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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 (NASDAQ: PRQR) announced an expanded collaboration with the Rett Syndrome Research Trust (RSRT), securing an additional $8.1 million in funding, bringing the total to $9.1 million. The funding will support the advancement of AX-2402, a therapeutic candidate based on ProQR's Axiomer RNA editing platform, into clinical trials.
The treatment targets individuals with Rett syndrome who have the R270X mutation in MECP2 gene. Rett Syndrome, affecting approximately 350,000 people worldwide, predominantly girls, is a rare neurodevelopmental disorder with no current cure. The expanded collaboration focuses on optimizing therapeutic candidates targeting the MECP2 protein and advancing them toward clinical development.
ProQR Therapeutics (Nasdaq: PRQR) has appointed Dr. Peter Beal as Chief ADAR Scientist. Dr. Beal, a professor of Chemistry at the University of California, Davis, and a former Scientific Advisory Board member, will oversee platform optimization and scientific developments in RNA editing oligonucleotides design.
As Chief ADAR Scientist, Dr. Beal will leverage his extensive expertise in ADAR biology and RNA chemistry to advance ProQR's Axiomer™ RNA editing technology platform. His research has focused on ADAR-mediated RNA-editing mechanisms, including applications for treating conditions like Rett syndrome, Alzheimer's disease, and Parkinson's disease.
ProQR Therapeutics (Nasdaq: PRQR) announced a virtual Analyst and Investor Event scheduled for December 11, 2024, from 10:00 am to 12:30 pm EST. The event will showcase the company's Axiomer™ ADAR-mediated RNA editing platform and provide updates on development candidates, including NTCP and B4GALT1 programs, AX-0810, and AX-1412.
Key presenters include Daniel A. de Boer (Founder and CEO), Gerard Platenburg (Chief Scientific Officer), and René Beukema (Chief Corporate Development Officer). Guest speakers include Dr. Peter Beal, Professor at UC Davis and expert in ADAR enzymes, and Prof. Gideon Hirschfield, specialist in autoimmune liver diseases from the Toronto Centre for Liver Disease.
The event will be accessible via webcast on ProQR's website, with a phone option available through pre-registration. A replay will be available for approximately 30 days following the event.
ProQR Therapeutics reported Q3 2024 financial results, highlighting €89.4 million in cash and cash equivalents, plus $82.1 million from October financing extending runway into mid-2027. The company's first Axiomer™ RNA editing program, AX-0810, targets NTCP for cholestatic diseases and is advancing toward clinical trials. Q3 net loss was €8.1 million (€0.10 per share), compared to €5.4 million (€0.07 per share) in Q3 2023. R&D costs increased to €9.4 million from €5.4 million year-over-year, while G&A costs remained stable at €3.3 million. The company achieved $5.5 million in milestones from its Eli Lilly collaboration during the first three quarters of 2024.
ProQR Therapeutics announced the closing of its underwritten public offering of 18,000,000 ordinary shares at $3.50 per share, raising gross proceeds of $63.0 million. Additionally, the company completed a concurrent private placement with Eli Lilly and Company for 3,523,538 ordinary shares at the same price, generating approximately $12.3 million in gross proceeds. The combined offerings total $75.3 million in gross proceeds. Evercore ISI, Cantor, Raymond James and Oppenheimer & Co. served as joint lead bookrunning managers for the public offering.
ProQR Therapeutics (Nasdaq: PRQR) has priced an underwritten public offering of 18 million ordinary shares at $3.50 per share, raising gross proceeds of $63.0 million. The company also granted underwriters a 30-day option to purchase up to 2.7 million additional shares. Concurrently, Eli Lilly agreed to purchase 3.52 million shares in a private placement at the same price to maintain its 16.4% ownership stake, contributing additional gross proceeds of $12.3 million. The combined proceeds will fund research, development, and clinical programs, along with working capital and general corporate purposes. The offering is expected to close on October 24, 2024.
ProQR Therapeutics N.V. (Nasdaq: PRQR), a company focused on RNA therapies using its Axiomer™ RNA editing technology platform, has announced a proposed underwritten public offering of its ordinary shares. The company intends to grant underwriters a 30-day option to purchase up to an additional 15% of the offering amount. Proceeds from the offering will be used to fund research and development, clinical development, and general corporate purposes.
Joint lead bookrunning managers for the offering include Evercore ISI, Cantor, Raymond James, and Oppenheimer & Co. A shelf registration statement on Form F-3 has been filed with the SEC and declared effective. The offering is subject to market conditions, and there is no assurance of its completion or terms.
ProQR Therapeutics NV (Nasdaq: PRQR), a company focused on transformative RNA therapies using its proprietary Axiomer™ RNA editing technology platform, has announced its participation in the 2024 Cantor Global Healthcare Conference. The event is scheduled for Tuesday, September 17 at 9:10 am ET in New York City. Company management will present in a fireside chat format, which will be accessible via live webcast from the 'Investors & Media' section of ProQR's website under 'Events'. For those unable to attend live, a replay of the webcast will be available for approximately 30 days following the presentation.
ProQR Therapeutics NV (Nasdaq: PRQR) reported its Q2 2024 financial and operating results. Key highlights include:
- AX-0810 (for cholestatic diseases) and AX-1412 (for cardiovascular diseases) programs advancing to clinic in late 2024/early 2025
- $4.5 million in milestones achieved in H1 from Lilly partnership
- €96.2 million cash position as of Q2 end, providing runway into mid-2026
- Net loss of €2.7 million (€0.03 per diluted share) for Q2 2024
- R&D costs increased to €7.1 million in Q2 2024 from €5.9 million in Q2 2023
- General and administrative costs decreased to €3.0 million in Q2 2024 from €4.1 million in Q2 2023
ProQR plans to announce translational data and clinical trial designs for AX-0810 and AX-1412 in H2 2024.
ProQR Therapeutics (Nasdaq: PRQR) announced its participation in the RNA Editing Summit from June 18-20, 2024, in Boston. Gerard Platenburg, Chief Scientific Officer and Co-Founder, will present on June 20 at 2:00pm ET. The presentation, titled “Developing Axiomer RNA Editing Technology Towards Clinical Development,” will focus on ProQR’s ADAR-mediated Axiomer RNA editing technology, optimization strategies, and the AX-0810 program targeting NTCP for cholestatic diseases. The presentation will also cover in vivo models of biomarkers for NTCP and will be accessible on ProQR’s website under Presentations & Publications.
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