ProQR Therapeutics (PRQR) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Platform and technology development

• Developed Axiomer, an RNA editing platform using endogenous ADAR to edit mRNA in vivo, enabling precise genetic sequence changes for therapeutic purposes.

• Built a robust IP portfolio with over twelve platform-oriented patents, surviving opposition and reflecting leadership in RNA editing technology.

• Achieved drug-like properties in molecules, allowing infrequent dosing and high editing in targeted organs, supporting a broad pipeline for rare and common diseases.

• Partnerships, including with Eli Lilly, expand the platform's reach to multiple targets and indications.

Pipeline programs and clinical plans

• Lead program targets cholestatic diseases by introducing a variant in NTCP to reduce bile acid uptake, aiming to prevent liver inflammation and failure.

• Presented in vivo data in non-human primates showing meaningful biomarker changes, with IND-enabling studies ongoing and clinical entry planned late 2024 or early 2025.

• Second program, AX-1412, targets cardiovascular disease by introducing a protective variant, with preclinical and translational data expected later this year.

• Clinical studies will begin in healthy volunteers, focusing on proof of mechanism and biomarker validation, with rapid data readout expected.

Biomarker strategy and clinical endpoints

• Total bile acids in serum serve as a key biomarker, with a twofold increase indicating reduced inflammation and fibrosis, based on prior NTCP-targeting trials.

• Updated clinical candidate data and trial design will be announced together, including delivery modality selection (LNP or GalNAc).

• Decision on delivery modality may vary by target, balancing exposure profiles and durability.

• Additional biomarkers, such as bile acid profiles, are under consideration for a comprehensive clinical assessment.