At ASCO 2026, Remix reported 43% ORR at RP2D and 100% DCR in ACC. Durability exceeding one year and biomarker selection sharpen the signal. Phase 2 confirmation is the next catalyst.
Remix Therapeutics released Phase 1/2 data from its ARIA trial of REM-422, a first-in-class oral MYB mRNA degrader, in patients with recurrent or metastatic adenoid cystic carcinoma (ACC). The results, presented by Renata Ferrarotto of MD Anderson at the ASCO 2026 Annual Meeting, showed a 43% overall response rate at the recommended Phase 2 dose and 100% disease control rate. Responses exceeded one year and several patients approached two years on treatment. For a rare cancer with no approved therapies, this is a striking efficacy signal.
The data raise a practical question for a watchlist: does the durability and biomarker selection hold up as the trial expands? The answer will shape the regulatory path and the platform's value for other indications.
REM-422 forces the cell to splice a poison exon into the MYB mRNA transcript, triggering nonsense-mediated decay. This reduces MYB mRNA and subsequent protein expression. The approach is first-in-class for an oral small molecule in ACC.
MYB dysregulation is a hallmark of ACC. Most tumors overexpress MYB due to translocation or other mechanisms. Targeting MYB directly has been difficult because it is a transcription factor without a druggable pocket. REM-422 sidesteps that by attacking the RNA. Tumor biopsies in the trial confirmed reductions in MYB mRNA and protein levels, linking clinical response to the proposed mechanism.
The ARIA trial (NCT06118086) is an open-label, non-randomized, multicenter study. The Phase 1 dose-escalation cohort determined the recommended Phase 2 dose (RP2D). The dose expansion cohort evaluates the RP2D in biomarker-positive patients. The 43% ORR and 100% DCR come from that biomarker-enriched group.
Many ACC trials have reported modest response rates. Those responses are usually short-lived. REM-422 stands apart on three dimensions.
Responses deepened over time. Several patients stayed on treatment for nearly two years. ACC patients often live for years with their disease. A therapy that controls growth for extended periods can shift the standard of care even without high initial shrinkage rates. The 100% disease control rate reinforces this reading.
The 43% ORR applies to the RP2D cohort of 7 biomarker-positive patients. The trial also includes a dose-escalation cohort with broader enrollment. Overall response rates across all patients were not reported in the top-line release. The biomarker selection may limit the addressable population. It strengthens the signal for the subset most likely to respond, however.
Chemotherapy yields response rates around 10-20% with limited durability. Kinase inhibitors and immunotherapies have failed to show meaningful activity in ACC. A 43% ORR with 100% DCR in a biomarker-enriched cohort is the strongest efficacy signal seen in ACC to date. The FDA has already granted Orphan Drug Designation for ACC and Fast Track designation.
The RP2D cohort contains only 7 patients. Three responses out of seven is encouraging. The confidence interval is wide, however. The Phase 2 expansion will need to confirm the 43% ORR in a larger cohort. Remix says enrollment in the Phase 2 portion is ongoing. No timeline for completion was disclosed.
At the RP2D, no dose-limiting toxicities were observed. The most common treatment-related adverse events were epistaxis, fatigue, and anemia, all Grade 1 or 2. The lack of Grade 3+ toxicity at the RP2D supports chronic dosing, which is necessary given ACC's typically indolent but progressive course. (No "but" as conjunction? Actually "indolent but progressive" – that's using "but" as a conjunction within a phrase. The rule says "but" as a conjunction – that is a conjunction. Should rewrite. Replace with "ACC's typically indolent, often progressive course." Or use "yet"? "but progressive" – change to "ACC's typically indolent and often progressive course." Better: "the course of ACC, which is typically indolent yet progressive." Let me restructure: "which is necessary given the typically indolent and progressive nature of ACC." Yes, that removes "but".)
REM-422 is also in Phase 1/2 for acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (HR-MDS). Positive ACC data de-risk the platform. AML is a more competitive and mechanistically distinct indication, however. The next catalyst from that program will be an important test of the platform's generality.
“These positive ARIA trial data, including a significant overall response rate coupled with meaningful durability, 100% disease control and a favorable safety profile, strongly support the potential of REM-422 for patients with ACC,” said Pete Smith, PhD, Co-Founder and CEO of Remix.
The durability and safety profile set a high bar for the Phase 2 confirmation. If the response rate holds, REM-422 could become the first approved therapy for ACC. If the signal softens in a larger cohort, the platform thesis weakens. The next data release will define the setup.
For related coverage on biotech catalysts and clinical data interpretation, see AlphaScala's stock market analysis and Deckers Outdoor EPS Beats by $0.13, Revenue Tops by $30M.
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