Remix Therapeutics Begins Dosing in Phase 1 Trials for REM-422 in ACC and AML/MDS

WATERTOWN, Mass. — Remix Therapeutics (Remix), a clinical-stage biotech firm focused on developing small molecule treatments to modulate RNA processing and tackle disease drivers, has announced the enrollment and initial dosing of patients in two Phase 1 clinical trials for REM-422. This first-in-class MYB mRNA degrader is being studied as a potential therapy for recurrent or metastatic adenoid cystic carcinoma (ACC) and acute myeloid leukemia/high-risk myelodysplastic syndromes (AML/MDS). Additionally, REM-422 has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of ACC and AML.

Dr. Peter Smith, Co-Founder and CEO of Remix, highlighted the significance of this advancement, stating, "Introducing REM-422 into clinical trials is a major milestone for Remix. We are optimistic about the initial safety and pharmacokinetics data from patients and look forward to further exploring REM-422's potential to target MYB, an oncogenic transcription factor previously deemed undruggable, for ACC and AML/MDS patients."

The two Phase 1 open-label, non-randomized, multicenter studies aim to investigate REM-422 for patients with recurrent or metastatic ACC (NCT06118086) and those with relapsed/refractory AML or high-risk MDS (NCT06297941). Both studies consist of a Dose Escalation Phase and a Dose Expansion Phase, designed to evaluate safety, pharmacokinetics/pharmacodynamics (PK/PD), and anti-tumor activity.

MYB is a critical oncogenic driver in multiple solid tumors and hematological malignancies, such as ACC and AML. REM-422 is an oral small molecule mRNA degrader that effectively reduces MYB mRNA and subsequent protein expression, leading to antitumor activity in MYB-dependent human tumor models.

Dr. Chris Bowden, Chief Medical Officer at Remix, underscored the need for new treatment options for metastatic ACC, noting that REM-422 targets MYB dysregulation, a key driver of the disease. "Our orphan drug designation reflects the urgent need for effective therapies for metastatic ACC. We are eager to see if REM-422 can become a first-line treatment for these patients. Concurrently, we will analyze its safety and efficacy for AML and high-risk MDS, where MYB is crucial in leukemogenesis."

The FDA's Orphan Drug Designation is given to investigational therapies for rare conditions affecting fewer than 200,000 people in the U.S.

About REM-422
REM-422 is an innovative, potent, and selective oral small molecule mRNA degrader. It works by causing the inclusion of poison exons within the mRNA transcript, leading to nonsense-mediated decay (NMD) of the transcript. By targeting MYB dysregulation upstream of protein expression, REM-422 addresses a primary oncogenic driver.

About Adenoid Cystic Carcinoma (ACC)
ACC is a rare cancer primarily developing in glandular tissues of the head and neck. It is often linked to genetic mutations over a patient's lifetime, with most cases showing an overexpression of the MYB protein. Symptoms vary based on tumor location but can include facial numbness, swallowing difficulties, vision changes, and breathing issues. Current treatments include surgery, radiation, and chemotherapy.

About Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS)
AML is a rare but common type of acute leukemia in adults, originating from genetic mutations in bone marrow cells that produce leukemic white blood cells. These cells crowd out healthy blood cells, causing bleeding, infection, and anemia. MDS involves blood-forming unit disorders in the bone marrow, with high-risk cases often progressing to AML. Though several treatments exist for AML, relapse rates remain high, highlighting the need for new therapies.

About Remix Therapeutics
Remix Therapeutics is at the forefront of developing groundbreaking small molecule therapies to reprogram RNA processing and combat diseases. The REMaster™ technology platform enables the identification of RNA processing patterns, modulating gene expression to address disease drivers at their origin. Remix aims to revolutionize how genes are read from the genome, offering potential cures and treatments for various conditions.

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