"Completion of enrollment in the first cohort of the Phase 1b MAD study is an important milestone in the development of this novel treatment for ADPKD," said Jay Hagan, CEO of Regulus. "Topline data from the first cohort are expected to be available around the end of the third quarter of this year."

The Phase 1b MAD study is a double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics (PK/PD) of RGLS8429 in adult patients with ADPKD. The study will evaluate the safety and PK/PD 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. The Company expects to begin dosing the second cohort following a review of all available cohort 1 safety data, which is planned for May 2023. Cohort 2 patients will receive 2 mg/kg of RGLS8429 or placebo every other week for three months.

More information about the MAD clinical trial is available at clinicaltrials.gov (NCT05521191).