH) Annual Meeting included a larger dataset with longer follow-up from the pivotal Phase 2 portion of the AUGMENT-101 trial in R/R KMT2Ar acute leukemia (搜索). Ongoing trials evaluating revumenib in mNPM1 and KMT2Ar acute leukemia across the treatment landscape were also presented:
- BEAT AML: A Phase 1 trial combining revumenib with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar AML patients, conducted as part of the Leukemia & Lymphoma Society's Beat AML Master Clinical Trial, showed an overall response rate (ORR) of 100% (37/37) and a composite complete remission (CRc) rate of 95% (35/37).
- SAVE: A Phase 1/2 trial evaluating an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with R/R AML or mixed-lineage acute leukemia (MPAL) harboring mNPM1, KMT2Ar, or NUP98r alterations, conducted by MD Anderson Cancer Center investigators, showed an ORR of 82% (27/33) and a CR/CRh rate of 48% (16/33).
Niktimvo (Axatilimab-csfr) Approval and Expansion
The FDA approved Niktimvo (axatilimab-csfr) for treating chronic graft-versus-host disease (GVHD (搜索)) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. Axatilimab-csfr was also added to the NCCN Guidelines as a category 2A recommendation for GVHD treatment after the failure of at least two prior lines of systemic therapy in eligible patients.
Results from the pivotal Phase 2 AGAVE-201 trial of axatilimab in recurrent/refractory active chronic GVHD (搜索) were published in the New England Journal of Medicine. A secondary analysis of overall and organ-specific responses from AGAVE-201 in patients who had received at least two prior lines of systemic therapy was presented at the 66th ASH Annual Meeting.
Syndax has initiated enrollment in the MAXPIRe trial, a 26-week randomized, double-blinded, placebo-controlled Phase 2 trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (搜索) (IPF). Incyte, Syndax's partner, has initiated a Phase 2 open-label trial of axatilimab with ruxolitinib in patients ≥12 years with newly diagnosed chronic GVHD (搜索), and a Phase 3 trial investigating axatilimab with corticosteroids as initial treatment for chronic GVHD.
Financial and Corporate Highlights
Syndax announced a $350 million royalty funding agreement with Royalty Pharma based on U.S. net sales of Niktimvo. The company anticipates that its cash, cash equivalents, and marketable securities, along with the royalty funding and anticipated product revenue, will enable it to reach profitability.
Anticipated 2025 Milestones
Key milestones expected in 2025 include:
- Maximizing U.S. adoption of Revuforj as the preferred menin inhibitor.
- Submitting a supplemental NDA (sNDA) filing for revumenib in R/R mNPM1 AML in the first half of 2025, with potential FDA approval by year-end.
- Publishing pivotal data from the AUGMENT-101 trial in R/R mNPM1 AML in the first half of 2025.
- Initiating a pivotal trial of revumenib with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients unfit for intensive chemotherapy in Q1 2025.
- Reporting Phase 1 data from a trial evaluating revumenib with intensive chemotherapy (7+3) followed by revumenib maintenance in newly diagnosed mNPM1 or KMT2Ar acute leukemias in H2 2025.
- Launching Niktimvo in the U.S. in early Q1 2025, co-commercialized with Incyte.
- Completing enrollment in the MAXPIRe Phase 2 IPF trial in 2025, with topline data expected in 2026.
Syndax's CEO, Michael A. Metzger, stated, "Building on a transformative 2024 with the FDA approvals of Revuforj and Niktimvo, we are focused on executing two outstanding U.S. launches for these first-in-class, practice-changing medicines. Syndax is well-positioned for continued success and long-term growth with two approved drugs launching into multi-billion-dollar markets, a clear strategy to expand into additional indications, and a strong cash position expected to fund operations through profitability."