Regulus Therapeutics Inc (NASDAQ:RGLS) has completed a pre-investigational new drug (Pre-IND) meeting with the FDA to obtain input on the Company's RGLS8429 for Autosomal Dominant Polycystic Kidney Disease (ADPKD).
- ADPKD is a genetic disorder characterized by the growth of numerous cysts in the kidneys.
- The Company will submit an IND application in Q2 of 2022 for the two-part Phase 1 trial.
- Part 1 will consist of a single-ascending dose study in 32 healthy volunteers to assess the safety and tolerability of RGLS8429 and characterize the pharmacokinetics.
- Subjects will be randomized to RGLS8429 or placebo into one of four sequential cohorts. Data expected in 2H Of 2022.
- Part 2 will be a multiple ascending dose study to evaluate the dose-response of RGLS8429 in 36 ADPKD patients, randomized to RGLS8429 or placebo into one of three sequential cohorts.
- Data readout is expected in 1H of 2023.
- In October 2021, the Company discontinued the development of RGLS4326 to allocate resources and efforts towards RGLS8429.
- The Company believes RGLS8429 has demonstrated a superior pharmacological profile, with the absence of the off-target central nervous system effects seen with RGLS4326 in preclinical toxicology studies.
- Price Action: RGLS shares closed at $0.24 on Thursday.
