Syndax Pharmaceuticals is at the forefront of developing innovative cancer therapies, focusing on targeted treatments that address significant unmet medical needs. One of their key advancements is Revuforj® (revumenib), the first and only menin inhibitor approved by the FDA for treating adult and pediatric patients with relapsed or refractory acute leukemia featuring a KMT2A translocation. This therapy is part of a broader pipeline that includes ongoing investigations into revumenib for other genetically defined acute leukemias, such as those with an NPM1 mutation. The company emphasizes scientific excellence and patient-centric approaches, aiming to enhance the quality of life and longevity for cancer patients.

In addition to revumenib, Syndax is developing axatilimab, a monoclonal antibody that targets the CSF-1 receptor, with applications in chronic graft-versus-host disease and idiopathic pulmonary fibrosis. The company is committed to advancing its pipeline through rigorous clinical trials, as evidenced by the positive topline results from the AUGMENT-101 trial for revumenib in NPM1m acute myeloid leukemia. Syndax's dedication to innovation and patient care drives its mission to deliver first-in-class therapies that provide meaningful clinical outcomes and address urgent healthcare needs in oncology.

United States

Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Highlights of the Company's pipeline include Revuforj® (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. Fueled by our commitment to reimagining cancer care, Syndax is working to unlock the full potential of its pipeline and is conducting several clinical trials across the continuum of treatment. Syndax corporate headquarters are in Waltham, MA.

Cancer

Hematologic Diseases

Lung Diseases

Syndax Pharmaceuticals

Graft-versus-host disease

Axatilimab (SNDX-6352) is a high affinity antibody targeting the colony stimulating factor 1 receptor (CSF-1R). CSF-1R signaling has been demonstrated in nonclinical studies to be a key regulatory pathway involved in the expansion and infiltration of donor-derived macrophages that mediate the disease processes involved in cGVHD (<a href="http://www.syndax.com/pipeline/sndx-6352-cgvhd/?gclid=CjwKCAjw8-78BRA0EiwAFUw8LD1dAhzOUT_5YIOr5ycDXqL_SO2p9EJP07QMljRRxBjyxpgr-QDJdBoC2PIQAvD_BwE">Learn more HERE</a>).&nbsp;

Niktimvo (axatilimab-csfr) (SNDX-6352) 

Blood cancer

Leukemia

Mixed lineage leukemia-rearranged (MLLr) is an &nbsp;acute leukemia with poor prognosis that is caused by spontaneous &nbsp;translocations at the MLL1 gene locus. MLLr fusion proteins &nbsp;bind with high affinity to the nuclear protein menin and the menin-MLL &nbsp;interaction enables leukemic transformation by driving a specific &nbsp;transcription program.1
In preclinical studies, inhibition of the Menin-MLL1 interaction led to tumor cell differentiation and death.1
<a href="https://documents.cap.org/documents/acute-leukemia-summary-of-recommendations.pdf" rel="noopener noreferrer" target="_blank">CAP/ASH Testing Guidelines</a> recommend the testing of MLL1 (KMT2A) and NPM1 mutations as part of the diagnostic workup of acute leukemia.1,2
<a href="https://syndax.com/pipeline/sndx-5613/">Source</a>

Syndax Pharmaceuticals

Company Details