Regulus Therapeutics Reports Fourth Quarter and Year-End 2024 Financial Results and Recent Updates

Core Insights - Regulus Therapeutics Inc. reported positive topline results from an interim analysis of the fourth cohort in the Phase 1b multiple-ascending dose clinical trial of farabursen for treating autosomal dominant polycystic kidney disease (ADPKD) [1][2] - The company has reached an agreement with the FDA on key components for a Phase 3 single pivotal trial, which may allow for Accelerated Approval based on a 12-month htTKV endpoint [2][5] Program Updates - The interim analysis involved 14 subjects from the fourth cohort, showing a favorable safety and tolerability profile, with evidence of a mechanistic dose response and a suggested reduction in kidney volume growth rate [2][3] - In the fourth cohort, 26 subjects received a fixed dose of 300 mg of farabursen every other week for three months, with results indicating similar effects on polycystin biomarkers compared to previous cohorts [3][4] - The Phase 1b MAD study is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of farabursen in adult patients with ADPKD [4] Financial Results - As of December 31, 2024, the company had $75.8 million in cash, cash equivalents, and short-term investments, with an expected cash runway extending into early 2026 [6] - Research and development expenses for Q4 2024 were $9.7 million, compared to $5.8 million in Q4 2023, while total R&D expenses for the year were $35.4 million, up from $21.2 million in 2023 [6] - General and administrative expenses for Q4 2024 were $4.1 million, compared to $2.5 million in Q4 2023, with total G&A expenses for the year at $14.7 million, up from $10.0 million in 2023 [7] - The net loss for Q4 2024 was $12.8 million, or $0.20 per share, compared to a net loss of $8.1 million, or $0.40 per share, in Q4 2023 [8][14] About ADPKD - Autosomal dominant polycystic kidney disease (ADPKD) is a common genetic disorder leading to end-stage renal disease, characterized by the development of multiple cysts in the kidneys [9] - Approximately 160,000 individuals are diagnosed with ADPKD in the U.S., with a global prevalence estimated between 4 to 7 million [9] About Farabursen - Farabursen (RGLS8429) is a novel oligonucleotide designed to inhibit miR-17 and target the kidney, showing improvements in kidney function and size in preclinical models [10] - The Phase 1 studies have demonstrated that farabursen is well-tolerated with no serious adverse events reported [10] About Regulus - Regulus Therapeutics Inc. focuses on discovering and developing innovative medicines targeting microRNAs, with a strong intellectual property portfolio in the microRNA field [11]