LEIDEN, Netherlands & CAMBRIDGE, Mass., May 02, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform today announced that Company management is scheduled to present at the Citizens (JMP) Life Sciences Conference in New York City on Thursday, May 8 at 11:30 AM EDT. A live webcast of the Company’s fireside chat will be available from the “Investors & M ...

Core Insights - ProQR Therapeutics has appointed Dennis Hom as Chief Financial Officer and Dr. Cristina Lopez Lopez as Chief Medical Officer to advance its Axiomer platform and RNA editing programs into clinical stages [1][2][3] Leadership Appointments - Dennis Hom brings over 25 years of financial leadership experience, having raised more than $4.5 billion in capital and executed transactions totaling over $57 billion in disclosed value [2] - Dr. Cristina Lopez Lopez has over 20 years of experience in translational R&D, previously serving as Global Head of Neurodegeneration at Johnson & Johnson and holding senior roles at Roche and Novartis [3] Transition of Current Executives - Current CFO Jurriaan Dekkers and Chief Corporate Development Officer René Beukema will step down but remain during a transitional period to ensure continuity [4][5] Axiomer Technology Overview - ProQR is pioneering Axiomer™, a next-generation RNA base editing technology that allows specific single nucleotide edits in RNA, potentially leading to new medicines for various diseases [5][7] - Axiomer™ Editing Oligonucleotides (EONs) utilize the human cell's ADAR machinery to correct disease-causing mutations in RNA, modulating protein expression or altering protein functions [5]

Core Viewpoint - ProQR reported a quarterly loss of $0.10 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.07, marking an earnings surprise of -42.86% [1] Financial Performance - The company posted revenues of $4.6 million for the quarter ended December 2024, missing the Zacks Consensus Estimate by 51.53%, compared to revenues of $3.54 million a year ago [2] - Over the last four quarters, ProQR has surpassed consensus EPS estimates three times and topped consensus revenue estimates two times [2] Stock Performance - ProQR shares have declined approximately 30.2% since the beginning of the year, contrasting with the S&P 500's decline of -4.8% [3] - The current consensus EPS estimate for the upcoming quarter is -$0.09 on revenues of $11.57 million, and for the current fiscal year, it is -$0.36 on revenues of $28.29 million [7] Industry Outlook - The Medical - Biomedical and Genetics industry, to which ProQR belongs, is currently in the top 26% of over 250 Zacks industries, indicating a favorable outlook [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact ProQR's stock performance [5]

Financial Reporting and Compliance - The financial statements have been prepared in accordance with IFRS, requiring estimates and assumptions that may differ from actual results[538] - There have been no material adjustments to prior period estimates for any of the periods included in the Annual Report[538] - The Company uses non-GAAP financial measures alongside IFRS measures to present its financial position and operating results[24] - The Company maintains its books and records in euro, presenting financial statements in this currency as its functional currency[25] Risks and Uncertainties - Forward-looking statements involve risks and uncertainties that may cause actual results to differ materially from expectations[32] - The Company faces risks related to geopolitical developments, including economic sanctions and trade barriers that could impact operations[37] - The impact of health epidemics and global supply chain pressures may slow or halt operations and activities[37] - The Company has not made any updates to forward-looking statements after the date of the Annual Report[36] Clinical Trials and Collaborations - The Company relies on contract research organizations (CROs) for timely enrollment in clinical trials, which may affect execution timelines[33] - The collaboration with Eli Lilly and Company includes potential milestone and royalty payments from commercial product sales[33]

Exhibit 99.1 ProQR Announces Year End 2024 Operating and Financial Results LEIDEN, Netherlands & CAMBRIDGE, Mass., March 13, 2025 – ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer RNA editing technology platform, today reported its financial and operating results for the year ended December 31, 2024, and provided a business update. "As we continue to advance our Axiomer ADAR-mediated RNA editing platfo ...

Core Viewpoint - ProQR Therapeutics is entering a significant growth phase with its Axiomer RNA editing platform, anticipating multiple clinical trial data readouts in 2025 and 2026, which could demonstrate the therapeutic potential of RNA editing [2][5]. Financial Highlights - As of December 31, 2024, ProQR held cash and cash equivalents of €149.4 million, an increase from €118.9 million at the end of 2023 [7]. - The net cash used in operating activities for the year ended December 31, 2024, was €36.4 million, compared to a net cash generation of €21.5 million in 2023 [7]. - Research and development costs for 2024 were €36.4 million, up from €25.1 million in 2023, reflecting increased investments in the platform and pipeline programs [8]. - The net loss for 2024 was €27.8 million, or €0.32 per diluted share, compared to a net loss of €27.7 million, or €0.35 per diluted share in 2023 [9]. Recent Progress - ProQR expanded its collaboration with the Rett Syndrome Research Trust, securing up to $9.2 million for advancing AX-2402 into clinical trials [6]. - The company appointed Dr. Peter A. Beal as Chief ADAR Scientist to enhance the development of its Axiomer platform [6]. - In October 2024, ProQR completed a public offering and private placement, raising approximately $82.1 million [6]. Anticipated Upcoming Events - The company plans to submit a Clinical Trial Application (CTA) for AX-0810 in Q2 2025, with the first clinical data readout expected in Q4 2025 [5][6]. - ProQR anticipates up to four clinical data readouts in 2025 and 2026 across various programs, including AX-2402 for Rett Syndrome and AX-2911 for MASH [5][13].

Revenue and Income - Total revenue for the three months ended September 30, 2024, was €3,843 million, a significant increase from €1,370 million in the same period of 2023, representing a growth of 180%[10] - The company’s total revenue from grant income for the nine-month period ended September 30, 2024, was €504,000, significantly higher than €76,000 for the same period in 2023, marking an increase of over 563%[89] Expenses and Costs - Research and development costs for the nine months ended September 30, 2024, were €25,745 million, compared to €17,415 million for the same period in 2023, reflecting an increase of 48%[10] - General and administrative costs decreased to €9,748,000 for the nine-month period ended September 30, 2024, down from €11,486,000 in the same period of 2023[17] - The company’s share-based compensation expenses for the nine-month period ended September 30, 2024, were €1,976,000, down from €2,304,000 for the same period in 2023, reflecting a decrease of approximately 14%[73] Financial Position - Total assets decreased to €106,851 million as of September 30, 2024, down from €137,883 million at the end of 2023, a decline of 22.5%[6] - Total equity attributable to owners of the Company was €24,982 million as of September 30, 2024, down from €41,390 million at the end of 2023, a decrease of 39.5%[6] - Total liabilities decreased to €81,869 million as of September 30, 2024, down from €96,493 million at the end of 2023, a decline of 15.1%[6] - Cash and cash equivalents decreased to €89,401 million as of September 30, 2024, from €118,925 million at the end of 2023, a reduction of 25%[6] Losses and Earnings - The operating result for the three months ended September 30, 2024, was a loss of €8,716 million, compared to a loss of €7,391 million in the same period of 2023[10] - The company reported a total comprehensive loss of €18,559 million for the nine months ended September 30, 2024, compared to a loss of €22,799 million in the same period of 2023, indicating a reduction in losses of 18.8%[10] - Basic loss per share for the nine months ended September 30, 2024, was €0.23, an improvement from €0.28 in the same period of 2023[10] - For the three months ended September 30, 2024, the net result was a loss of €8,108,000 compared to a loss of €5,420,000 for the same period in 2023[17] Cash Flow and Activities - Cash used in operating activities for the nine months ended September 30, 2024, was €29,046,000, a significant decrease from cash generated of €25,120,000 in the same period of 2023[17] - The company reported a net cash generated by investing activities of €16,714,000 for the three months ended September 30, 2024, compared to a cash used of €339,000 in the same period of 2023[17] - The company recognized interest received of €860,000 for the three months ended September 30, 2024, compared to €802,000 in the same period of 2023[17] Deferred Revenue and Payments - The total deferred revenue balance as of September 30, 2024, was €55,588,000, with €55,236,000 related to the Eli Lilly performance obligation[39] - During the nine-month period ended September 30, 2024, the company recognized milestone payments of $5,500,000 (€5,094,000) under its collaboration agreement with Eli Lilly[88] - The company received an upfront payment of $60,000,000 (€56,412,000) in February 2023 under the amended collaboration agreement with Eli Lilly, which was recognized under deferred income[82] Investments and Financial Assets - The company has invested in financial assets with an original maturity of longer than three months but shorter than twelve months during the nine-month period ended September 30, 2024[27] - ProQR holds a 3.9% interest in Phoenicis Therapeutics Inc., which discontinued operations in Q3 2024, resulting in a fair value loss recognized in other comprehensive income[96] - ProQR holds a 5.1% interest in Yarrow Biotechnology Inc., with the fair value of this financial asset remaining at €nil as of September 30, 2024[97] Other Financial Metrics - The carrying amount of the right-of-use asset related to the lease of the Leiden office and laboratory space was €11,873,000 as of September 30, 2024, down from €14,524,000 as of December 31, 2023, a decrease of about 18%[55] - Other current liabilities decreased to €7,439,000 as of September 30, 2024, from €8,509,000 as of December 31, 2023, indicating a reduction of approximately 13%[56] - The income tax benefit for the nine-month period ended September 30, 2024, amounted to €197,000, an increase from €83,000 in the same period of 2023, consisting of refunds received for prior years[103] Company Outlook - The company expects to continue as a going concern based on its existing funding and projected cash flows[29] - No significant temporary differences exist between accounting and tax results, and the current income tax liability is €nil as of September 30, 2024[102] - The amount of convertible loans waived under agreements with Amylon in the nine-month period ended September 30, 2024, is €nil, compared to €1,866,000 in the same period of 2023[100] - Amylon Therapeutics B.V. was legally dissolved in Q3 2023, impacting the company's financial liabilities[101]

Core Viewpoint - ProQR Therapeutics has successfully closed a public offering of 18 million ordinary shares at a price of $3.50 per share, raising total gross proceeds of $63 million, and has also completed a private placement of 3,523,538 shares to Eli Lilly for approximately $12.3 million [1][2]. Group 1: Public Offering Details - The public offering consisted of 18,000,000 ordinary shares sold at a public offering price of $3.50 per share, resulting in gross proceeds of $63.0 million before expenses [1]. - The offering was managed by Evercore ISI, Cantor, Raymond James, and Oppenheimer & Co. as joint lead bookrunning managers [2]. Group 2: Private Placement Details - The private placement involved the sale of 3,523,538 ordinary shares to Eli Lilly at the same price as the public offering, totaling approximately $12.3 million in gross proceeds [2]. - Shares sold in the private placement were not subject to underwriting discounts or commissions [2]. Group 3: Regulatory Information - A shelf registration statement on Form F-3 for the offering was filed with the SEC on September 30, 2024, and was declared effective on October 10, 2024 [3]. - The offering was conducted only through a prospectus and prospectus supplement that are part of the registration statement [3]. Group 4: Company Overview - ProQR Therapeutics focuses on developing transformative RNA therapies using its proprietary Axiomer™ RNA editing technology, which aims to create a new class of medicines for diseases with unmet needs [5].

Core Viewpoint - ProQR Therapeutics has announced a public offering of 18 million ordinary shares at a price of $3.50 per share, aiming to raise approximately $63 million in gross proceeds to fund research and development initiatives [1][3]. Group 1: Offering Details - The public offering consists of 18,000,000 ordinary shares priced at $3.50 each, totaling gross proceeds of $63 million before expenses [1]. - ProQR has granted underwriters a 30-day option to purchase up to 2,700,000 additional shares at the public offering price [1]. - The offering is expected to close on October 24, 2024, subject to customary closing conditions [4]. Group 2: Concurrent Private Placement - ProQR has entered into a share purchase agreement with Eli Lilly to sell 3,523,538 ordinary shares, allowing Lilly to maintain its 16.4% ownership stake post-offering [2]. - The shares sold to Lilly will generate approximately $12.3 million in gross proceeds, with a purchase price cap of $15 million [2]. - The private placement shares will not be registered as part of the public offering and are exempt from registration under the Securities Act [3][6]. Group 3: Use of Proceeds - ProQR intends to use the net proceeds from both the public offering and the private placement primarily for research and development, clinical development, and expansion of its programs [3]. - Remaining funds will be allocated for working capital, capital expenditures, and other general corporate purposes [3]. Group 4: Company Overview - ProQR Therapeutics focuses on developing transformative RNA therapies using its proprietary Axiomer™ RNA editing technology [7]. - The company aims to create a new class of medicines targeting both rare and prevalent diseases with unmet medical needs [7].

Core Viewpoint - ProQR Therapeutics has initiated an underwritten public offering of its ordinary shares to fund research and development, clinical development, and general corporate purposes [1][2]. Group 1: Offering Details - The offering includes a 30-day option for underwriters to purchase an additional 15% of the offering amount at the public offering price [1]. - The offering is subject to market conditions, and there is no assurance regarding its completion or terms [1]. Group 2: Use of Proceeds - ProQR plans to utilize the net proceeds from the offering, along with existing cash, primarily for research and development and clinical development of current and future product candidates [2]. - Remaining funds will be allocated for working capital, capital expenditures, and other general corporate purposes [2]. Group 3: Regulatory Filings - A shelf registration statement on Form F-3 was filed with the SEC on September 30, 2024, and became effective on October 10, 2024 [3]. - A preliminary prospectus supplement will be filed with the SEC to describe the terms of the offering [3]. Group 4: Company Overview - ProQR Therapeutics focuses on transformative RNA therapies using its proprietary Axiomer™ RNA editing technology platform [5]. - The Axiomer™ technology aims to make specific single nucleotide edits in RNA, potentially leading to new medicines for both rare and prevalent diseases [5].