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ATR 1072 · 1 trial · 4 indications
Incidence and severity of treatment-emergent adverse events (TEAEs).
| Arm | Type | Description |
|---|---|---|
| Part A - MAD Dose Escalation Description Three sequential ascending dose | EXPERIMENTAL | Three sequential ascending dose cohorts (Dose Level 1, Dose Level 2 and Dose Level 3) with approximately 4 evaluable participants per cohort. ATR 1072 administered intravenously (IV) every 6 weeks (Q6W) for up to 8 doses (48-week treatment period). |
| Part B - Expansion Cohort at the Recommended Phase 2 Dose | EXPERIMENTAL | Approximately 25 participants enrolled at the recommended phase 2 dose determined in Part A. ATR 1072 administered IV Q6W to further evaluate safety, PK, and pharmacodynamics. |
| Name | Type | Description |
|---|---|---|
| ATR 1072 | DRUG | ATR 1072 is an investigational therapy that delivers an siRNA to reduce PRKAG2 gene expression. Administered as an intravenous (IV) infusion every 6 weeks (Q6W). Formulated as a sterile liquid for injection. |
Inclusion Criteria: 1. Adults 18 to 65 years of age. 2. Pathogenic, likely pathogenic, or variant of uncertain significance (VUS) in PRKAG2 confirmed by genetic testing. 3. Clinical manifestations consistent with PRKAG2 Syndrome. 4. Able and willing to comply with study procedures. 5. Able and will...
ATR 1072 is an investigational small molecule being developed for the treatment of cardiac glycogenosis, a condition characterized by abnormal glycogen accumulation in the heart. It is also being studied in related conditions such as hypertrophic cardiomyopathy and Wolff-Parkinson-White syndrome, which are associated with PRKAG2 syndrome.
ATR 1072 targets the PRKAG2 gene. PRKAG2 encodes the regulatory subunit of AMP-activated protein kinase, and mutations in this gene are linked to cardiac glycogenosis and associated cardiomyopathy. By targeting this gene, ATR 1072 aims to address the underlying genetic cause of the disease.
ATR 1072 is being developed by Atrium Therapeutics, Inc., a biopharmaceutical company. The company's stock is listed under the ticker symbol RNA. Atrium Therapeutics is conducting clinical research to evaluate the safety and efficacy of ATR 1072 in patients with PRKAG2 syndrome.
ATR 1072 is currently in Phase 1 clinical development. It is an investigational drug and has not yet been approved by regulatory authorities. The ongoing Phase 1 trial is designed to assess the safety, tolerability, and pharmacokinetics of ATR 1072 in adult participants with PRKAG2 syndrome.
ATR 1072 is being evaluated in a Phase 1 clinical trial with the identifier NCT07738107. This trial, titled 'ATR 1072 in Participants With PRKAG2 Syndrome,' is not yet recruiting and plans to enroll 37 participants. The study is a controlled trial that includes adults aged 18 years and older.
No alternative names for ATR 1072 have been reported. It is identified solely by its developmental code name ATR 1072. As a novel small molecule targeting PRKAG2, it is distinct from other therapies and is being investigated specifically for cardiac glycogenosis and related PRKAG2 syndrome manifestations.