Regulus Therapeutics Inc. (NASDAQ: RGLS) has announced prioritizing of its next-gen RGLS8429 drug candidate for Autosomal Dominant Polycystic Kidney Disease treatment.
Regulus finalized dosing in IND-enabling studies
Recently the company finalized the dosing period for the in-life part of the IND-enabling toxicity trials. Regulus anticipates submitting an Investigational New Drug application and initiating a phase 1 trial in Q2 2022, subject to clearance from the FDA. Based on second cohort data of patients in the phase 1b study of first-gen RGLS4326 in ADPKD and discussions with the FDA, Regulus holds the RGLS8429’s strategic prioritization represents a judicious use of resources instead of continuing with RGLS4326 development based on dose limitations and duration of therapy.
CEO Jay Hagan said, “In light of our discussions with FDA and early analysis of data from the second cohort of our Phase 1b trial in ADPKD, we have determined that advancing our next-generation compound RGLS8429 is more compelling than further development of RGLS4326. The extensive work and investment we have made in RGLS4326 will directly inform the advancement of RGLS8429, and we believe will make this transition both expeditious and productive. This prioritization of RGLS8429 is supported both by robust data in preclinical models, where we have seen clear improvements in kidney function, size, and other measures of disease severity, as well as the compound’s superior pharmacologic profile.”
RGLS8429 has shown a superior profile in IND-enabling studies
With the dosing phase of the IND-enabling toxicity studies completed, the company believes RGLS8429 has shown a superior profile, including off-target CNS effects absence witnessed with RGLS4326 at the highest doses evaluated in chronic preclinical toxicology studies, and equal potency for the molecular target (miR-17) in both in-vivo and in-vitro efficacy studies. As a result, regulus will meet the FDA for a pre-IND meeting for RGLS8429 before the end of the year and plans to submit an IND and begin a Phase 1 clinical trial in Q2 2022.
