Regulus Therapeutics (RGLS) Oppenheimer 35th Annual Healthcare Life Sciences Conference (Virtual) 2025 summary

Key clinical updates and regulatory alignment

• Positive data from the fourth cohort of farabursen, a novel oligonucleotide for ADPKD, with FDA alignment on a single pivotal phase II for accelerated approval.

• Transitioned to a 300 mg fixed dose to facilitate future product formats and regulatory submissions.

• Safety and tolerability profile remains strong, with no serious adverse events or discontinuations in the latest cohort.

• Efficacy assessments show consistent biomarker and kidney volume improvements, supporting dose selection for phase III.

• Large safety margins confirmed by chronic toxicology studies in two species, supporting long-term dosing.

Phase III study design and efficacy outlook

• Final protocol for phase III is being finalized, with operational planning underway and enrollment expected within six months.

• Study powered to demonstrate at least a 50% reduction in kidney volume growth rate and 33% reduction in GFR decline over two years.

• Early data suggest the potential to nearly halt kidney progression over four months, indicating high likelihood of meeting primary endpoints.

• FDA acceptance of kidney volume as a surrogate endpoint underpins accelerated approval strategy.

• Additional Cohort IV data to be presented in March will further clarify treatment effect and de-risk phase III.

Market landscape and commercialization strategy

• Tolvaptan, the only approved therapy, has limited uptake due to tolerability and safety issues, with less than 7% of the addressable U.S. population treated.

• Market research indicates high intent to use a therapy with tolvaptan-like efficacy but better safety and tolerability.

• U.S. and Europe are primary focus regions, with plans to engage EMA and Japanese regulators for global trial execution.

• Commercialization outside the U.S. may involve partnerships to maximize reach and shareholder value.

• Genericization of tolvaptan is not expected to be a major barrier for new entrants with superior profiles.