Welcome to our dedicated page for Proqr Therapeuti news (Ticker: PRQR), a resource for investors and traders seeking the latest updates and insights on Proqr Therapeuti stock.
ProQR Therapeutics N.V. (Nasdaq: PRQR) generates a steady flow of news as a clinical-stage biopharmaceutical company advancing RNA editing therapies. Its updates center on the Axiomer RNA base editing platform and a pipeline of editing oligonucleotides designed to correct or modulate disease-related targets at the RNA level. News coverage on this page reflects company announcements, clinical milestones, financial updates, and scientific presentations related to its programs in liver, CNS, and other genetically defined diseases.
Investors and followers of PRQR can expect news about the progress of AX-0810, ProQR’s lead investigational RNA editing oligonucleotide targeting NTCP for cholestatic diseases. Recent releases describe the submission and authorization of a Clinical Trial Application, the initiation of a Phase 1 first-in-human study in healthy volunteers in the Netherlands, and early safety and pharmacokinetic observations from the first cohort. Updates also outline plans for target engagement readouts and the future inclusion of a patient cohort in the same trial.
News items also highlight earlier-stage pipeline programs such as AX-2402 for Rett syndrome (MECP2 R270X), AX-2911 for metabolic liver disease including MASH (PNPLA3 I148M), and AX-1412 for cardiovascular disease (B4GALT1). ProQR reports non-clinical proof-of-concept data, development candidate selections, and functional results in disease-relevant models. Financial press releases provide quarterly operating and financial results, cash and cash equivalents, collaboration revenue from Eli Lilly, and comments on cash runway.
Additional PRQR news covers participation in scientific and investor conferences, including ASGCT, the RNA Editing Summit, OTS, TIDES USA, and healthcare investor events. These communications feature presentations on ADAR-mediated RNA editing of NTCP, PNPLA3, and MECP2, as well as platform-focused talks on optimizing editing oligonucleotides for therapeutic use. Bookmark this page to follow ProQR’s disclosed clinical data, pipeline milestones, collaboration developments, and financial reporting as they are released through press announcements and SEC-furnished materials.
ProQR Therapeutics has completed enrollment in the pivotal Phase 2/3 Illuminate trial for sepofarsen, targeting LCA10, with top-line results expected in H1 2022. The company is progressing its pipeline, with the Brighten pediatric trial planned in 2021 and further data from the InSight extension study anticipated in H2 2021. Key financials reveal a decrease in cash equivalents to €75.8 million from €112.0 million in 2019, with a net loss of €46.6 million for 2020. The company strengthened its advisory board and secured a strategic financing deal, providing operational flexibility.
ProQR Therapeutics (Nasdaq: PRQR) announces an Expert Perspectives call on February 22, 2021, at 12:00 PM EST. Led by CMO Aniz Girach and Dr. Paul Yang, the session will discuss key vision measures in Usher Syndrome and non-syndromic Retinitis Pigmentosa, focusing on the Phase 1/2 Stellar trial of QR-421a. QR-421a is a first-in-class RNA therapy targeting vision loss caused by mutations in the USH2A gene. The call will include a Q&A session and an archived presentation will be available for 30 days post-event.
ProQR Therapeutics (Nasdaq: PRQR) announced the completion of patient enrollment in its Phase 2/3 Illuminate trial for sepofarsen, an investigational RNA therapy targeting Leber Congenital Amaurosis 10 (LCA10) caused by a mutation in the CEP290 gene. This marks a significant milestone, with top-line results expected in H1 2022. The study aims to seek marketing approval, as there are currently no approved treatments for LCA10. Sepofarsen has received orphan drug and fast-track designations, highlighting its potential to address a critical unmet medical need.
ProQR Therapeutics (Nasdaq: PRQR) announces participation in the HC Wainwright Virtual BIOCONNECT 2021 Conference, scheduled for January 11, 2021. The company, focused on developing RNA therapies for inherited retinal diseases, aims to transform lives through its proprietary RNA repair platform. A webcast of the presentation will be available on the ProQR website under the 'Investors & Media' section. ProQR emphasizes its commitment to addressing severe genetic rare diseases such as Leber congenital amaurosis 10 and Usher syndrome.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced that its management will participate in a fireside chat at the 3rd Annual Evercore ISI Virtual HealthCONx Conference. The event is scheduled for December 2, 2020, at 3:55 PM EST and will be webcast live. Following the presentation, an archived version will be accessible for 30 days on ProQR's website. ProQR focuses on developing RNA therapies for inherited retinal diseases utilizing its proprietary RNA repair platform.
ProQR Therapeutics reported Q3 2020 results, emphasizing clinical trial advancements and financial stability. The Illuminate Phase 2/3 trial of sepofarsen is projected to complete enrollment in Q1 2021. Data from the QR-421a and QR-1123 trials is expected in 2021, with the QR-504a study on Fuchs to begin in H1 2021. Financially, cash reserves stand at €88.8 million, with a net loss of €13.2 million for the quarter. The company’s runway extends into 2023 due to strategic financing agreements.
ProQR Therapeutics (Nasdaq: PRQR) announced virtual presentations at the American Academy of Ophthalmology Annual Meeting from November 13-15, 2020. Findings will include Phase 1b/2 trial results of sepofarsen for Leber congenital amaurosis 10 (LCA10), presented by Stephen R. Russell, MD. Another presentation by Allen Ho, MD, will focus on patient responses assessed via full-field stimulus testing. Sepofarsen is a pivotal RNA therapy targeting the p.Cys998X mutation in the CEP290 gene, designed to restore normal protein function and has received several designations from health authorities.
ProQR Therapeutics has appointed Naveed Shams, MD, PhD as its new Chief Scientific Officer, succeeding David Rodman, MD. Dr. Shams, with over 20 years of experience in ophthalmology, previously held prominent roles at Santen and Genentech, where he contributed to key drug developments like Lucentis. His expertise will focus on enhancing ProQR's research and development of innovative RNA therapies for inherited retinal diseases. David Rodman will assist during the transition in an advisory capacity.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced virtual presentations at key ophthalmology forums, focusing on advancements in treatments for inherited retinal diseases. Highlights include:
- Ophthalmology Futures Retina Forum: Presentation on managing inherited retinal diseases by CMO Aniz Girach on September 30, 2020.
- Euretina Congress: Results from Phase 1b/2 trial of sepofarsen in Leber congenital amaurosis (LCA10) presented by Stephen R. Russell, available from October 2, 2020.
- AAOpt Meeting: Discussion on early genetic diagnosis and patient referrals in retinal diseases on October 7, 2020.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced participation in several upcoming investor conferences aimed at discussing its focus on RNA medicines for inherited retinal diseases. Key events include the Citi's 15th Annual BioPharma Virtual Conference on September 10, the H.C. Wainwright 22nd Annual Conference on September 15, the Cantor Virtual Global Healthcare Conference on September 16, and the Chardan Virtual 4th Annual Genetic Medicines Conference on October 6. Details of the presentations can be found on the company's website, with archived webcasts available post-presentation.
FAQ
What is the current stock price of Proqr Therapeuti (PRQR)?
What is the market cap of Proqr Therapeuti (PRQR)?
