Regulus Therapeutics Reports Fourth Quarter and Year-End 2022 Financial Results and Recent Updates.

SAN DIEGO: Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today reported financial results for the fourth quarter and year ended December 31, 2022, and provided a corporate update.

"We ended 2022 in a position of strength, with enrollment in our Phase 1b MAD study under way. This momentum was reinforced by the appointments of Drs. Rekha Garg and Claire Padgett, who bring significant depth of industry, clinical and regulatory experience to help us advance development of RGLS8429," commented Jay Hagan, CEO of Regulus. "With three months of dosing and one month of follow-up for each patient in the study, we would anticipate topline data approximately five months after we complete enrollment of the first cohort."

Program Updates

RGLS8429 for ADPKD: The Phase 1b MAD study is a double-blind, placebo-controlled trial to assess safety, tolerability, and pharmacokinetics of RGLS8429 in adult patients with ADPKD. The study will evaluate the safety and efficacy of RGLS8429 treatment across three different dose levels, including measuring changes in polycystins, height-adjusted total kidney volume (htTKV), and overall kidney function. The first cohort is being dosed at 1 mg/kg of RGLS8429 or placebo every other week for three months, with top-line data anticipated in the second half of 2023. The company also recently completed the in-life portion of the 27-week chronic mouse toxicity study for RGLS8429. No CNS toxicity was observed at all dose levels up to the top dose of 300 mg/kg administered every other week.

Corporate Highlights

Expanded Team: In November 2022, the Company announced the appointments of Rekha Garg, M.D., M.S., to Senior Vice President, Clinical Development and Regulatory, and Claire Padgett, Ph.D., to Senior Vice President, Clinical Operations. In addition, the company announced the promotions of Morgan Carlson, Ph.D., to Vice President, Biology and Edmund Lee, Ph.D., to Vice President, Translational Medicine.

Presented Data at the 5th Chronic Kidney Disease Drug Development Summit: On March 9, 2023, the Company presented preclinical data on RGLS8429 at the 2023 CKD Summit, entitled "Targeting miR-17 to Address the Genetic Cause of ADPKD." Drs. Edmund Lee and Rekha Garg represented...