ProQR Therapeutics (PRQR) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Key announcements and clinical updates

• Initiated clinical dosing of AX-0811, a more potent RNA editing oligo, marking the second program in the clinic, with two more to follow soon.

• Five clinical trial readouts are expected across four programs within the next 12 months, signaling a pivotal year for the pipeline.

• An investor event at the end of September will outline expectations for AX-0811, review AX-0810 data, and set targets for three key bile acid endpoints.

• Remaining AX-0810 data, including cohort 3 and 12-week follow-up, will be presented at a scientific conference later this year.

Scientific and technological advancements

• Dose-dependent, concordant RNA editing responses have been observed across three human endpoints, validating preclinical models.

• AI-driven high-throughput screening has accelerated the development of high-editing oligonucleotides, enabling rapid optimization for new targets.

• The AI platform, combined with roboticized lab testing, allows for efficient iteration and optimization across multiple variables, including editing efficiency, stability, manufacturability, and safety.

• Editing efficiency for AX-0811 is projected at 40%-65%, a significant improvement over earlier molecules.

Pipeline and future plans

• AX-0811 and AX-0810 target NTCP for cholestatic diseases, with AX-0811 expected to show higher editing and faster biomarker effects.

• AX-0422 for MPS I (Hurler syndrome) will enter the clinic soon, with initial data expected in the first half of next year; both systemic and CNS delivery are planned.

• AX-2911 targets PNPLA3 for lean MASH, with first clinical data also expected in the first half of next year.

• The next 12 months will see five clinical data readouts across four molecules, including proof-of-concept data for several programs.