Regulus reports Q4 EPS (20c), consensus (22c)

In December 31, 2024, Regulus (RGLS) had $75.8M in cash, cash equivalents and short-term investments. The company expects its cash runway to extend into early 2026. “Following the rapid progress in 2024 advancing farabursen for ADPKD, we recently announced interim topline data from 14 subjects in the fourth cohort of patients in the Phase 1b MAD trial, where we continued to see an encouraging safety and tolerability profile and evidence of a mechanistic dose response through increases in polycystin biomarker levels as well as results suggesting a reduction on kidney volume (htTKV) growth rate,” said Jay Hagan, CEO. “Furthermore, we have reached alignment with FDA on the key components of the design of a Phase 3 single pivotal trial. The proposed pivotal trial includes an interim analysis to enable Accelerated Approval based on a 12-month htTKV endpoint to expedite farabursen’s development for patients living with ADPKD, who currently have limited treatment options. We look forward to sharing topline data from all 26 subjects in cohort 4 in the coming weeks.”