Remix
Remix Therapeutics to Present Preclinical Data Demonstrating Anti-Leukemic Activity of REM-422 in AML at the 66th American Society of Hematology Annual Meeting and Exposition (ASH)
Remix Therapeutics (Remix), a clinical-stage biotechnology company developing small molecule therapies to modulate RNA processing and address the underlying drivers of disease, today announced an upcoming oral presentation at the 66th ASH Annual Meeting taking place from December 7-10, 2024, in San Diego, CA.
Results demonstrate oral dosing of REM-422, a selective mRNA degrader of the MYB oncogene, leads to robust anti-leukemic activity observed both as a monotherapy and in combination across a genetically diverse set of preclinical models of acute myeloid leukemia (AML), including eradication of AML blasts in engrafted patient-derived xenograft (PDX) models of AML. The presentation also highlights the differentiated mechanism of action of REM-422 as it can be combined effectively with other agents used in the treatment of AML/MDS and retains activity in cell models engineered with mutations known to confer resistance to other targeted agents.
"The preclinical data for REM-422 provide strong therapeutic rationale for our ongoing Phase I study in AML and High-Risk MDS," said Peter Smith, Ph.D., President and Chief Executive Officer of Remix Therapeutics. "REM-422 is the first compound from our REMaster™ platform to enter clinical development. Its unique mechanism of action, coupled with the robust anti-leukemic activity observed both as a monotherapy and in combination, positions REM-422 as a promising candidate to address the unmet needs in these patient populations."
The MYB oncogenic transcription factor plays a crucial role in hematopoietic cell differentiation and proliferation. Its dysregulation and aberrant activity have been identified in various cancers, including adenoid cystic carcinoma (ACC), AML, acute lymphoblastic leukemia (ALL), and lymphoma. In AML, functional genomics studies have demonstrated a lineage-wide dependency on MYB, consistent with its involvement in disease driven by multiple oncogenic abnormalities (e.g. MLLr, NPM1, FLT3, p53, etc.).
REM-422 is a first-in-class, potent, selective, oral small molecule degrader of MYB mRNA currently in clinical development for AML/HR-MDS (NCT06297941) and ACC (NCT06118086). It functions by inducing the inclusion of a normally unused 'poison exon' (PE) in the MYB pre-mRNA transcript, activating the nonsense-mediated decay pathway and preventing MYB protein expression.
