Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RGLS8429 · 1 trial · 3 indications
Incidence of adverse events over time
Change from baseline in PC1, PC2, NGAL, and KIM-1 urine biomarkers
| Arm | Type | Description |
|---|---|---|
| RGLS8429 | EXPERIMENTAL | The randomized,double blind part of the study (Part A) will consist of three sequential cohorts of approximately 12 subjects each randomized centrally to receive RGLS8429 or placebo by subcutaneous injection every other week (Q2W) x 7 doses (36 subjects total). * Cohort 1: first dose level of RGLS8429 or placebo * Cohort 2: second dose level of RGLS8429 or placebo * Cohort 3: third dose level of RGLS8429 or placebo |
| Placebo | EXPERIMENTAL | The randomized double blind part of the study (Part A) will consist of three sequential cohorts of approximately 12 subjects each randomized centrally to receive RGLS8429 or placebo by subcutaneous injection every other week (Q2W) x 7 doses (36 subjects total). * Cohort 1: first dose level of RGLS8429 or placebo * Cohort 2: second dose level of RGLS8429 or placebo * Cohort 3: third dose level of RGLS8429 or placebo |
| Open Label Fixed Dose RGLS8429 | EXPERIMENTAL | The open-label fixed dose part of the study (Part B and Cohort 4) will consist of a single cohort of up to 30 subjects each receiving 300 mg RGLS8429. |
| Name | Type | Description |
|---|---|---|
| RGLS8429 | DRUG | Solution for subcutaneous injection |
| Placebo | DRUG | Solution for subcutaneous injection |
Key Inclusion Criteria: 1. Male or female ADPKD patients, 18 to 70 years old 2. Class 1C, 1D, or 1E Mayo Imaging Classification of ADPKD (based upon either the MRI obtained during screening, or a prior MRI obtained within 5 years of screening with documented Mayo classification) 3. eGFR between 30 ...
RGLS8429 is an investigational small molecule developed by Regulus Therapeutics Inc. for autosomal dominant polycystic kidney disease, or ADPKD. It is designed to target microRNAs, a class of RNA molecules. The drug has completed a Phase 1 clinical trial and is not approved for any use.
RGLS8429 is being developed as a treatment for autosomal dominant polycystic kidney disease, a genetic kidney disorder. It is an investigational therapy and has not been approved for marketing. Its only completed clinical study enrolled adults with ADPKD in the United States.
RGLS8429 targets microRNAs, which are short RNA molecules that regulate gene expression. By acting on microRNAs, the small molecule is intended to influence the biological pathways involved in autosomal dominant polycystic kidney disease. Regulus Therapeutics Inc. is the developer of this RNA-targeted candidate.
RGLS8429 is being developed by Regulus Therapeutics Inc., a biopharmaceutical company that trades under the ticker RGLS. The company is advancing the small molecule for autosomal dominant polycystic kidney disease. No partner or collaborator is named for this program.
RGLS8429 has completed a Phase 1 clinical trial. It is an investigational drug and is not FDA approved. The completed study was a randomized, double-blind, controlled Phase 1 trial conducted in the United States. No active trials of RGLS8429 are currently listed.
RGLS8429 has been studied in one completed clinical trial, NCT05521191, titled A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease. The Phase 1 trial enrolled 68 participants aged 18 years and older in the United States and used a randomized, double-blind, controlled design.