Regulus Therapeutics Inc. (Nasdaq: RGLS) is a biopharmaceutical company that focuses on the discovery and development of innovative medicines targeting microRNAs. The company, also known as Regulus, has recently announced the dosing of the first patient in the third cohort of the Phase 1b MAD study for RGSL8429, which is aimed at treating Autosomal Dominant Polycystic Kidney Disease (ADPKD).

Regulus Therapeutics' CEO, Jay Hagan, expressed his excitement about this milestone, saying, "After announcing the completed safety review of available data and the advancement to cohort three last month, we are excited to now be dosing our first patient in the third cohort. Our Phase 1b MAD clinical trial remains on track, and we look forward to sharing topline data from the second cohort of the study in the first quarter of 2024."

The Phase 1b MAD study is a double-blind, placebo-controlled trial that evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics of RGLS8429 in adult ADPKD patients. The study is designed to assess RGLS8429 treatment across three different dose levels, with a focus on measuring changes in polycystins, height-adjusted total kidney volume (htTKV), cyst architecture, and overall kidney function. In the second cohort, patients were dosed at 2 mg/kg of RGLS8429 or placebo every other week for three months. Notably, no issues were identified in this cohort based on the review of all available blinded safety data, which led to the advancement to cohort three. In the third cohort, patients will receive 3 mg/kg of RGLS8429 or placebo every other week for three months.

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is caused by mutations in the PKD1 or PKD2 genes and is one of the most common human monogenic disorders. It is a leading cause of end-stage renal disease and is characterized by the development of multiple fluid-filled cysts, primarily in the kidneys, with some presence in the liver and other organs. The disease is primarily driven by excessive kidney cyst cell proliferation, which eventually leads to end-stage renal disease in about 50% of ADPKD patients by age 60. In the United States, approximately 160,000 individuals are diagnosed with the disease, and the estimated global prevalence is 4 to 7 million.

RGLS8429 is a novel oligonucleotide developed for the treatment of ADPKD. It is designed to inhibit miR-17 and selectively target the kidney. Preclinical studies have shown promising data for RGLS8429, including improvements in kidney function, size, and other measures of disease severity. RGLS8429 exhibits a superior pharmacologic profile compared to Regulus' first-generation compound, RGLS4326. In September 2022, Regulus completed the Phase 1 SAD study, demonstrating the favorable safety and pharmacokinetic profile of RGLS8429. The Phase 1b MAD study is progressing, with the dosing of the third cohort commencing, where patients will receive 3 mg/kg of RGLS8429 or placebo every other week for three months.

 

Source: prnewswire.com

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