Key Takeaways
Remix Therapeutics reported positive early-stage data for its experimental drug in a rare cancer that has no approved therapies, showing the potential of a new class of medicines that degrade harmful mRNA. The drug, REM-422, produced a 43 percent overall response rate in a cohort of patients with adenoid cystic carcinoma, or ACC.
The results, presented at the American Society of Clinical Oncology (ASCO) Annual Meeting, showed that all patients in the cohort experienced some level of disease control. "Patients with ACC have long lacked effective therapeutic options, and REM-422 represents a novel first-in-class oral mRNA degrader targeting MYB," said Renata Ferrarotto, a professor at The University of Texas MD Anderson Cancer Center who presented the data. "Notably, tumor responses were durable and deepened over time suggesting potential for long-term benefit.”
In the seven-patient cohort receiving the recommended Phase 2 dose, three patients saw their tumors shrink significantly. The responses have lasted for more than a year and are ongoing. The drug was also well-tolerated, with the most common side effects being mild cases of nosebleeds, fatigue, and anemia.
The positive data offers a glimmer of hope for patients with ACC, a cancer that most commonly arises in the salivary glands and has seen minimal therapeutic progress. The U.S. Food and Drug Administration has granted REM-422 both Orphan Drug and Fast Track designations, potentially speeding its path to market. The results also provide a key proof-of-concept for Remix's broader platform of targeting RNA processing.
REM-422 is a first-in-class oral small molecule designed to degrade the mRNA of the MYB oncogene, which is a key driver of ACC. It works by causing a "poison exon" to be incorporated into the MYB mRNA transcript, leading to its destruction and preventing the production of the cancer-driving MYB protein. Pharmacokinetic and pharmacodynamic analyses from the trial confirmed the drug successfully reduced MYB mRNA and protein levels in patient tumors.
The challenge of treating rare cancers is a recurring theme in oncology. At the same ASCO meeting, researchers from Sylvester Comprehensive Cancer Center presented a different approach for another rare disease, high-grade neuroendocrine tumors. Their Phase 1 trial is testing a combination of immunotherapy with an oncolytic virus, SVV-001, which selectively targets a biomarker on cancer cells to help the immune system recognize and attack the tumor.
While early, both the Remix and Sylvester studies highlight a growing focus on developing highly targeted therapies for cancers that have been left behind by broader treatment advances. For Remix, the company is now focused on completing enrollment in the Phase 2 portion of the ARIA trial to further validate these promising initial findings.
This article is for informational purposes only and does not constitute investment advice.
