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Dec 31, 2024
Regulus Therapeutics Q4 2024 Earnings Report
Regulus Therapeutics reported its financial results for the fourth quarter and year ended December 31, 2024, highlighting positive topline results from its Phase 1b MAD clinical trial for farabursen and alignment with the FDA on key components of a Phase 3 pivotal trial.
Key Takeaways
Regulus Therapeutics reported a net loss of $12.8 million for the fourth quarter of 2024, or $0.20 per share. The company ended the year with $75.8 million in cash, cash equivalents, and short-term investments, with a projected cash runway into early 2026. Key updates include positive interim data from the Phase 1b MAD trial of farabursen for ADPKD and agreement with the FDA on the design of a Phase 3 pivotal trial.
Positive interim topline results from the fourth cohort of the Phase 1b MAD clinical trial of farabursen for ADPKD were announced, showing encouraging safety, tolerability, and mechanistic dose response.
The company reached alignment with the FDA on the key components of a Phase 3 single pivotal trial for farabursen, including a 12-month htTKV endpoint for potential Accelerated Approval.
Net loss for the fourth quarter of 2024 was $12.8 million, or $0.20 per share.
Cash, cash equivalents, and short-term investments totaled $75.8 million as of December 31, 2024, with a cash runway expected into early 2026.
Regulus Therapeutics
Regulus Therapeutics
Forward Guidance
Regulus Therapeutics anticipates continued progress in the development of farabursen for ADPKD, with an expected cash runway into early 2026. The company plans to share topline data from all 26 subjects in cohort 4 of the Phase 1b MAD trial in the coming weeks.
Positive Outlook
- Positive interim topline results from the fourth cohort of the Phase 1b MAD trial of farabursen for ADPKD, showing encouraging safety, tolerability, and mechanistic dose response.
- Agreement with the FDA on key components of a Phase 3 single pivotal trial for farabursen, including a 12-month htTKV endpoint for potential Accelerated Approval.
- The proposed pivotal trial includes an interim analysis to enable Accelerated Approval, which could expedite farabursen's development.
- Farabursen continues to demonstrate a well-tolerated profile similar to previous cohorts.
- The company expects its cash runway to extend into early 2026.
Challenges Ahead
- The approach to discover and develop drugs is novel and may never lead to marketable products.
- Preliminary or topline results are based on a preliminary analysis and may change following a more comprehensive review.
- An Accelerated Approval pathway designation may not be received, or if received, may not lead to a faster development or approval process.
- Clinical development strategy may change, and preclinical and clinical studies may not be successful.
- Additional data may be negative, and there are risks related to the company's ability to successfully secure and deploy capital.