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ProQR Therapeutics N.V., trading under the symbol PRQR, is a pioneering biotech company headquartered in Leiden, Netherlands, and Cambridge, Massachusetts. The company is at the forefront of developing RNA therapeutics for patients suffering from severe genetic rare diseases. ProQR's core focus is on leveraging a proprietary RNA editing technology platform known as Axiomer™ to create transformative RNA therapies.
The Axiomer™ technology utilizes a natural cellular mechanism called ADAR (Adenosine Deaminase Acting on RNA) to make precise edits at the RNA level, correcting disease-causing mutations, modulating protein expression, or adding new protein functions to treat diseases. This innovative approach has the potential to yield treatments for various genetic disorders such as cystic fibrosis, Leber's congenital amaurosis type 10, dystrophic epidermolysis bullosa, and beyond.
Recently, ProQR announced a notable collaboration with the Rett Syndrome Research Trust (RSRT) to develop editing oligonucleotides (EONs) using Axiomer™ technology to correct mutations in the transcription factor Methyl CpG binding protein 2 (MECP2), which is associated with Rett syndrome. This collaboration could significantly advance therapeutic options for this rare neurodevelopmental disorder.
Furthermore, the company continues to impress with its cutting-edge research and intellectual property milestones. ProQR has robustly defended its patents globally, ensuring a strong IP estate around its Axiomer technology. Notably, the company successfully defended its patents in Japan and Europe, showcasing the innovative and proprietary nature of its RNA editing platform.
ProQR's pipeline is rapidly advancing, with key programs targeting cholestatic diseases and cardiovascular diseases. The AX-0810 program, targeting the NTCP gene, has shown promising preclinical proof of concept, indicating its potential to become a disease-modifying treatment for conditions like primary sclerosing cholangitis (PSC) and biliary atresia (BA). The AX-1412 program aims to tackle cardiovascular diseases by introducing protective gene variants.
The company also reported positive financial results for the year ended December 31, 2023, with a solid cash position of €118.9 million, up from €94.8 million in 2022. ProQR continues to invest in advancing its RNA editing platform and pipeline, with a strategic focus on entering clinical trials in late 2024 or early 2025.
Under the leadership of CEO Daniel A. de Boer, ProQR is well-positioned to continue its mission of changing lives through transformative RNA therapies, backed by strong scientific evidence, a robust IP portfolio, and strategic collaborations, including a fruitful partnership with Eli Lilly. Keep up with the latest updates and developments by visiting their website at www.proqr.com.
ProQR Therapeutics (PRQR) announced encouraging post-hoc analyses from the Illuminate trial of sepofarsen, indicating efficacy when comparing treated and untreated eyes. The company plans to engage with regulators in Q3 regarding these findings. ProQR's strategy now focuses on accelerating its Axiomer® RNA base-editing platform and selected inherited retinal disease programs, along with a 30% workforce reduction to extend its cash runway into 2025. Despite setbacks in achieving the primary endpoint of the Illuminate trial, the results support ongoing development and further regulatory discussions.
On April 4, 2022, ProQR Therapeutics (Nasdaq: PRQR) announced its participation in the 3rd RNA Editing Summit in Boston, MA. Co-founder Gerard Platenburg will present Axiomer®, a proprietary RNA base editing platform, on April 6 at 11:00am ET. This innovative technology utilizes the body's ADAR editing machinery to reverse disease-causing RNA mutations. Axiomer® has broad potential, targeting genetic diseases like Leber congenital amaurosis and Usher syndrome, with a strong patent portfolio reinforcing its promise in treating previously untreatable conditions.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced participation in the Cantor Virtual Rare Orphan Disease Summit on March 29, 2022, from 2:45-3:45 PM EDT. The Company focuses on developing transformative RNA therapies for genetic eye diseases, including Leber congenital amaurosis 10 and Usher syndrome. ProQR uses proprietary RNA repair technologies to grow its pipeline, prioritizing patients' needs. For more information, visit www.proqr.com.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced the appointment of Dr. John Maraganore as a strategic advisor to its Supervisory Board. Maraganore, a biopharma leader and founding CEO of Alnylam Pharmaceuticals, will aid in defining ProQR's future direction as the company analyzes data from its Illuminate trial of sepofarsen. Despite recent setbacks, Maraganore highlighted ProQR's solid fundamentals, including deep RNA expertise and a strong cash position. This partnership is expected to bring valuable insights to advance ProQR's RNA therapies for genetic eye diseases.
ProQR Therapeutics N.V. (Nasdaq: PRQR) reported its Q4 and full-year 2021 results, revealing a net loss of €61.7 million or €0.96 per diluted share. The Phase 2/3 Illuminate trial of sepofarsen for LCA10 failed to meet primary endpoints, prompting ongoing analyses. The company plans to advance several clinical trials including ultevursen for Usher syndrome and retinitis pigmentosa. Despite recent setbacks, ProQR maintains a strong cash position with €187.5 million, providing a runway into mid-to-late 2024. Updates on key programs are expected throughout 2022.
ProQR Therapeutics announced that its pivotal Phase 2/3 Illuminate trial of sepofarsen for Leber congenital amaurosis 10 (LCA10) failed to meet its primary endpoint of Best Corrected Visual Acuity (BCVA) at Month 12. The trial showed no significant benefits in either treatment group compared to the sham. While sepofarsen was generally well-tolerated, and the company maintains a strong cash position into mid-2024, the disappointing trial results pose a setback for the targeted RNA therapy aimed at a high unmet medical need. Further analyses are planned for future presentations.
ProQR Therapeutics N.V. (Nasdaq: PRQR) announced its participation in the virtual SVB Leerink 11th Annual Global Healthcare Conference on February 17, 2022. The presentation will be available via live webcast from 1:40 – 2:05 PM EST. ProQR focuses on transformative RNA therapies targeting genetic eye diseases like Leber congenital amaurosis 10 and Usher syndrome. Details for the webcast can be accessed through the company’s website, with archived content available for 30 days post-event.
ProQR Therapeutics N.V. (NASDAQ: PRQR) announced the completion of the last patient visit in the Phase 2/3 Illuminate trial for sepofarsen, an RNA therapy targeting Leber Congenital Amaurosis 10 (LCA10). Results are expected to be announced in Q1 2022. The trial involved 36 patients aged 8 and older and aims to assess improvements in visual acuity. Previous Phase 1/2 studies showed significant improvements in visual acuity at a target registration dose. LCA10 affects approximately 2,000 individuals in the Western world, and sepofarsen has received several fast-track designations.
ProQR Therapeutics N.V. (Nasdaq:PRQR) has amended its convertible debt financing agreement, allowing access to an additional $90 million in three tranches. These tranches, each worth $30 million, will mature over 54 months with an interest-only period of 33 months. This amendment is set to enhance ProQR's balance sheet and extend its cash runway into 2024, supporting its commercialization efforts. The agreement allows for conversion into ordinary shares at a premium of 50% over the average closing price. ProQR aims to use these funds for key milestones in its pipeline.
ProQR Therapeutics (Nasdaq: PRQR) announced the first patient doses in Phase 2/3 clinical trials, Sirius and Celeste, for the investigational RNA therapy QR-421a targeting USH2A mediated retinitis pigmentosa and Usher syndrome. Previous studies suggest QR-421a is well-tolerated and may stabilize vision loss. The Sirius trial aims to enroll 81 participants with advanced vision loss, while the Celeste trial seeks to enroll 120 participants with early to moderate vision loss. Both trials are pivotal as no approved treatments currently exist for these conditions.
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