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ProQR Therapeutics N.V. reports developments as a clinical-stage biopharmaceutical company built around its proprietary Axiomer™ RNA editing platform. The technology uses ADAR-mediated RNA editing to make single-nucleotide changes in RNA, with pipeline updates centered on AX-0810 and additional programs across rare and prevalent diseases with unmet need.
Recurring company news includes scientific conference presentations, investor and analyst events, clinical-program updates, development-candidate selections, AI-enabled discovery initiatives, and collaborations such as Ginkgo Bioworks and Eli Lilly. ProQR also reports annual operating and financial results, annual general meeting materials, board composition changes, and other governance matters tied to its Nasdaq-listed public-company profile.
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.
ProQR Therapeutics (Nasdaq: PRQR) reported positive preliminary data from the InSight extension study of sepofarsen for LCA10, reinforcing confidence in the Phase 2/3 Illuminate trial. A strategic convertible debt financing extends the company’s runway into 2023. ProQR is actively enrolling patients across three clinical-stage RNA therapies, with a fourth program set for clinical testing. Financially, cash and cash equivalents stood at €87.1 million, while net cash used in operating activities was €10.5 million. The net loss for Q2 2020 was €4.1 million, down from €11.7 million in the prior year.