Regulus Therapeutics (RGLS) Canaccord Genuity 44th Annual Growth Conference & Private Company Showcase 2024 summary

Program and clinical development update

• Currently conducting a Phase 1b multiple ascending dose study in ADPKD, with three weight-based cohorts completed and a fourth fixed-dose cohort enrolling; primary focus is safety, tolerability, and biomarker engagement.

• Cohort data from the ongoing study will be available early next year, with study wrap-up in the first half of the year; an end-of-Phase 1 FDA meeting is planned for late this year to align on the pivotal study protocol for accelerated approval.

• Preparations for the pivotal study are underway, including drug manufacturing and recent PIPE financing extending cash runway into mid-2026.

Market and disease landscape

• ADPKD is a prevalent orphan kidney disorder with about 160,000 diagnosed U.S. patients; only one approved drug, tolvaptan, has limited market penetration due to safety concerns.

• The market opportunity is significant, with high unmet need and regulatory incentives for orphan indications.

Mechanism of action and preclinical rationale

• Disease is driven by upregulation of microRNA miR-17, causing widespread gene dysregulation; blocking miR-17 normalizes gene networks.

• Preclinical models show that anti-miR-17 therapy reverses gene expression changes and significantly ameliorates disease progression in multiple PKD models.