Foundation Medicine and Syndax Collaborate on Hematology Diagnostic and Regulatory Approval Efforts

Foundation Medicine, Inc. has announced a collaboration with Syndax Pharmaceuticals to create a companion diagnostic aimed at identifying acute myeloid leukemia (AML) patients with NPM1 mutations. This collaboration will also support Foundation Medicine’s goal to secure regulatory approval for an assay based on their FoundationOne® Heme platform.

NPM1 mutations are found in approximately 30% of newly diagnosed AML cases, making them the most common genetic alteration in these patients. Presently, no targeted treatments are approved for those with NPM1-mutated AML, and the 5-year survival rate for these patients is about 50%. Menin inhibitors, such as Syndax's revumenib, are emerging as potential targeted therapies for NPM1-mutated AML.

Currently, only six biomarkers associated with hematologic malignancies have approved targeted therapies. However, over 40 additional biomarkers are being investigated in clinical trials for hematologic cancers. According to Ashley Yocum, PhD, of The Leukemia & Lymphoma Society, advancements in genomic profiling and scientific research have significantly shifted the diagnosis and treatment landscape for blood cancers, allowing for more personalized treatment options.

If approved, the new assay could be the first next-generation sequencing companion diagnostic for detecting genomic alterations in hematologic cancers, including identifying patients with NPM1 mutations who might benefit from revumenib. Troy Schurr, Chief Biopharma Officer at Foundation Medicine, emphasized the importance of high-quality tests in making informed treatment decisions for patients as the number of biomarkers with targeted therapies grows.

Foundation Medicine offers a range of FDA-approved diagnostic tests, providing both blood and tissue-based testing options to detect genomic alterations. This helps guide personalized treatment decisions. The company is a global leader in companion diagnostic approvals, holding about 60% of all U.S. approvals for next-generation sequencing (NGS) testing.

FoundationOne® Heme is a lab-developed test that uses RNA sequencing along with DNA sequencing to detect various genomic alterations such as base pair substitutions, insertions and deletions, copy number alterations, and gene fusions. Developed by Foundation Medicine, the test's performance characteristics have been determined internally, although it has not yet been cleared or approved by the U.S. Food and Drug Administration.

Revumenib, being developed by Syndax Pharmaceuticals, is a selective small molecule inhibitor targeting the menin-KMT2A interaction. It is intended for the treatment of KMT2A-rearranged (KMT2Ar) and mutant nucleophosmin (mNPM1) AML, as well as acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML). Revumenib has been granted Orphan Drug Designation by the FDA for AML, ALL, and mixed phenotype acute leukemia (MPAL), and Fast Track designation for the treatment of adult and pediatric patients with relapsed or refractory (R/R) acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. The U.S. FDA is currently reviewing a New Drug Application (NDA) for revumenib, with a decision anticipated by December 26, 2024.

This collaboration marks a significant step in advancing diagnostic and treatment options for AML patients, particularly those with NPM1 mutations, by leveraging cutting-edge genomic profiling technologies and emerging targeted therapies.