Preliminary Results Yield 43% Overall Response Rates (via RECIST) in the Efficacy Population
Data Demonstrate REM-422, the First-in-Class Small Molecule MYB mRNA Degrader, Reduces Expression of the MYB Oncogene
WATERTOWN, Mass., Oct. 24, 2025 (GLOBE NEWSWIRE) -- 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 positive preliminary results from its ongoing clinical trial evaluating REM-422, a first-in-class mRNA degrader, in patients with recurrent or metastatic (R/M) adenoid cystic carcinoma (ACC). The data will be presented in late-breaking oral and poster presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics on Friday, October 24.
REM-422 is the first oral mRNA degrader of MYB demonstrating proof-of-mechanism and proof-of-concept in ACC along with a favorable safety profile. Anti-tumor activity was observed in patients with R/M ACC, particularly among biomarker positive patients. As of October 1, 2025, the overall response rate (via RECIST) was 43% in the efficacy population.
“The favorable safety and tolerability of REM-422 along with encouraging anti-tumor activity, particularly among biomarker selected patients with ACC, are important findings and I look forward to the final results from this ongoing trial,” said Glenn J. Hanna, MD, Director, Center for Cancer Therapeutic Innovation (Early Drug Development Program), Medical Oncologist, Center for Head & Neck Oncology, Dana-Farber Cancer Institute and Associate Professor of Medicine, Harvard Medical School. “These data represent the first clinical results successfully targeting MYB, a previously intractable cancer driver. The results support REM-422’s potential to address a critical unmet need for patients.”
REM-422 demonstrated linear pharmacokinetics in blood across all dose levels accompanied by a reduction of MYB mRNA and protein levels in ACC tumors. In the subset of biomarker positive patients that were on study for more than 6 months, tumor shrinkage of more than 20% was observed in 71% (10/14) with 6 partial responses (4 confirmed) ongoing at 4-6 months. REM-422 was generally well tolerated. The most common treatment-related adverse events included anemia, fatigue and epistaxis at the recommended Phase 2 dose.
“These positive preliminary REM-422 data are the first clinical demonstration of MYB inhibition in patients with cancer and the first human data for a molecule generated using our proprietary REMasterTM technology platform,” said Pete Smith, PhD, Co-Founder and CEO of Remix Therapeutics. “Our company was created to bring new medicines for patients who have not seen significant advances in treatment options and we are proud of the progress that these data represent for patients with ACC. We look forward to expanding our work with REM-422 in other MYB-driven leukemias and solid tumors.”
ACC is a cancer of salivary glands, typically originating in the head and neck region. Many different approaches (e.g. chemotherapy, kinase inhibitors, immunotherapy) have been studied in ACC, which is often driven by MYB, with modest or disappointing results, and there remain no approved treatment options.
About REM-422
REM-422 is a first-in-class, potent, selective, and oral small molecule mRNA degrader that induces the reduction of MYB mRNA and subsequent protein expression. REM-422 functions by facilitating the incorporation of a poison exon in the MYB mRNA transcript, leading to nonsense-mediated decay of the transcript. REM-422 is currently in Phase 1 clinical studies in both Adenoid Cystic Carcinoma (ACC) and Acute Myeloid Leukemia (AML) or high-risk myelodysplastic syndrome (HR-MDS). REM-422 was granted Orphan Drug Designation by the U.S Food and Drug Administration for ACC and AML.
About the Phase 1 Trial of REM-422 in Adenoid Cystic Carcinoma
This Phase 1, open-label, non-randomized, multicenter study (NCT06118086) is investigating REM-422 in patients with recurrent or metastatic Adenoid Cystic Carcinoma (ACC). The study includes a Dose Escalation Phase and a Dose Expansion Phase. The purpose of the Dose Escalation Phase is to determine the maximum tolerated dose and/or recommended Phase 2 dose (RP2D) of REM-422 in patients with recurrent or metastatic ACC. The purpose of Dose Expansion is to further evaluate the safety and anti-tumor activity of the REM-422 RP2D in biomarker positive patients.
About Remix Therapeutics
Remix Therapeutics is a clinical-stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and address disease drivers at their origin. Remix's REMaster™ technology platform leverages cutting-edge data science, biomolecular sciences and chemistry approaches to identify orally administered compounds that modulate gene expression. Remix's innovative therapeutic approach led to the discovery of REM-422, a first-in-class RNA processing modulator in oncology, now being evaluated in Phase 1 clinical studies to treat acute myeloid leukemia (AML), high-risk myelodysplastic syndrome (HR-MDS) and adenoid cystic carcinoma (ACC). For more information visit www.remixtx.com.
Disclosure: Dr. Hanna receives institutional research support from and has served in a consulting/advisory role for Remix.
Contacts:
Media Contact:
Lisa Buffington Buffington Comms
lbuffington@remixtx.com
Investor Contact:
Will O'Connor Precision AQ
Will.OConnor@precisionaq.com
