Overview of the Company

Regulus Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing drugs targeting microRNAs to treat diseases with significant unmet medical needs. The company was formed in 2007 as a joint venture between Alnylam Pharmaceuticals and Ionis Pharmaceuticals.

Regulus is currently focused on developing treatments for orphan kidney diseases, where microRNA drivers are implicated. The company’s lead product candidate, RGLS8429, is an anti-miR oligonucleotide drug targeting miR-17 for the treatment of autosomal dominant polycystic kidney disease (ADPKD). RGLS8429 is currently in Phase 1b clinical trials.

Financial Performance

Revenue and Profit Trends

As Regulus is still in the clinical development stage, the company does not currently generate any revenue. The company has incurred significant operating losses since inception. Net losses were $9.0 million for Q1 2024, compared to $7.7 million for Q1 2023. The increased net loss was primarily driven by increased research and development expenses.

Cash Position

Regulus raised approximately $93.9 million through a private placement financing in March 2024, resulting in total cash, cash equivalents and investments of $107.7 million as of March 31, 2024. This is expected to fund planned operations and expenditures for at least the next 12 months.

R&D Program Analysis

RGLS8429 for ADPKD

RGLS8429 is currently in Phase 1b clinical trials evaluating safety, tolerability, pharmacokinetics and efficacy in ADPKD patients across multiple ascending doses. Early data from the first two patient cohorts showed increases in polycystin biomarker levels and encouraging reductions in total kidney volume in patients with the highest polycystin increases. The third patient cohort is fully enrolled with data expected mid-2024. The fourth cohort initiated in May 2024.

A 27-week chronic mouse study showed no toxicity up to the maximum dose tested. The FDA also confirmed the potential for an accelerated approval pathway based on a single pivotal Phase 2 trial.

Preclinical Pipeline

Regulus continues to advance and expand its internal discovery pipeline to identify new microRNA-targeted therapies for diseases of high unmet need where delivery to target tissues can be effectively achieved.

Strengths and Weaknesses

Strengths

• Promising early stage clinical data for lead program RGLS8429
• Potential for accelerated approval pathway for RGLS8429
• Strong cash position to advance development programs
• Focus on targets backed by strong scientific rationale

Weaknesses

• No approved products and still in early stages of clinical development
• History of operating losses and need for additional financings
• Clinical development risks still remain
  

Future Outlook

Key upcoming milestones for Regulus include:

• RGLS8429 Phase 1b topline data from cohorts 3 and 4 in mid-2024
• Decision on Phase 2 initiation for RGLS8429 by end of 2024
• Continued advancement of preclinical pipeline programs

The company’s clinical progress and data readouts for RGLS8429 will be the key drivers of value over the next 12-18 months. Successful outcomes could enable initiation of a pivotal Phase 2 trial as the next step towards potential approval.

Regulus is well-financed currently but will likely need to raise additional capital if all programs advance as currently planned. The company’s net losses will also continue increasing driven by expanded clinical development activities.

Overall the future outlook will be determined by clinical execution on the RGLS8429 program. The encouraging early stage data provides a promising start as Regulus works to establish microRNA therapies as a new therapeutic modality.