Regulus Therapeutics (RGLS) Piper Sandler 36th Annual Healthcare Conference summary

Key presentations and announcements

• Highlighted recent Nobel Prize for Medicine awarded for microRNA discovery, underscoring the scientific foundation of the lead program.

• Reported positive phase I data for RGLS8429 in autosomal dominant polycystic kidney disease (ADPKD), showing safety and tolerability at all tested doses.

• Noted statistically significant biological activity and reduction in kidney volume at higher dose levels, with final cohort data expected late Q1 or early Q2 next year.

• Outlined plans to meet with the FDA before year-end to align on pivotal study design and readiness for phase II/III trials.

• Emphasized a $100 million financing completed to support phase II readiness and CMC activities.

Industry and market analysis

• ADPKD affects about 100,000 high-risk patients in the U.S., with current treatment (tolvaptan) limited by tolerability and safety concerns.

• Tolvaptan's uptake remains below 7% of the addressable population, despite $1.3 billion in annual sales and broad commercial coverage.

• Market research with nephrologists and payers indicates strong enthusiasm for a safe, well-tolerated alternative with similar efficacy.

• Concentrated prescriber base allows for efficient commercialization with a focused sales force.

• Underdiagnosis suggests the true patient population may be 3–4 times higher than current estimates.

Scientific and clinical insights

• RGLS8429 targets miR-17, a microRNA upregulated in ADPKD, aiming to restore gene networks and increase polycystin production.

• Preclinical and clinical data show preferential kidney distribution, dose-dependent target engagement, and pleiotropic effects on disease pathways.

• Phase I results demonstrate dose-dependent increases in polycystin and early evidence of arresting kidney cyst expansion.

• Safety profile remains favorable, with no significant adverse events or safety concerns observed.

• Next steps include reporting cohort four data and finalizing pivotal trial design with the FDA for accelerated approval based on total kidney volume.