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Pipeline

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We are dedicated to reimagining how cancer is treated and focused on developing first-in-class solutions that support patient journeys.

We have a proven ability to advance innovative and differentiated cancer programs and a strong record of adhering to scientific excellence, following promising clinical data and real-world utilization. 

Highlights of the Company's pipeline include revumenib, a highly selective menin inhibitor, and axatilimab, a monoclonal antibody that blocks the CSF-1 receptor. 

As our leading core value states, we are fueled by our passion for patients—this passion drives us in our pursuit to execute on our mission and continues to drive our innovation to realize the full potential of our pipeline.

Doctor discussing medical information with a patient, holding a tablet and gesturing.

Revumenib for NPM1m Acute Myeloid Leukemia 

In March 2024, the Company announced that sixty-four (64) adult and up to 20 pediatric patients with NPM1m AML have been enrolled into the pivotal portion of AUGMENT-101, a pivotal trial designed to evaluate the safety, tolerability, pharmacokinetics, and efficacy of orally administered revumenib. The primary endpoint for the trial is efficacy as measured by complete remission (CR) or a CR with partial hematological recovery (CRh) rate (CR + CRh) per protocol, with secondary endpoints including duration of response (DOR) and overall survival (OS). Syndax anticipates reporting topline data for this pivotal cohort in the fourth quarter of this year.

Revumenib for KMT2Ar Acute Leukemias

In October 2023, the Company announced positive topline data from the protocol-defined interim analysis of the pivotal portion of the AUGMENT-101 trial in patients with R/R KMT2Ar acute leukemia. AUGMENT-101 is a Phase 1/2 open-label trial designed to evaluate the safety, tolerability, pharmacokinetics and efficacy of orally administered revumenib. The Phase 2 pivotal portion of AUGMENT-101 enrolled R/R patients across the following trial populations: patients with NPM1m AML, patients with KMT2Ar AML, and patients with KMT2Ar ALL. The primary endpoint for each of the cohorts is efficacy as measured by complete remission rate (CR + CRh) per protocol, with secondary endpoints including duration of response and overall survival (OS). Positive data from the pivotal AUGMENT-101 trial of revumenib in adult and pediatric patients with KMT2Ar AML and ALL were published in the Journal of Clinical Oncology in August 2024 and served as the basis for the NDA submission that is currently under review by the FDA.

Menin InhibitorMenin InhibitorRead more

Menin-KMT2A Disruption

Phase 1

Phase 2

Pivotal

Registration

Approved

Indication(s)

  • AUGMENT-101-2A

    monotherapy

    Read more
    R/R KMT2Ar AML and ALL
    registration

  • AUGMENT-101-2C

    monotherapy

    Read more
    R/R NPM1m AML
    pivotal

  • AUGMENT-102

    chemotherapy combination

    Read more
    R/R KMT2Ar and NPM1m acute leukemias
    phase-1

  • BEAT-AML

    ven/aza combination

    Frontline KMT2Ar and NPM1m AML
    phase-1

  • INTERCEPT

    monotherapy

    MRD- progression in KMT2Ar and NPM1m AML
    phase-1

  • Colorectal Cancer

    monotherapy

    Unresectable metastatic microsatellite stable CRC
    phase-1

  • 7+3

    intensive chemotherapy combination

    Frontline NPM1m or KMT2Ar acute leukemias
    phase-1

Anti-CSF-1R Monoclonal AntibodyAnti-CSF-1R Monoclonal AntibodyRead more

Anti-CSF-1R mAb

Phase 1

Phase 2

Pivotal

Registration

Approved

Indication(s)

  • AGAVE-201

    monotherapy

    Read more
    APPROVED
    Refractory Chronic GVHD
    approved

  • Frontline Chronic GVHD

    ruxotilinib combination

    Frontline Chronic GVHD
    phase-2

  • Idiopathic Pulmonary Fibrosis

    monotherapy

    Idiopathic Pulmonary Fibrosis
    phase-2

EntinostatEntinostatRead more

Class 1 HDAC Inhibitor

Received marketing approval in China