mRNA degrader REM-422

REM-422 is a first-in-class orally available, small molecule mRNA degrader that targets the pre-mRNA of MYB, a previously undruggable transcription factor known to drive a diverse range of diseases.

Active Trials

REM-422 is currently in Phase 1 clinical studies in both ACC and AML or high-risk myelodysplastic syndrome (HR-MDS). REM-422 may also have therapeutic potential beyond ACC and AML. MYB has been implicated in tumorigenesis across a wide variety of hematological malignancies and solid tumors including breast cancers, colon cancers, and lymphomas, among others.

LEARN MORE ABOUT OUR CLINICAL TRIALS:

ADENOID CYSTIC CARCINOMA

(NCT06118086)

ACUTE MYELOID LEUKEMIA

(NCT06297941)

Orchestrating New Possibilities in MYB-driven Cancers

Preclinical data with REM-422 demonstrates the importance of addressing the MYB protein as the driver of diseases like adenoid cystic carcinoma (ACC), a cancer of secretory glands, typically originating in the head and neck region, and acute myeloid leukemia (AML). REM-422 has been shown to exhibit anti-tumor activity across a variety of preclinical models in both ACC and AML. Preliminary positive results from the Phase 1 trial of REM-422 in ACC has demonstrated inhibition of MYB mRNA and anti-tumor activity at well-tolerated doses. REM-422 was granted Orphan Drug Designation by the FDA for ACC and AML.

REM-422 acts by promoting myb poison exon inclusion to induce mRNA degradation.

Normal Splicing

Remix Splicing

Remix™ is dedicated to transforming scientific breakthroughs into impactful medicines through innovative research and development. Building on this foundation, we continue to advance breakthrough science aimed at treating additional malignancies and neurodegenerative diseases. Our work to advance REM-422, our platform and pipeline, has been published in important scientific journals and medical conferences.