FX.co ★ Regulus Therapeutics Provides Positive Clinical And Regulatory Updates For ADPKD Program

Regulus Therapeutics Inc. (RGLS) announced on Wednesday encouraging clinical and regulatory developments within its autosomal dominant polycystic kidney disease (ADPKD) initiative.

Recent interim analysis from the fourth cohort of the Phase 1b Multiple Ascending Dose (MAD) study on farabursen, aimed at treating ADPKD, revealed a consistent mechanistic dose response. Furthermore, the safety and tolerability profile aligned with findings from previous cohorts.

The company also reported a successful meeting with the U.S. Food and Drug Administration (FDA) in December, marking the end of Phase 1. During this meeting, they reached an agreement on the key aspects of a Phase 3 pivotal trial. This trial holds the potential for accelerated approval of farabursen. Jay Hagan, CEO of Regulus, expressed optimism following the FDA's feedback and the positive interim results, stating, "We are encouraged by the feedback from the agency and are excited about the path forward for farabursen in ADPKD."