May | 2 | 2024
Press Release

Remix Therapeutics™ Announces First Patients Dosed in Two Phase 1 Clinical Trials Investigating REM-422 for Treatment of Adenoid Cystic Carcinoma (ACC) and Acute Myeloid Leukemia/Myelodysplastic Syndromes (AML/MDS)

  • REM-422 granted Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of ACC and AML
  • Advances REM-422, an oral small molecule messenger RNA (mRNA) degrader to two clinical studies to evaluate safety and clinical activity in ACC and AML/MDS


Watertown, Mass. (May 2, 2024) – Remix Therapeutics (Remix), a clinical-stage biotechnology company developing small molecule therapies to modulate RNA processing and address underlying drivers of disease, today announced the first patients enrolled and dosed in two Phase 1 clinical trials assessing REM-422, a first-in-class MYB mRNA degrader, as a potential treatment for recurrent or metastatic adenoid cystic carcinoma (ACC) and acute myeloid leukemia/high-risk myelodysplastic syndromes (AML/MDS). Additionally, the company also announced that REM-422 has received Orphan Drug Designation from the FDA for the treatment of ACC and AML.

“Advancing REM-422, the first compound from the REMaster discovery platform, into the clinic marks a significant milestone for Remix,” said Peter Smith, Ph.D., Co-Founder and Chief Executive Officer of Remix. “We are pleased with our first look at safety and pharmacokinetics data in patients and we look forward to further investigating the use of REM-422 to target MYB, a previously undruggable oncogenic transcription factor, for people living with ACC and AML/MDS.”

The two Phase 1, open-label, non-randomized, multicenter studies are investigating REM-422 for patients with recurrent or metastatic ACC (NCT06118086) and for patients with relapsed/refractory AML or high-risk MDS (NCT06297941). Both studies include a Dose Escalation Phase and a Dose Expansion Phase to further evaluate safety, PK/PD and anti-tumor activity.

MYB is an oncogenic driver of multiple solid tumors and hematological malignancies, including ACC and AML. REM-422 is a potent, selective, and oral small molecule mRNA degrader that induces the reduction of MYB mRNA and subsequent protein expression, resulting in antitumor activity in MYB-dependent human tumor models.

“People living with metastatic ACC need effective treatment options, which is reflected in our orphan drug designation, and REM-422 is a targeted therapy that addresses MYB dysregulation, the underlying driver of this disease,” said Chris Bowden, M.D., Chief Medical Officer of Remix. “We are eager to further evaluate the use of REM-422 and assess its potential to be a first-line therapeutic option for patients living with metastatic ACC. Simultaneously, we look forward to analyzing the safety and efficacy of REM-422 for AML and high-risk MDS where MYB is known to play a critical role in leukemogenesis.”

The U.S. FDA Orphan Drug Designation is granted to investigational therapies for rare medical conditions that impact fewer than 200,000 people in the United States.

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 poison exons within the mRNA transcript, leading to nonsense-mediated decay (NMD) of the transcript. REM-422 addresses MYB dysregulation, a driver of oncogenesis, upstream of protein expression.

About ACC

Adenoid cystic carcinoma (ACC) is a rare cancer that commonly develops in glandular tissues in the head and neck. It is caused by genetic mutations, likely developed over a patient’s lifetime, with the majority of ACC cases linked to an overexpression of the MYB protein. Depending on the location of the tumor, symptoms may include numbness of the face, difficulties swallowing, changes in vision, or difficulty breathing, among others. Current treatment solutions include surgery, radiation therapy, and chemotherapy.


Acute myeloid leukemia (AML), a rare cancer of the blood and bone marrow, is the most common type of acute leukemia in adults. AML is caused by genetic mutations within bone marrow cells, which in turn causes the production of leukemic white blood cells that crowd out healthy blood cells. This may cause problems with bleeding, infection, and anemia. Myelodysplastic Syndromes (MDS) are disorders of blood-forming units in the bone marrow.  High-risk MDS patients have a higher percentage of blast cells in the bone marrow and that, in many cases, progresses to AML. There are several approved agents to treat AML, however, many patients relapse after achieving a response, underscoring the need for new therapies.

About Remix Therapeutics

Remix Therapeutics is a clinical-stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and treat disease. The REMasterTM technology platform facilitates RNA processing pattern identification, leveraging these learnings to modulate gene expression. Remix’s innovative therapeutic approach has the potential to alter the way genes are read from the genome, to correct, enhance, or eliminate the gene message, thereby addressing disease drivers at their origin. For more information visit


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