On March 19, 2026 Remix Therapeutics (Remix), a clinical-stage biotechnology company developing small molecule therapies to modulate RNA processing and address the underlying drivers of disease, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to first-in-class small molecule MYB mRNA degrader, REM-422, for the treatment of patients with recurrent, metastatic or unresectable adenoid cystic carcinoma (ACC) whose tumors express MYB transcripts containing a poison exon.
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"Receiving Fast Track designation for REM-422 underscores the urgent need for new treatment options for patients with ACC," said Peter Smith, PhD, Co-Founder and Chief Executive Officer of Remix Therapeutics. "REM-422 represents a novel approach designed to target MYB, a key oncogenic driver in ACC that has historically been difficult to drug. This designation supports our ongoing efforts to expeditiously advance REM-422 through clinical development to bring this potential therapy to patients as quickly as possible."
REM-422 is a first-in-class, oral small molecule MYB mRNA degrader designed to reduce MYB expression by inducing incorporation of a poison exon in the MYB transcript, leading to degradation of the mRNA and suppression of MYB protein expression. MYB dysregulation is a hallmark driver of ACC and several hematologic malignancies. REM-422 is currently being investigated in the ongoing Phase 1/2 ARIA (A study of REM-422 In Adenoid cystic carcinoma) study, evaluating safety, pharmacokinetics and preliminary anti-tumor activity in patients with recurrent or metastatic, and unresectable ACC.
The FDA’s Fast Track program is designed to accelerate the development and review of drugs that treat serious conditions and address unmet medical needs. The designation for REM-422 was based on positive preliminary results from a Phase 1 clinical trial evaluating REM-422 in patients with recurrent or metastatic ACC. 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 recurrent, metastatic or unresectable ACC whose tumors express MYB transcripts containing a poison exon.
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 1/2 clinical studies in both Adenoid Cystic Carcinoma (ACC) and Acute Myeloid Leukemia (AML) or high-risk myelodysplastic syndrome (HR-MDS). The U.S. Food and Drug Administration granted REM-422 Orphan Drug Designation for ACC and AML and Fast Track designation for ACC.
About the ARIA (A study of REM-422 In Adenoid cystic carcinoma) Clinical Trial
This Phase 1/2, open-label, non-randomized, multicenter study (NCT06118086) is investigating REM-422 in patients with recurrent, metastatic or unresectable 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, metastatic, or unresectable 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 Adenoid Cystic Carcinoma
Adenoid cystic carcinoma (ACC) is a solid tumor that most commonly arises in the salivary glands characterized by frequent recurrent, perineural invasion and dysregulation of the MYB oncogene. Depending on the location of the tumor, symptoms may include numbness of the face, difficulties swallowing, changes in vision, or difficulty breathing, among others. Many therapeutic approaches, such as chemotherapy, kinase inhibitors, and immunotherapy have been studied in ACC with modest or disappointing results, and there remain no approved treatment options.
(Press release, Remix Therapeutics, MAR 19, 2026, View Source [SID1234663775])