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Regulus Therapeutics Reports First Quarter 2025 Financial Results and Recent Updates

In a significant development, Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical firm dedicated to innovative microRNA-targeting therapies, announced its financial results for Q1 2025 alongside noteworthy corporate updates on May 8, 2025. The highlight of this announcement was the agreement for acquisition by Novartis, valued at $1.7 billion, contingent on the achievement of specific regulatory milestones.

Acquisition Details

Regulus has entered into a merger agreement with Novartis, entering a new chapter for both companies. Under the terms of the agreement, Novartis will acquire Regulus for an initial payment of $7.00 per share in cash, supplemented by a contingent value right (CVR) potentially adding another $7.00 per share if specific regulatory achievements are met. This deal, approved unanimously by the boards of both companies, is set to finalize in the latter half of 2025, pending standard closing conditions such as shareholder approval and necessary regulatory clearances. Until then, Regulus will function as an independent entity.

CEO Jay Hagan expressed optimism regarding this agreement, stating that Novartis's established global development capabilities could significantly advance the treatment options for patients suffering from autosomal dominant polycystic kidney disease (ADPKD), the disease target of their lead product, farabursen.

Progress with Farabursen

Farabursen (RGLS8429) is a groundbreaking oligonucleotide therapy focusing on ADPKD, aimed at inhibiting microRNA-17 (miR-17) primarily affecting kidney function. Regulus has reported promising topline data from the fourth cohort of a Phase 1b multiple-ascending dose (MAD) study, which evaluated farabursen’s effects on kidney volume and function. The results showed a notable pause in kidney volume growth after a mere short treatment duration, indicating the drug's potential efficacy in halting disease progression.

Notably, the fourth cohort comprised 26 patients who received the 300 mg dose bi-weekly over three months. Similar responses were observed across cohorts, reinforcing the appropriateness of this fixed dosage for the upcoming pivotal Phase 3 trial aimed for initiation in Q3 2025. This pivotal trial is crucial as it will impact Regulus's regulatory approval pathways moving forward.

In conjunction with these updates, the company revealed strong safety profiles from completed studies, further enhancing its outlook on farabursen as a potential treatment for the 160,000 diagnosed ADPKD patients in the U.S. alone, and millions worldwide.

Financial Snapshot

As of March 31, 2025, Regulus reported cash, cash equivalents, and short-term investments amounting to $65.4 million, projecting a cash runway extending well into early 2026. The company's research and development expenses rose slightly to $6.8 million during the first quarter compared to $6.0 million from the same period in 2024, reflecting ongoing advancements in their clinical pipeline. General and administrative expenses similarly increased to $3.7 million from $2.8 million in the prior year.

Regulus experienced a net loss of $9.6 million in Q1 2025, illustrating the financial challenges biopharmaceutical firms face during the developmental phases of drug discovery and trials. Despite this, the essential investments in research and clinical trials are foundational for potential future success.

Outlook

As Regulus Therapeutics positions itself for this significant merger with Novartis, both firms are poised to enhance treatment options for ADPKD patients. The upcoming Phase 3 trial will be pivotal in determining farabursen's efficacy and bolstering the company's capabilities in the competitive landscape of biotechnology. As the September 2025 target for the trial initiation approaches, stakeholders will keep a close watch on how these developments unfold in the biopharmaceutical sector, contributing to the broader goal of improving patient outcomes for ADPKD and other microRNA-related disorders.

With the merger set to potentially reshape the future of Regulus and augment Novartis's portfolio, both companies are aimed at leveraging their strengths to bring innovative therapies to market and address the unmet needs of patients worldwide.