Remix Therapeutics to Present Phase 1 Data of First-in-Class MYB RNA Degrader REM-422 for Adenoid Cystic Carcinoma at AACR Conference
核心洞察
Remix Therapeutics (搜索) will present Phase 1 clinical trial data for REM-422, a first-in-class oral small molecule MYB (搜索) mRNA degrader, in patients with recurrent or metastatic adenoid cystic carcinoma (搜索) at the AACR-NCI-EORTC International Conference.
The presentation will highlight safety, dose-limiting toxicities, recommended Phase 2 dose, and preliminary antitumor activity data from the ongoing Phase 1 trial.
REM-422 functions by facilitating poison exon incorporation in MYB (搜索) mRNA transcripts, leading to nonsense-mediated decay and has received FDA Orphan Drug Designation for both adenoid cystic carcinoma (搜索) and acute myeloid leukemia (搜索).
Remix Therapeutics (搜索) announced that Phase 1 clinical trial data for its first-in-class MYB (搜索) RNA degrader REM-422 in patients with recurrent or metastatic adenoid cystic carcinoma (搜索) has been accepted for a late-breaking oral presentation at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, taking place October 22-26 at the Hynes Convention Center in Boston.
Phase 1 Trial Results to be Presented
The presentation will highlight results from the ongoing Phase 1 trial's primary objectives of defining safety, dose-limiting toxicities, and a recommended phase 2 dose (RP2D). Secondary objectives include evaluating preliminary antitumor activity and pharmacokinetics of REM-422 in patients with recurrent or metastatic adenoid cystic carcinoma (搜索).
Dr. Glenn J. Hanna from Dana-Farber Cancer Institute will present the data during the Clinical Trials Plenary Session on Friday, October 24, from 10:00-11:40 am. The same data will also be presented as a poster during Poster Session B on the same day.
Novel Mechanism of Action
REM-422 represents a first/best-in-class, potent, selective, and oral small molecule mRNA degrader that induces the reduction of MYB (搜索) mRNA and subsequent protein expression. The drug functions by facilitating the incorporation of poison exons in the MYB mRNA transcript, leading to nonsense-mediated decay of the transcript.
This innovative approach targets the MYB (搜索) oncogene, which plays a critical role in adenoid cystic carcinoma (搜索) pathogenesis. The mechanism represents a novel therapeutic strategy in oncology, addressing disease drivers at their RNA processing origin.
Broader Clinical Development Program
REM-422 is currently in Phase 1 clinical studies for multiple indications, including adenoid cystic carcinoma (搜索) and acute myeloid leukemia (搜索) (AML) or high-risk myelodysplastic syndrome (搜索) (HR-MDS). The U.S. Food and Drug Administration has granted Orphan Drug Designation for both ACC and AML indications.
Technology Platform
The drug emerged from Remix Therapeutics (搜索)' REMaster™ technology platform, which leverages cutting-edge data science, biomolecular sciences and chemistry approaches to identify orally administered compounds that modulate gene expression. This platform is designed to reprogram RNA processing and address disease drivers at their origin.
Charles Kung, PhD, Vice President of Biology at Remix Therapeutics (搜索), will also present on REM-422 as a first-in-class mRNA degrader of the MYB (搜索) oncogene during a concurrent session on targeting RNA with small molecules on Saturday, October 25.