Regulus reports progress in ADPKD trial and FDA talks

SAN DIEGO - Regulus Therapeutics Inc . (NASDAQ:RGLS), a biopharmaceutical company currently trading at $1.17 per share, today announced significant clinical and regulatory advancements in its Autosomal Dominant Polycystic Kidney Disease (ADPKD) program. According to InvestingPro data, while the company maintains a strong liquidity position with a current ratio of 14.14, it faces near-term challenges with accelerated cash burn rates. The company reported positive interim results from the fourth cohort of its Phase 1b Multiple Ascending Dose study for the investigational drug farabursen, and a successful End-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA).

The Phase 1b trial, which is placebo-controlled and double-blind, assesses the safety, tolerability, pharmacokinetics, and pharmacodynamics of farabursen in adult patients with ADPKD. In the latest cohort, 26 subjects received a 300 mg dose of farabursen every other week for three months. Interim efficacy data from the first 14 subjects showed a mechanistic dose response and a reduction in the growth rate of height-adjusted total kidney volume (htTKV), a key measure of disease progression. The complete safety data from all subjects indicated that the drug was well tolerated.

In December 2024, Regulus aligned with the FDA on the components of a Phase 3 trial design, which could potentially lead to Accelerated Approval. This pivotal study will include a single active dose and placebo, administered every other week with a 2:1 randomization scheme, and will focus on a 12-month htTKV endpoint for Accelerated Approval and a 24-month estimated glomerular filtration rate (eGFR) endpoint for Full Approval.

Dr. Preston Klassen, President and Head of Research & Development at Regulus, highlighted the trial results as extending the understanding of the potential benefits of their approach in treating ADPKD, a condition with a high unmet medical need. Dr. Alan Yu from the University of Kansas Medical (TASE:PMCN) Center also noted the promising results from Cohort 4, validating the impact of farabursen on polycystin levels and kidney volume growth rate.

CEO Jay Hagan expressed encouragement by the FDA’s feedback and the interim results, reinforcing the company’s conviction in farabursen’s potential for ADPKD treatment. Regulus plans to advance farabursen into a pivotal study later in the year.

ADPKD is one of the most common human monogenic disorders and a leading cause of end-stage renal disease. Farabursen, which targets miR-17 with preferential kidney exposure, has shown improvements in kidney function, size, and disease severity in preclinical models. The drug has been well-tolerated with no serious adverse events reported across studies.

This article is based on a press release statement from Regulus Therapeutics Inc. With a market capitalization of $76.64 million and a beta of 1.6, the company’s stock has shown significant volatility. InvestingPro subscribers can access 10+ additional ProTips and comprehensive financial metrics to better evaluate this emerging biotech opportunity. Get detailed insights into the company’s financial health score of 2.01 (FAIR) and expert analysis to inform your investment decisions.

In other recent news, Regulus Therapeutics has been the subject of significant analyst attention. Jones Trading has affirmed a Buy rating on the company, anticipating pivotal study and fixed-dose data release in 2025. The firm maintains a steady price target of $8.00 on Regulus stock, highlighting the ongoing high unmet need in the autosomal dominant polycystic kidney disease (ADPKD) market. Other analyst firms, such as H.C. Wainwright and Canaccord Genuity, have also maintained Buy ratings on Regulus Therapeutics, indicating confidence in the company’s ongoing projects.

Regulus Therapeutics has recently secured an exclusive license from The University of Texas System for patents and technology aimed at treating ADPKD. This agreement includes initial payments, milestone payments for clinical, regulatory, and commercial achievements, as well as tiered royalties on net sales of licensed products. The company’s clinical candidate RGLS8429, developed for ADPKD, is on schedule for an End of Phase 1 meeting with the FDA by the end of 2024, with plans to start a pivotal study in 2025.

Among other recent developments, Regulus Therapeutics has disclosed additional exploratory results from its Phase 1b study of RGLS8429, which consistently impacted the height-adjusted total kidney volume across high-risk classes. The company also anticipates releasing fixed-dose Phase 1 data in early 2025. Financially, Regulus is reported to have cash reserves of $87.3 million, which is expected to fund operations into the first half of 2026.

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