Regulus Therapeutics (RGLS) 7th Annual Evercore ISI HealthCONx Healthcare Conference summary

Company background and technology

• Formed as a joint venture in 2009, exclusively licensed to develop oligonucleotides targeting microRNA, with a focus now on kidney distribution motifs.

• No opt-in rights for original partners; company pays low single-digit royalties on commercialized products.

Disease focus and current treatment landscape

• Targeting autosomal dominant polycystic kidney disease (ADPKD) by inhibiting miR-17, which plays a central role in disease initiation and progression.

• Current standard of care, tolvaptan, slows kidney growth and GFR decline but has significant tolerability issues and black box liver toxicity warning.

• Tolvaptan is used in less than 7% of high-risk patients due to side effects and dosing challenges.

Clinical development and data

• Phase 1B study with weight-based and fixed-dose cohorts showed dose-dependent biomarker response and reductions in kidney volume at higher doses.

• Fixed-dose cohort (300 mg) aims to match or exceed effective weight-based dosing, with safety and tolerability as key endpoints.

• Reductions in kidney volume observed in mid and high dose groups, a result not seen with current therapies.