Regulus Therapeutics (RGLS) Leerink’s Global Healthcare Conference 2025 summary

Recent accomplishments and 2025 goals

• Completed phase I-B trial of oligonucleotide Farabursen (RGLS8429) targeting miR-17, with plans to initiate a pivotal phase III study following a successful end-of-phase I FDA meeting.

• 2025 goal is to start the pivotal phase III study.

Regulatory and clinical pathway

• FDA supports total kidney volume as a surrogate endpoint for accelerated approval, with GFR as the endpoint for full approval.

• Accelerated approval requires volume data; full approval requires GFR data, with both endpoints being tracked in the phase III design.

• European regulatory engagement is planned, with a focus on full approval for reimbursement.

Clinical trial design and data

• Phase III is a two-year, global study with 325-350 patients, using MRI to measure kidney volume at baseline, 6, and 12 months, and GFR every eight weeks.

• Interim analyses by an independent committee will assess sample size adequacy at key points.

• Dose selection is based on urinary polycystin response, with 3 mg/kg or 300 mg fixed dose showing optimal effect.

• Enrollment targets moderate to severe ADPKD (Mayo Class 1C, 1D, 1E) with broad GFR criteria.