Study of REM-422 in Patients With Recurrent, Metastatic, or Unresectable Adenoid Cystic Carcinoma

NCT06118086 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: REM-422-101
• Study Type: interventional
• Target: 100
• Locations: 2 countries
• Sites: 8

Brief Summary

The goal of this study is to determine the safety and antitumor effects of REM-422, a MYB mRNA degrader, in people with advanced Adenoid Cystic Carcinoma (ACC)

Conditions

Adenoid Cystic Carcinoma; Metastatic Adenoid Cystic Carcinoma; Recurrent Adenoid Cystic Carcinoma

Outcome Measures

Primary Outcomes (3)

• Frequency and severity of Treatment Emergent Adverse Events (TEAEs)

  Frequency and severity of Treatment Emergent Adverse Events (TEAEs) will be evaluated according to the NCI-CTCAE version 5.0 and number of participants with Dose Limiting Toxicities will be assessed to determine Safety and Tolerability of REM-422

  18 months

• Overall Response Rate (ORR) in Phase 2 Confirmatory Cohort

  ORR will be evaluated using the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline version 1.1 following treatment with REM-422

  18 months

• Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D)

  Frequency and severity of Treatment Emergent Adverse Events (TEAEs) will be evaluated according to the NCI-CTCAE version 5.0 and the number of participants with Dose Limiting Toxicities will be assessed

  Assessed at the end of Cycle 1 for each participant

Secondary Outcomes (9)

• Median Progression Free Survival (mPFS)

  6 months, 12 months

• Duration of Response (DoR)

  18 months

• Time to Response (mTTR)

  18 months

• Disease Control Rate (DCR)

  6 months

• Median Overall Survival (mOS)

  18 months

Study Arms (1)

REM-422

EXPERIMENTAL

* Dose Escalation: Participants will receive escalating doses of REM-422 to determine Maximum Tolerated Dose (MTD) and/or Recommended Phase 2 Dose (RP2D)-422, oral capsule administered once daily * Ph 2 Confirmatory Cohort: Participants will receive REM-422 at the identified RP2D * Treatment will continue until disease progression, therapy intolerance, or participant withdrawal * Safety evaluation will continue until 30 days of last administration of REM-422

Drug: REM-422

Interventions

REM-422 DRUG

* REM-422 is a first in class, small molecule mRNA inhibitor that reduces expression of the MYB transcription factor * REM-422 will be administered orally once daily

REM-422

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Be able to provide informed consent.
• Be 18 years or older at the time of informed consent.
• Disease criteria:
• Histologically confirmed ACC, any site of origin.
• Dose Escalation phase ONLY:
• Have locally advanced or metastatic ACC
• Evidence of radiographic progression and/or signs and symptoms associated with their disease (eg, pain, dyspnea, reduced performance status). Participants who have stable disease while being treated with another agent that is not tolerated are eligible after the appropriate washout period.
• Confirmatory Cohort phase ONLY:
• Have metastatic, recurrent, or unresectable ACC
• Measurable disease at the time of enrollment. At least 1 measurable lesion according to RECIST v1.1 criteria. Participants must have radiographic evidence of disease progression by RECIST v1.1 criteria ≤ 6 months prior to study enrollment. Radiographic eligibility as determined by Central IUO assay.
• MYB poison exon biomarker positive tumor(s) confirmed by central IUO assay.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
• Tumor Tissue Requirements
• Dose Escalation Phase ONLY: be able to provide during Screening a tissue specimen of either a fresh biopsy of a non-target lesion or an archival tumor sample obtained within the last 6 years. A formalin-fixed paraffin-embedded (FFPE) block can be submitted or a minimum of 15 freshly sectioned unstained slides. Agree to an on-treatment biopsy to be obtained \~4-8 weeks after initiation of REM-422 unless medically contraindicated.
• Confirmatory Cohort phase ONLY: be able to provide, during Pre-Screening, a tissue specimen of either a fresh biopsy of non-targetable lesion or an archival tumor sample obtained within the last 6 years that is interpretable for the biomarker positivity. An FFPE block can be submitted or a minimum of 15 fresh sectioned unstained slides.

You may not qualify if:

• Known hypersensitivity or contraindication to any component of REM-422 or to drugs chemically related to REM-422 or its excipients.
• Clinically significant active infection. Simple urinary tract infection, uncomplicated bacterial pharyngitis responding to active treatment are permitted. Participants receiving intravenous antibiotics ≤ 7 days prior to enrollment are excluded (prophylactic antibiotics, antivirals or antifungals are permitted).
• Evidence of active HIV infection.
• Evidence of currently active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection.
• Primary immunodeficiency.
• Current or expected need for daily systemic corticosteroid therapy ≥ 10 mg of prednisone equivalent. Topical or inhaled corticosteroids with minimal systemic absorption may enroll and continue minimal corticosteroids if the participant is on a stable dose.
• Live vaccine ≤ 6 weeks prior to the start of REM-422.
• Use of strong CYP3A inhibitors or CYP3A inducers
• Drugs that reduce gastric acidity, such as H2-receptor antagonists (eg, ranitidine, famotidine) and proton pump inhibitors (e.g., omeprazole, esomeprazole) within 7 days prior to the initiation of REM-422 administration or during the study
• Pregnancy or participants planning to become pregnant during the duration of the study, or lactation.
• Participants with malabsorption syndrome, a disease significantly affecting gastrointestinal function, or resection of the stomach or bowel.
• Current use of prohibited medication ≤ 1 week before starting REM-422.
• Clinically significant cardiovascular disease:
• Participants who have undergone major surgery (opening a mesenchymal barrier such as the pleural cavity, peritoneum, meninges, or surgical procedures requiring general anesthesia) \< 4 weeks prior to enrollment.
• History of organ transplant that requires use of immunosuppressive agents.

Sponsors & Collaborators

• Remix Therapeutics: lead

Study Sites (8)

University of California San Francisco Helen Diller Comprehensive Cancer Center

San Francisco, California, 94143, United States

RECRUITING

Dana Farber Cancer Research Institute

Boston, Massachusetts, 02215, United States

RECRUITING

University of Michigan Rogel Cancer Center

Ann Arbor, Michigan, 48109, United States

RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

RECRUITING

Sarah Cannon Research Institute

Nashville, Tennessee, 37203, United States

RECRUITING

MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Centre Antoine Lacassagne

Nice, 06189, France

NOT YET RECRUITING

Institut de Cancerologie Gustave-Roussy

Villejuif, 94805, France

NOT YET RECRUITING

MeSH Terms

Conditions

Carcinoma, Adenoid Cystic

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms

Study Officials

• Mythili Koneru, MD, PhD

  Remix Therapeutics

  STUDY CHAIR

Central Study Contacts

Remix Therapeutics

CONTACT

781-827-0902; ClinicalTrials@remixtx.com

Rosalie Jiang

CONTACT

781-584-9390; rjiang@remixtx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 13, 2023
• First Posted: November 7, 2023
• Study Start: December 20, 2023
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2027
• Last Updated: February 18, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (6); FR (2)