RGLS8429 / Regulus Therapeutics - LARVOL DELTA

A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease (clinicaltrials.gov) - P1 | N=68 | Completed | Sponsor: Regulus Therapeutics Inc. | Recruiting ➔ Completed

Trial completion • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Nephrology • Polycystic Kidney Disease • Renal Disease

RGLS8429 Increases Urinary PC1 and PC2 and May Reduce Height-Adjusted Total Kidney Volume (htTKV) in Patients with ADPKD (KIDNEY WEEK 2024) - "Results provide clinical proof of dose-responsive RGLS8429 mechanistic activity on PC1 and PC2. Exploratory analysis suggests a reduction of htTKV at 2 and 3 mg/kg doses over a relatively short treatment period. Findings validate miR-17 as a potential therapeutic target for ADPKD."

Clinical • Late-breaking abstract • Autosomal Dominant Polycystic Kidney Disease • Polycystic Kidney Disease • MIR17 • PKD1 • PRKD1

RGLS8429-Mediated miR-17 Inhibition Leads to Acute PKD1/2 De-repression and Ameliorates Preclinical ADPKD (KIDNEY WEEK 2024) - "Thus, our goals were to characterize the next-gen anti-miR-17 drug RGLS8429 and its impact on PKD1/2 and ADPKD. We treated murine and human ADPKD kidney cell lines and mouse models with PBS, control oligo, or RGLS8429 to assess for miR-17 inhibition, PKD1/2 de-repression, and therapeutic efficacy before or after PKD onset and in synergy with tolvaptan. RGLS8429-mediated acute pharmaceutical PKD1/2 de-repression holds the potential for a significant disease-modifying effect in ADPKD. This promising drug is currently being evaluated in Phase 1b ADPKD clinical trials."

Preclinical • Autosomal Dominant Polycystic Kidney Disease • Polycystic Kidney Disease • MIR17 • PKD1 • PRKD1

A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease (clinicaltrials.gov) - P1 | N=70 | Recruiting | Sponsor: Regulus Therapeutics Inc. | N=36 ➔ 70 | Trial completion date: Dec 2023 ➔ Jun 2025 | Trial primary completion date: Dec 2023 ➔ Apr 2025

Enrollment change • Trial completion date • Trial primary completion date • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Nephrology • Polycystic Kidney Disease • Renal Disease • KIM1

A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease (clinicaltrials.gov) - P1 | N=36 | Recruiting | Sponsor: Regulus Therapeutics Inc.

Trial completion date • Trial primary completion date • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Nephrology • Polycystic Kidney Disease • Renal Disease • KIM1

A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease (clinicaltrials.gov) - P1 | N=36 | Recruiting | Sponsor: Regulus Therapeutics Inc. | Phase classification: P1b ➔ P1

Phase classification • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Nephrology • Polycystic Kidney Disease • Renal Disease • KIM1

Discovery of Next-Generation Anti-miR-17 Oligonucleotide RGLS8429 for Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) (KIDNEY WEEK 2022) - "Importantly, treatment with the first-generation anti-miR-17 oligonucleotide RGLS4326 (1mg/kg) resulted in a statistically significant increase in urinary exosome PC1 and PC2 levels in patients with ADPKD. As RGLS8429 did not cause off-target binding and inhibition of the AMPA-R, no CNS-related toxicity was observed in single- or repeat-dose toxicity studies in mice and monkeys. Conclusion A Phase 1b clinical trial evaluating RGLS8429 in ADPKD patients is planned for the second-half of 2022."

Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Nephrology • Polycystic Kidney Disease • Renal Disease • MIR17 • PKD1 • PRKD1

A Study of RGLS8429 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=36 | Completed | Sponsor: Regulus Therapeutics Inc. | Active, not recruiting ➔ Completed

Trial completion

A Study of RGLS8429 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=36 | Active, not recruiting | Sponsor: Regulus Therapeutics Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed

A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease (clinicaltrials.gov) - P1b | N=36 | Recruiting | Sponsor: Regulus Therapeutics Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Nephrology • Polycystic Kidney Disease • Renal Disease • KIM1

A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease (clinicaltrials.gov) - P1b | N=36 | Not yet recruiting | Sponsor: Regulus Therapeutics Inc.

New P1 trial • Autosomal Dominant Polycystic Kidney Disease • Genetic Disorders • Nephrology • Polycystic Kidney Disease • Renal Disease • KIM1

A Study of RGLS8429 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=32 | Recruiting | Sponsor: Regulus Therapeutics Inc.

New P1 trial