ProQR Therapeutics (PRQR) Evercore ISI 8th Annual HealthCONx Conference summary

Company overview and technology

• Focuses on RNA editing medicines to treat genetic and common diseases by modifying messenger RNA.

• Holds foundational IP for its RNA editing technology, invented about 10 years ago.

• Entered a $4 billion partnership with Eli Lilly in 2021, covering up to 15 targets, with $125 million upfront and up to $250 million in milestones per target plus royalties.

• Maintains a wholly owned pipeline targeting liver and CNS indications, including rare and common diseases.

Lead program: AX-0810 for cholestatic diseases

• AX-0810 targets NTCP to modulate bile acid uptake for diseases like PSC and BA.

• Phase 1 trial in healthy volunteers is starting, with 33 subjects across three cohorts, randomized, placebo-controlled, and double-blinded.

• Initial safety and PK data expected by year-end; full PD and biomarker data in the first half of next year.

• Phase 1b in PSC patients planned for the second half of next year, with results before year-end.

• Indication selection between PSC and BA for phase 2 will be announced with phase 1 results.

Rationale and trial design

• Chose NTCP as the initial target due to measurable engagement in healthy volunteers and high unmet need in PSC/BA.

• Trial measures bile acid concentration, conjugated/unconjugated ratio, and response to TUDCA challenge.

• Success defined as at least a twofold increase in serum bile acid, shift toward conjugated bile acids, and improved clearance post-TUDCA.

• Editing percentage correlates linearly with bile acid increase; 5% editing yields twofold, 10% yields fourfold increase.