REM-422 / Remix Therap - LARVOL DELTA

A phase I trial of the MYB RNA degrader REM-422 in patients with recurrent or metastatic (R/M) adenoid cystic carcinoma (ACC) (AACR-NCI-EORTC 2025) - P1 | "REM-422 is the first oral RNA degrader of MYB demonstrating proof-of-mechanism in both blood and tumor tissue along with a favorable safety profile. Anti-tumor activity was observed in patients with R/M ACC, particularly among biomarker (+) patients, where novel therapies are urgently needed."

Clinical • Late-breaking abstract • Metastases • P1 data • Adenoid Cystic Carcinoma • Oncology

Clinical activity of REM-422, a MYB mRNA degrader, in recurrent/metastatic adenoid cystic carcinoma: Final results from the phase 1/2 dose-escalation cohort. (ASCO 2026) - P1/2 | "REM-422 is the first small molecule MYB mRNA degrader to demonstrate clinical activity in R/M ACC and is generally well-tolerated. These findings support further evaluation of REM-422 in a biomarker-selected population. Accrual to the Phase 2 Confirmatory Cohort is ongoing."

Clinical • Metastases • P1/2 data • Adenoid Cystic Carcinoma • Oncology • NFIB

Remix Therapeutics to Present at AACR Drug Discovery and Development Conference (GlobeNewswire) - "Presentation to Highlight Phase 1 REM-422 ACC Data Demonstrating 43% Overall Response Rate and Durable Responses Exceeding One Year with Favorable Safety Results."

P1 data • Adenoid Cystic Carcinoma

REM-422-101: Study of REM-422 in Patients With Recurrent, Metastatic, or Unresectable Adenoid Cystic Carcinoma (clinicaltrials.gov) - P1/2 | N=125 | Recruiting | Sponsor: Remix Therapeutics | Trial completion date: Jun 2027 ➔ Sep 2027 | Trial primary completion date: Jun 2026 ➔ Sep 2027

Trial completion date • Trial primary completion date • Adenoid Cystic Carcinoma • Oncology

Remix Therapeutics Announces Positive Data from Phase 1/2 ARIA Study of First-in-Class MYB mRNA Degrader, REM-422, in Patients with Adenoid Cystic Carcinoma (ACC) at the American Society of Clinical Oncology Annual Meeting 2026 (GlobeNewswire) - "Results from the Phase 1 dose-escalation cohort found anti-tumor activity in patients with R/M ACC across molecular subtypes and previous lines of therapy, including patients who received antibody-drug conjugates. In the recommended Phase 2 dose (RP2D) biomarker positive cohort, the overall response rate was 43% (3 out of 7 patients) with the duration of response exceeding one year and ongoing. The disease control rate was 100% with several patients approaching two years on treatment with dosing ongoing. REM-422 was generally well tolerated at the RP2D with no dose-limiting toxicities observed."

P1 data • Adenoid Cystic Carcinoma

Targeting MYB with REM-422 for the treatment of pediatric T-cell acute lymphoblastic leukemia - A report from the Pediatric Preclinical Testing Consortium (PIVOT) (AACR 2026) - P1, P1/2 | "REM-422 exhibited potent single-agent in vivo activity against a large panel of pediatric T-ALL PDXs, supporting its potential clinical evaluation in this disease. Future research directions will include efforts to identify effective combinations with targeted and/or standard-of-care drugs."

Preclinical • Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor • T Acute Lymphoblastic Leukemia • T-cell Acute Lymphoblastic Lymphoma

Remix Therapeutics Announces Oral Presentation of First-in-Class Small Molecule MYB mRNA Degrader REM-422 Phase 1/2 ARIA Trial Results at the 2026 American Society of Clinical Oncology Annual Meeting (GlobeNewswire) - "The presentation...will highlight results from the Phase 1 portion of the study evaluating the safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of REM-422 in patients with R/M ACC, a disease driven by MYB dysregulation, for which there are no approved systemic therapies."

P1 data • Adenoid Cystic Carcinoma

REM-422, a first-in-class mRNA degrader of the MYB oncogene, demonstrates anti-tumor activity in xenograft models of adenoid cystic carcinoma and acute myeloid leukemia (AACR 2025) - P1, P1/2 | "Immunohistochemistry staining confirmed the reduction of MYB protein in the bone marrow of treated animals. Flow cytometry analysis of bone marrow and whole blood demonstrated that REM-422 treatment led to an increase in blasts expressing CD14 and CD11b, known myeloid differentiation markers, as well as an increased frequency of Annexin V+ blasts, a marker of early apoptosis.These data illustrate the potential for REM-422 as an investigational agent for the treatment of a variety of solid tumor and hematological malignancies."

Preclinical • Acute Myelogenous Leukemia • Adenoid Cystic Carcinoma • Colorectal Cancer • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Solid Tumor • ANXA5 • CD14 • ITGAM • MYB

Remix Therapeutics Granted FDA Fast Track Designation for REM-422 for the Treatment of Recurrent, Metastatic or Unresectable Adenoid Cystic Carcinoma (The Manila Times) - "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."

Fast track • Adenoid Cystic Carcinoma

REM-422-101: Study of REM-422 in Patients With Recurrent, Metastatic, or Unresectable Adenoid Cystic Carcinoma (clinicaltrials.gov) - P1/2 | N=100 | Recruiting | Sponsor: Remix Therapeutics | Phase classification: P1 ➔ P1/2

Phase classification • Adenoid Cystic Carcinoma • Oncology

REM-422, a Small Molecule MYB mRNA Degrader, Demonstrates Anti-Leukemic Activity As Monotherapy and in Combination with Standards of Care in Preclinical Models of AML (ASH 2024) - P1 | "The activity of REM-422 was assessed in vitro in combination with AML standards of care by co-treating leukemia cell lines with REM-422 and other agents including venetoclax, gilteritinib, revumenib, and azacitidine. The REM-422 and revumenib combination arm demonstrated more durable activity with delayed tumor regrowth kinetics after cessation of dosing compared to the monotherapy arms. Altogether, these data support the therapeutic potential for REM-422 in AML patients both as monotherapy and in combination therapy."

Combination therapy • IO biomarker • Monotherapy • Preclinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Adenoid Cystic Carcinoma • Hematological Malignancies • Leukemia • Lymphoma • Oncology • ANXA5 • BCL2 • CD14 • FLT3 • ITGAM • KMT2A • MYB

REM-422, a Potent, Selective, Oral Small Molecule mRNA Degrader of the MYB Oncogene, Demonstrates Anti-Tumor Activity in Mouse Xenograft Models of AML (ASH 2023) - "Flow cytometry analysis of bone marrow and peripheral blood samples demonstrated that REM-422 led to the eradication of human leukemia cells from both compartments (Figure 1 right). Together, these data demonstrate that REM-422 treatment leads to degradation of MYB mRNA in preclinical models of AML and supports the therapeutic potential for REM-422 in AML patients."

IO biomarker • Preclinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Adenoid Cystic Carcinoma • Brain Cancer • CNS Tumor • Glioma • Hematological Malignancies • Leukemia • Oncology • Pediatrics • Solid Tumor • T Acute Lymphoblastic Leukemia • T-cell Acute Lymphoblastic Lymphoma • BCL2 • FLT3 • MYB

REM-422, a first-in-class mRNA degrader of the MYB oncogene (AACR-NCI-EORTC 2025) - No abstract available

Oncology

A phase I trial of the MYB RNA degrader REM-422 in patients with recurrent or metastatic (R/M) adenoid cystic carcinoma (ACC) (AACR-NCI-EORTC 2025) - P1 | "REM-422 is the first oral RNA degrader of MYB demonstrating proof-of-mechanism in both blood and tumor tissue along with a favorable safety profile. Anti-tumor activity was observed in patients with R/M ACC, particularly among biomarker (+) patients, where novel therapies are urgently needed."

Clinical • Metastases • P1 data • Adenoid Cystic Carcinoma • Oncology

REM-422-101: Study of REM-422 in Patients With Recurrent or Metastatic Adenoid Cystic Carcinoma (clinicaltrials.gov) - P1 | N=100 | Recruiting | Sponsor: Remix Therapeutics | N=65 ➔ 100

Enrollment change • Adenoid Cystic Carcinoma • Oncology

Remix Therapeutics Announces Positive Preliminary Data from Phase 1 Study of REM-422 in Patients with Adenoid Cystic Carcinoma at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics (GlobeNewswire) - "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 R/M ACC, particularly among biomarker positive patients. As of October 1, 2025, the overall response rate (via RECIST) was 43% in the efficacy population....In the subset of biomarker positive patients that were on study for more than 6 months, tumor shrinkage of more than 20% was observed in 71% (10/14) with 6 partial responses (4 confirmed) ongoing at 4-6 months."

P1 data • Adenoid Cystic Carcinoma

Remix Therapeutics to Present Phase 1 Data of REM-422 at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics Conference in Late-Breaking Oral Presentation (GlobeNewswire) - "The presentation will highlight results from the ongoing Phase I trial’s primary objectives of defining safety, dose-limiting toxicities, and a recommended phase 2 dose (RP2D). Secondary objectives include evaluating preliminary antitumor activity and pharmacokinetics of REM-422 in patients with R/M ACC."

Late-breaking abstract • P1 data • Adenoid Cystic Carcinoma

REM-422-101: Study of REM-422 in Patients With Recurrent or Metastatic Adenoid Cystic Carcinoma (clinicaltrials.gov) - P1 | N=65 | Recruiting | Sponsor: Remix Therapeutics | Trial completion date: Jun 2026 ➔ Jun 2027 | Trial primary completion date: Jun 2025 ➔ Jun 2026

Trial completion date • Trial primary completion date • Adenoid Cystic Carcinoma • Oncology

REM-422, A SMALL MOLECULE MYB MRNA DEGRADER, DEMONSTRATES ANTI-LEUKEMIC ACTIVITY AS A MONOTHERAPY AND IN COMBINATION WITH STANDARDS OF CARE IN PRECLINICAL MODELS OF AML (EHA 2025) - P1 | "REM-422's anti-proliferative activity was unchanged in the resistant models, which is consistent with the differentiated mechanism of action of REM-422 compared to these standards of care.The activity of REM-422 was assessed in vitro in combination with AML standards of care by co-treating leukemia cell lines with REM-422 and other agents including venetoclax, gilteritinib, revumenib, and azacitidine. These data support the therapeutic potential for REM-422 in AML patients both as monotherapy and in combination therapy."

Combination therapy • Monotherapy • Preclinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ANXA5 • CD14 • FLT3 • ITGAM • KMT2A • MYB

Remix Therapeutics to Present Preclinical Data Demonstrating Anti-Leukemic Activity of REM-422 in AML at the 66th American Society of Hematology Annual Meeting and Exposition (ASH) (PRNewswire) - "Remix Therapeutics...announced an upcoming oral presentation at the 66th ASH Annual Meeting taking place from December 7-10, 2024, in San Diego, CA. Results demonstrate oral dosing of REM-422, a selective mRNA degrader of the MYB oncogene, leads to robust anti-leukemic activity observed both as a monotherapy and in combination across a genetically diverse set of preclinical models of acute myeloid leukemia (AML), including eradication of AML blasts in engrafted patient-derived xenograft (PDX) models of AML."

Preclinical • Acute Myelogenous Leukemia

REM-422, a potent, selective, oral small molecule mRNA degrader of the MYB oncogene, induces regressions in mouse patient-derived xenograft models of adenoid cystic carcinoma (EORTC-NCI-AACR 2024) - P1 | "These data demonstrate that REM-422 monotherapy treatment leads to degradation of MYB mRNA and protein resulting in tumor regressions in preclinical PDX models of ACC and supports the therapeutic potential for REM-422 in ACC patients."

Preclinical • Acute Myelogenous Leukemia • Adenoid Cystic Carcinoma • Hematological Malignancies • Leukemia • Oncology • MYB • NFIB

Remix Therapeutics to Present Preclinical Data Demonstrating Tumor Regressions Induced by REM-422 in Adenoid Cystic Carcinoma Patient-Derived Xenograft Models at the 2024 EORTC-NCI-AACR Symposium (PRNewswire) - "Remix Therapeutics...will deliver a poster presentation demonstrating the therapeutic potential of REM-422, a potent, selective, oral small molecule MYB mRNA degrader for the treatment of acute myeloid leukemia (AML) and adenoid cystic carcinoma (ACC), at the upcoming 2024 EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics being held on October 23-25, 2024 in Barcelona, Spain....The poster presentation will include preclinical data from biophysical and cellular assays that demonstrate the mechanism of action of REM-422 in suppressing MYB mRNA and protein expression by promoting inclusion of a poison exon into the MYB mRNA transcript. In vivo studies in ACC patient-derived xenograft (PDX) mouse models harboring the MYB::NFIB fusion demonstrates that REM-422 induces tumor regressions, including in models that harbor co-occurring mutations in the Notch pathway, a profile that represents especially aggressive clinical cases of ACC."

Preclinical • Adenoid Cystic Carcinoma

Tempus Announces Expanded Data and Sequencing Collaboration with Remix Therapeutics (Businesswire) - "Tempus AI, Inc...announced an expanded collaboration with Remix Therapeutics, a clinical-stage biotechnology company developing small molecule therapies to modulate RNA processing and address underlying drivers of disease. The collaboration between Remix and Tempus began with the licensing of specific, de-identified data cohorts and has since expanded into a broader, strategic alliance. Remix is leveraging Tempus’ multimodal data to interrogate specific cohorts, including Adenoid Cystic Carcinoma (ACC) and Acute Myeloid Leukemia (AML) with Tempus’ data analytics platform, Lens. The newly expanded scope of work also includes next-generation sequencing support for Remix’s Phase I trial for REM-422, the company’s potent, selective, oral small molecule messenger RNA (mRNA) degrader."

Diagnostic • Licensing / partnership • Acute Myelogenous Leukemia • Adenoid Cystic Carcinoma • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Solid Tumor

REM-422, A POTENT, SELECTIVE, ORAL SMALL MOLECULE MRNA DEGRADER OF THE MYB ONCOGENE, DEMONSTRATES ANTI-TUMOR ACTIVITY IN MOUSE XENOGRAFT MODELS OF AML (EHA 2024) - "These data demonstrate that REM-422 treatment leads to degradation of MYB mRNA in preclinical models ofAML and supports the therapeutic potential for REM-422 in AML patients."

IO biomarker • Preclinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Adenoid Cystic Carcinoma • Brain Cancer • CNS Tumor • Glioma • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Pediatrics • Solid Tumor • T Acute Lymphoblastic Leukemia • BCL2 • FLT3 • MYB

Remix Therapeutics to Present Preclinical Data Demonstrating Anti-Tumor Activity of REM-422 in AML at the European Hematology Association (EHA) 2024 Congress (PRNewswire) - "Remix Therapeutics (Remix)...will deliver a poster presentation demonstrating the therapeutic potential of REM-422...at the European Hematology Association (EHA) 2024 Hybrid Congress in Madrid, Spain. The poster presentation will include preclinical data from MYB-dependent AML cell lines and human leukemia xenograft models showing REM-422 is broadly active across various AML models. Furthermore, REM-422 induces tumor regressions in cell line-derived xenograft (CDX) and patient-derived xenograft (PDX) models of AML at well-tolerated doses."

Preclinical • Acute Myelogenous Leukemia