Research analysts covering 0PQ.
Recent press releases and 8-K filings for 0PQ.
ProQR Therapeutics Provides Update on RNA Editing Pipeline and Eli Lilly Partnership
0PQ
New Projects/Investments
Product Launch
- ProQR Therapeutics is a biopharmaceutical company based in the Netherlands, focused on developing RNA editing medicines for genetic and common diseases, with shares traded on NASDAQ.
- The company has a $4 billion collaboration with Eli Lilly for up to 15 RNA editing targets, which included a $125 million upfront payment and potential milestone payments of up to $250 million per target, plus royalties on approved products.
- ProQR's wholly-owned pipeline includes AX-0810 for cholestatic diseases (PSC and biliary atresia), with a Phase 1 study in healthy volunteers starting soon.
- For the AX-0810 program, initial safety and PK data are expected around the end of 2025, and PD data (target engagement and biomarkers) in the first half of 2026. Success is defined by at least a twofold increase in serum bile acid.
- ProQR is also advancing a program for Rett syndrome, aiming to select a clinical candidate shortly, noting its differentiation from gene therapy by avoiding MECP2 overexpression.
Dec 4, 2025, 1:20 PM
ProQR Therapeutics Provides Update on RNA Editing Pipeline and Eli Lilly Partnership
0PQ
New Projects/Investments
- ProQR Therapeutics, a biopharmaceutical company focused on RNA editing, has a $4 billion partnership with Eli Lilly for up to 15 targets, which included $125 million in upfront payments.
- Its lead wholly-owned program, AX-0810 for cholestatic diseases (PSC and BA), has an open CTA and is initiating a clinical study in healthy volunteers.
- First human clinical data (PD data) for AX-0810 is anticipated in the first half of next year, following initial safety and PK data expected by the end of this year.
- ProQR plans a Phase 1B study in PSC subjects in the second half of next year, with results expected before the end of next year, and believes there is potential for accelerated approval for AX-0810.
Dec 4, 2025, 1:20 PM
ProQR Therapeutics Initiates AX-0810 Phase I Trial and Provides Data Timelines
0PQ
New Projects/Investments
Product Launch
- ProQR Therapeutics announced that its lead program, AX-0810, has received CTA authorization in Europe and is starting a Phase I trial in healthy volunteers to assess safety, tolerability, and pharmacokinetics.
- The company expects to release initial safety, tolerability, and PK data for AX-0810 towards the end of 2025, with target engagement data anticipated in the first half of 2026.
- AX-0810, based on the proprietary Axiomer RNA editing platform, targets NTCP for cholestatic diseases and represents the first RNA editing oligonucleotide designed to modulate wild-type protein to enter the clinic.
- ProQR highlighted its $3.9 billion collaboration with Eli Lilly focused on Axiomer RNA editing and stated that it is funded into mid-2027.
Nov 3, 2025, 3:00 PM
ProQR Provides Update on AX-0810 Clinical Trial and Corporate Outlook
0PQ
New Projects/Investments
Guidance Update
- ProQR's AX-0810 program, a first-in-class RNA editing therapy targeting NTCP for cholestatic diseases, is entering a first-in-human (FIH) trial with the Clinical Trial Authorization (CTA) authorized.
- Safety, tolerability, and PK data for Cohort 1 of the AX-0810 FIH trial are expected by the end of 2025, with full safety, tolerability, PK, and target engagement data for all cohorts anticipated in H1 2026.
- The company reported €119.8 million in cash and cash equivalents as of end of Q2 2025, providing a financial runway into mid-2027.
- ProQR is also advancing AX-2402 for Rett syndrome and has earlier programs including AX-1412 for CVD and AX-2911 for MASH.
Nov 3, 2025, 3:00 PM
ProQR Provides Updates on RNA Editing Programs, Including AATD Clinical Data and AX-0810 Phase 1 Initiation
0PQ
New Projects/Investments
- ProQR presented clinical data for its Alpha-1 Antitrypsin Deficiency (AATD) program, showing patients achieved MZ-like protein levels with 12 micromolar of total protein, increasing to 13 micromolar, and a shift from 0% to 44% M protein after a single dose in the lowest 200 mg cohort.
- The AATD program also demonstrated nearly 11 micromolar of M protein and over 20 micromolar of endogenous protein production during an acute phase event, with sustained editing for over two months from a single dose.
- ProQR announced clearance to initiate a Phase One trial for AX-0810, its lead program for cholestatic diseases, which is designed to edit NTCP and block bile acid transport into the liver.
- The company is also advancing its pipeline in CNS, having shown up to 60% editing efficiency in non-human primates, and is targeting PNPLA3 for hepatic diseases, with a research day scheduled for October 29 to discuss these developments.
Oct 21, 2025, 5:00 PM
WaveLife, ProQR, and Airena Provide Updates on ADAR RNA Editing Programs
0PQ
New Projects/Investments
Product Launch
- WaveLife reported clinical data for its alpha-one antitrypsin deficiency (AATD) program, demonstrating 12-13 micromolar total protein and 44-65% M protein after single/multi-dose, with editing sustained for over two months.
- ProQR received clearance to initiate a Phase 1 trial for AX-810, targeting cholestatic diseases, with an event scheduled for November 3 to detail the trial design. Preclinical data indicated 15% editing led to a two-fold increase in serum bile acids.
- Airena presented preclinical data for its AATD program, demonstrating high mAAT (39 micromolar) and total AAT (63 micromolar) in mouse models, and durability (half-life over 30 days) in non-human primates, with a CTA filing expected by year-end.
- The companies are expanding their ADAR pipelines; WaveLife will discuss its PMVLA-three program for liver diseases on October 29, and ProQR highlighted its CNS pipeline, achieving up to 60% editing efficiency in non-human primates.
Oct 21, 2025, 5:00 PM
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