ProQR Therapeutics used its slot at the 12th Annual Cantor Fitzgerald Global Healthcare Conference to map out a rapid‑track development plan for its RNA‑editing pipeline. The company highlighted a series of clinical milestones slated through 2025 and beyond.
In June 2024, ProQR reported positive direct engagement data for its second‑generation candidate AX‑0810, which targets the NTCP transporter in cholestatic diseases such as biliary atresia. The firm expects AX‑0810 to achieve 10‑15% editing at the two lowest clinical doses (3 mg/kg and 6 mg/kg). Its successor, AX‑0811, is projected to reach 40‑65% editing – a four‑ to six‑fold improvement.
ProQR outlined a dense readout schedule for the next year, with five clinical trial results across four therapeutic programs. The Phase II biliary atresia study is slated to start in mid‑2025, with a potential interim readout in mid‑2028. Initial data for AX‑0422 (targeting IDUA in MPS I/Hurler syndrome) and AX‑2911 (targeting PNPLA3 in lean MASH) are expected in the first half of 2025, alongside the first data from an investigator‑initiated biliary atresia trial.
Before the J.P. Morgan Healthcare Conference in January 2025, ProQR plans to release two pharmacodynamic cohorts for AX‑0811. An investor event at the end of September 2024 will outline clinical expectations for AX‑0811 and review AX‑0810 data, with remaining AX‑0810 results – including cohort 3 (9 mg/kg) and 12‑week follow‑up – slated for presentation at a scientific conference later in the year.
The company also discussed dosing strategies for its MPS I program, proposing sub‑cutaneous administration every five to six months and intrathecal dosing every nine to twelve months, compared with weekly intravenous enzyme replacement.
Financially, ProQR’s shares traded around $2.025, down 1.22% on the day, and the firm reported a negative free‑cash‑flow of $57 million over the last twelve months. Analyst price targets range from $3 to $12.
CEO Daniel de Boer emphasized the platform’s consistency, noting that Axiomer RNA editing with ADAR shows dose‑dependent editing across endpoints and that pre‑clinical models have proven predictive of clinical outcomes. He contrasted the editing approach with NTCP inhibitors, arguing that editing preserves normal protein function while removing pathogenic mutations, a safety advantage supported by natural human variants.
ProQR also referenced collaborations with Cantor Fitzgerald, Ginkgo Bioworks, Assembly Bio and Gilead’s bulevirtide peptide, underscoring a broader ecosystem supporting its RNA‑editing technology.
