Regulus Therapeutics Unveils Promising Results in Phase 1b Clinical Trial of RGLS8429 for the Treatment of ADPKD

Regulus Therapeutics, Inc. (Nasdaq: RGLS) is thrilled to announce that it is progressing to the second cohort of patients in the Phase 1b MAD study of RGSL8429 for the treatment of ADPKD! After a comprehensive review of all available safety data, the biopharmaceutical company is confident that its innovative drug targeting microRNAs will be beneficial in treating the condition. Regulus is committed to providing the best possible treatments for its patients and is excited to take this next step in its research and development journey.

We are thrilled to announce the launch of the second cohort of patients in our Phase 1b MAD trial for the development of a novel treatment for ADPKD. This marks another major milestone as we progress towards our goal of providing an effective treatment for this condition.

With topline data from the first cohort expected towards the end of the third quarter of this year, we remain committed to our mission of improving the lives of those living with ADPKD.

The Phase 1b MAD study is an exciting double-blind, placebo-controlled trial that is evaluating the safety, tolerability, and pharmacokinetics and pharmacodynamics (PK/PD) of RGLS8429 in adult patients with ADPKD.

Following a successful safety review of the first cohort, which involved all 12 patients receiving 1 mg/kg of RGLS8429 or placebo every other week for three months, the second cohort will receive a dose of 2 mg/kg of RGLS8429 or placebo every other week for the same period.

Additionally, the 27-week chronic toxicity study of RGLS8429 in mice showed no toxic effects, including CNS effects, at any dose level up to the highest dose of 300 mg/kg administered every other week. These encouraging findings are paving the way for the continued development of this promising drug.


ADPKD, an Autosomal Dominant Polycystic Kidney Disease caused by mutations of the PKD1 or PKD2 genes, is a devastatingly common monogenic disorder and a major cause of end-stage renal disease. It is characterized by the development of multiple fluid-filled cysts in the kidneys, and to a lesser extent, the liver and other organs.

With an estimated global prevalence of 4 to 7 million, and approximately 160,000 individuals in the US alone diagnosed with ADPKD, it is particularly concerning that excessive kidney cyst cell proliferation leads to end-stage renal disease in around half of those affected by age 60.

About RGLS8429

Regulus Therapeutics recently unveiled RGLS8429, a novel oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and preferentially target the kidney. The Phase 1 SAD study has confirmed the drug’s favorable safety and PK profile, with no serious adverse events reported.

Now, the Phase 1b MAD study has completed enrollment for its first cohort of patients and is advancing to the second cohort, where participants will receive 2 mg/kg of RGLS8429 or placebo every other week for three months.

Preclinical models have demonstrated robust improvements in kidney function, size, and other measures of disease severity, as well as a superior pharmacologic profile when compared to Regulus’ first-generation compound, RGLS4326.

About Regulus

Regulus Therapeutics Inc. (Nasdaq: RGLS) is a cutting-edge biopharmaceutical company that strives to discover and develop revolutionary medicines that target microRNAs. Regulus has harnessed their talents in oligonucleotide drug discovery and development to cultivate a pipeline of potential treatments and build an impressive intellectual property estate in the microRNA field. As a leader in the industry, Regulus proudly calls San Diego, CA its corporate home.

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