Financial Performance - Regulus reported a net loss of $12.8 million, or $0.20 per share, for Q4 2024, compared to a net loss of $8.1 million, or $0.40 per share, in Q4 2023[8] - Total operating expenses for the year ended December 31, 2024, were $50.0 million, compared to $31.1 million in 2023[15] - General and administrative expenses for Q4 2024 were $4.1 million, up from $2.5 million in Q4 2023[7] Research and Development - Research and development expenses increased to $9.7 million for Q4 2024, up from $5.8 million in Q4 2023, and totaled $35.4 million for the year, compared to $21.2 million in 2023[6] - The Phase 1b MAD trial of farabursen for ADPKD showed a reduction in kidney volume growth rate, with 26 subjects receiving a fixed dose of 300 mg every other week for three months[3] - The interim analysis of the fourth cohort indicated a mechanistic dose response with increases in polycystin biomarker levels[2] Cash and Assets - As of December 31, 2024, Regulus had $75.8 million in cash, cash equivalents, and short-term investments, with an expected cash runway extending into early 2026[6] - The company reported total assets of $84.2 million as of December 31, 2024, compared to $30.8 million in 2023[15] Regulatory and Market Information - The FDA has agreed on key components for a Phase 3 pivotal trial, including a 12-month htTKV endpoint for potential Accelerated Approval[5] - Approximately 160,000 individuals are diagnosed with ADPKD in the United States, with a global prevalence estimated at 4 to 7 million[9]

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]

Core Insights - Regulus Therapeutics Inc. announced positive clinical and regulatory updates from its ADPKD program, including favorable topline results from an interim analysis of the fourth cohort of its Phase 1b Multiple Ascending Dose (MAD) study of farabursen (RGLS8429) [1][4] - The company successfully held an End-of-Phase 1 meeting with the FDA, achieving alignment on key components for a Phase 3 pivotal trial aimed at potential Accelerated Approval [1][4] Clinical Trial Details - The Phase 1b MAD study is a double-blind, placebo-controlled trial assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of farabursen in adult patients with ADPKD [2] - The fourth cohort involved 26 subjects receiving a fixed dose of 300 mg of farabursen every other week for three months, showing a mechanistic dose response and a reduction in height-adjusted total kidney volume (htTKV) growth rate [3][5] - Safety data from all subjects indicated that farabursen was well tolerated [3][9] Regulatory Progress - The End-of-Phase 1 meeting with the FDA confirmed the acceptability of the program's chemistry, manufacturing, and controls (CMC), non-clinical, and clinical pharmacology plans [4] - The trial design for the Phase 3 study includes a 12-month htTKV endpoint for potential Accelerated Approval and a 24-month eGFR endpoint for potential Full Approval [5][6] Biomarker and Efficacy Insights - Exploratory results indicated a notable impact on urinary polycystin levels and htTKV growth rate, consistent across different imaging classes and PKD1 mutations [5][6] - The data suggest a high probability of success in meeting or exceeding targeted htTKV efficacy thresholds [5] Company Overview - Regulus Therapeutics Inc. focuses on developing innovative medicines targeting microRNAs, with a strong pipeline and intellectual property in the microRNA field [10] - The company is headquartered in San Diego, CA, and is publicly traded on Nasdaq under the ticker RGLS [10]

Core Points - Regulus Therapeutics Inc. is participating in two upcoming investor conferences: Evercore 7th Annual HealthCONx Conference on December 3, 2024, and Piper Sandler 36th Annual Healthcare Conference on December 4, 2024 [1] - The live events and replays will be available on the company's investor relations website for 90 days following the presentations [1] Company Overview - Regulus Therapeutics Inc. is a biopharmaceutical company focused on discovering and developing innovative medicines that target microRNAs [2] - The company has developed a pipeline supported by a strong intellectual property portfolio in the microRNA field and is headquartered in San Diego, CA [2]

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]

Announced positive topline data from the third cohort of patients in the Phase 1b MAD study of RGLS8429 in autosomal dominant polycystic kidney disease (ADPKD) On track for an End-Of-Phase 1 meeting by year-end Appointed Rekha Garg, M.D., M.S., to Chief Medical Officer Ended second quarter 2024 with cash, cash equivalents, and investments of $95.9 million; Cash runway into H1 2026 SAN DIEGO, Aug. 8, 2024 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the dis ...

SAN DIEGO, Aug. 6, 2024 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced that members of the management team will present at the Canaccord Genuity 44th Annual Growth Conference on Tuesday, August 13th at 8:30 a.m. ET. A replay of the live presentation will be available under "Events and Presentations" through the investor relations section of ...

SAN DIEGO, July 9, 2024 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced that the Company will participate in the following investor conferences: Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 ...

SAN DIEGO, June 4, 2024 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced that it expects to be added to the broad-market Russell 3000® Index and the Russell 2000® Index at the conclusion of the 2024 Russell US Indexes annual reconstitution, effective at the open of US equity markets on Monday, July 1st, according to a preliminary list of addi ...

SAN DIEGO, May 16, 2024 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today announced the promotion of Rekha Garg, M.D., M.S., to Chief Medical Officer effective May 16, 2024. Dr. Garg previously served as the Company's Senior Vice President, Clinical Development and Regulatory. "Dr. Garg has been leading the development and regulatory teams since joining ...