Regulus Therapeutics Reports Favorable Phase 1b MAD Trial Results for ADPKD Drug RGLS8429

Regulus Therapeutics has reported promising outcomes from the second group of participants in a Phase 1b clinical trial for a new drug, RGLS8429, intended to treat Autosomal Dominant Polycystic Kidney Disease (ADPKD). The drug demonstrated a clear dose-dependent response at a dosage of 2mg/kg, as indicated by changes in urinary biomarkers. Notably, patients with the most significant increases in the biomarkers PC1 and PC2 exhibited over 4% reductions in height-adjusted total kidney volume (htTKV) and total kidney cyst volume (TKCV). The third group of the study is fully enrolled, with results expected in mid-2024, and screening for the fourth group will commence in the second quarter of 2024. The company also plans to increase the sample size of the fourth cohort to 30 patients to further investigate the drug's impact on cystic volume in ADPKD patients. The drug's development aligns with the company's focus on microRNA-targeting medicines and follows a successful Phase 1 SAD study of RGLS8429. A conference call to discuss the findings was held on March 12, 2024.

The Phase 1b study is a rigorous, double-blind, placebo-controlled trial assessing the safety, tolerability, and pharmacological effects of RGLS8429 in adults with ADPKD across various dosage levels. The drug targets the inhibition of miR-17, with a particular focus on the kidney, and has shown significant improvements in preclinical models. The company has held a Type D meeting with the FDA to discuss a potential accelerated approval pathway for RGLS8429, based on the results of a single Phase 2 study.

As of December 31, 2023, Regulus Therapeutics had approximately $23.8 million in cash and cash equivalents. More details about the MAD clinical trial can be found at clinicaltrials.gov under the identifier NCT05521191. The company's headquarters are located in San Diego, CA, and it is listed on the NASDAQ stock exchange under the ticker symbol RGLS.

ADPKD is a prevalent genetic disorder characterized by the growth of multiple cysts in the kidneys and is a leading cause of end-stage renal disease. The disease affects an estimated 160,000 people in the United States and has a global prevalence of 4 to 7 million individuals. The development of RGLS8429 represents a significant step towards a potential treatment for ADPKD, which could address the genetic roots of the condition.

Regulus Therapeutics is a biopharmaceutical company dedicated to creating innovative medicines that target microRNAs. The company's expertise in oligonucleotide drug discovery and development has led to a robust pipeline and a strong intellectual property portfolio in the microRNA field.

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