Core Viewpoint - Regulus Therapeutics has entered into an acquisition agreement with Novartis, with an initial cash payment of $7.00 per share and potential additional cash through a contingent value right, totaling up to approximately $1.7 billion, expected to close in the second half of 2025 [1][8][9] Company Updates - The company reported positive topline data from the fourth cohort of patients in the Phase 1b multiple-ascending dose study of farabursen for treating autosomal dominant polycystic kidney disease (ADPKD), indicating a mechanistic dose response and halting of kidney volume growth [3][4] - A pivotal Phase 3 trial is set to begin in the third quarter of 2025, with alignment confirmed with the FDA on trial design and endpoints [6][14] Financial Results - As of March 31, 2025, the company had $65.4 million in cash, cash equivalents, and short-term investments, with a cash runway expected to extend into early 2026 [10] - Research and development expenses for Q1 2025 were $6.8 million, up from $6.0 million in Q1 2024, while general and administrative expenses increased to $3.7 million from $2.8 million in the same period [11][12] - The net loss for Q1 2025 was $9.6 million, or $0.15 per share, compared to a net loss of $8.5 million, or $0.29 per share, in Q1 2024 [12][22]

Core Viewpoint - Regulus Therapeutics Inc. is actively participating in the 2025 Oppenheimer Innovation on the Island Biotech Summit, highlighting its focus on innovative medicines targeting microRNAs [1]. Company Overview - Regulus Therapeutics Inc. is a biopharmaceutical company listed on Nasdaq (RGLS) that specializes in the discovery and development of medicines aimed at microRNAs [3]. - The company has developed a robust pipeline supported by a strong intellectual property portfolio in the microRNA sector [3]. - Regulus is headquartered in San Diego, California [3]. Event Participation - Jay Hagan, the CEO of Regulus, will present at the summit on April 9th, 2025, at 11:32 a.m. ET [1]. - The live event and its replay will be accessible through the investor relations section of the company's website for 90 days post-presentation [2].

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]