Regulus Therapeutics Reports Third Quarter 2024 Financial Results and Recent Updates

Core Insights - Regulus Therapeutics has completed enrollment in the fourth cohort of the Phase 1b multiple-ascending dose clinical trial for RGLS8429, targeting autosomal dominant polycystic kidney disease (ADPKD) [1][3] - The company plans to present topline data from this cohort in early 2025, following positive results from earlier cohorts [2][4] - As of September 30, 2024, Regulus reported $87.3 million in cash and equivalents, providing a cash runway into the first half of 2026 [1][8] Program Updates - The fourth cohort of the Phase 1b MAD study involves patients receiving a 300 mg fixed dose of RGLS8429 every other week for three months [3] - Previous cohorts demonstrated a mechanistic dose response, with increases in urinary polycystins and reductions in height-adjusted total kidney volume (htTKV) [4][5] - An End-of-Phase 1 meeting with the FDA is anticipated by the end of 2024 [4] Corporate Highlights - Regulus presented a late-breaking poster at ASN Kidney Week, showcasing data from the first three cohorts, which indicated dose-responsive activity of RGLS8429 [5] - The company has entered into a Patent & Technology License Agreement with The University of Texas at Southwestern Medical Center, acquiring rights to jointly-owned patent rights targeting miR-17 for ADPKD treatment [7] Financial Results - For the third quarter of 2024, research and development expenses were $11.3 million, up from $5.5 million in the same period of 2023 [8][9] - General and administrative expenses increased to $3.9 million from $2.6 million year-over-year [9] - The net loss for the third quarter was $14.1 million, compared to $7.8 million in the same quarter of 2023 [10][16]