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].

Core Insights - ProQR Therapeutics announced the presentation of preclinical data for its Axiomer™ RNA editing technology and AX-0810 pipeline program targeting cholestatic diseases at the 20th Annual Meeting of the Oligonucleotide Therapeutics Society [1][2] Group 1: Axiomer™ Technology - Axiomer™ is a next-generation RNA base editing technology that allows for specific single nucleotide changes in RNA, potentially leading to new medicines for various diseases [3][4] - The technology utilizes the human cell's own ADAR machinery to convert Adenosine (A) to Inosine (I), which is then translated as Guanosine (G), effectively correcting disease-causing mutations [3] Group 2: Pipeline and Therapeutic Focus - The AX-0810 program specifically targets NTCP to lower bile acid reuptake in cholestatic diseases, with a presentation scheduled for October 8, 2024 [2] - ProQR aims to address both rare and prevalent diseases with unmet medical needs through its proprietary RNA repair platform [4]

Financial Performance - Total revenue for Q2 2024 reached €6,305 thousand, a significant increase from €1,205 thousand in Q2 2023, representing a growth of 424%[6] - The operating result for Q2 2024 was a loss of €3,600 thousand, an improvement compared to a loss of €8,811 thousand in Q2 2023, reflecting a reduction in losses by 59%[6] - The total comprehensive income for the six months ended June 30, 2024, was a loss of €10,074 thousand, compared to a loss of €17,044 thousand in the same period of 2023, showing an improvement of 41%[6] - Basic loss per share for Q2 2024 was €(0.03), an improvement from €(0.10) in Q2 2023[6] - For the three months ended June 30, 2024, the net result was a loss of €2,692,000, an improvement from a loss of €7,977,000 in the same period last year, representing a 66.2% reduction in losses[8] Research and Development - Research and development costs for the six months ended June 30, 2024, were €16,331 thousand, up from €11,969 thousand in the same period of 2023, indicating a rise of 36%[6] - Research and development expenditures are reflected in the income statement, with development expenses currently not capitalized due to unmet criteria[22] - Research and development costs for the six-month period ended June 30, 2024, increased to €16,331,000, compared to €11,969,000 for the same period in 2023, reflecting increased outsourced activities and full-time employee equivalents[49] Assets and Liabilities - Total assets decreased to €118,051 thousand as of June 30, 2024, down from €137,883 thousand at the end of 2023, a decline of 14%[3] - Equity attributable to owners of the Company fell to €32,854 thousand at June 30, 2024, down from €41,390 thousand at December 31, 2023, a decrease of 21%[3] - Cash and cash equivalents decreased to €578,970 thousand as of June 30, 2024, from €118,925 thousand at December 31, 2023, indicating a significant reduction in liquidity[3] - Total current liabilities increased to €37,279 thousand as of June 30, 2024, compared to €34,203 thousand at the end of 2023, an increase of 6%[3] - Other current liabilities decreased to €5,675,000 as of June 30, 2024, from €8,509,000 as of December 31, 2023[28] Cash Flow - Cash used in operating activities for the six months ended June 30, 2024, was €22,747,000, compared to cash generated of €33,513,000 in the same period last year, indicating a significant cash outflow[8] - The cash and cash equivalents at the end of the period were €78,970,000, down from €118,925,000 at December 31, 2023, reflecting a decrease of 33.5%[23] - Interest received during the three months ended June 30, 2024, was €610,000, compared to €685,000 in the same period last year, showing a decrease of 10.9%[8] - The company reported a net cash used in investing activities of €267,000 for the three months ended June 30, 2024, compared to €294,000 in the same period last year[8] Income and Grants - The company recognized grant income of €366,000 for the six-month period ended June 30, 2024, compared to €76,000 in the same period of 2023[47] - During the six-month period ended June 30, 2024, the company reached milestones amounting to $4,500,000 (€4,196,000) under its collaboration agreement with Eli Lilly[46] Borrowings and Tax - The company’s total borrowings as of June 30, 2024, amount to €4,437,000, an increase from €4,292,000 as of December 31, 2023[29] - The current income tax liability amounts to € nil as of June 30, 2024, consistent with December 31, 2023[54] - The company has not recognized any deferred tax asset related to operating losses due to uncertainty in future earnings[54] - Tax losses can be carried forward indefinitely, but offset against taxable profit is limited to the first € 1 million, with only 50% of excess profit eligible for offset[54] Company Structure and Operations - The company operates in one reportable segment focused on the discovery and development of innovative RNA-based therapeutics[15] - Amylon Therapeutics B.V., an 80% subsidiary, had convertible loans with an average interest rate of 8% per annum[53] - In the six-month period ended June 30, 2024, the amount of convertible loans and accumulated interest waived was € nil, compared to € 509,000 for the same period in 2023[53] - Amylon was legally dissolved in the third quarter of 2023[53]

The presentation will be available on ProQR's website under Presentations & Publications. Learn more about ProQR at www.proqr.com. Investor contact: Sarah Kiely ProQR Therapeutics N.V. T: +1 617 599 6228 skiely@proqr.com or Peter Kelleher LifeSci Advisors T: +1 617 430 7579 pkelleher@lifesciadvisors.com LEIDEN, Netherlands & CAMBRIDGE, Mass., June 18, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (NASDAQ:PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announ ...

ProQR Therapeutics N.V. (NASDAQ:PRQR) ASGCT Investor Webcast May 9, 2024 8:00 AM ET Company Participants Sarah Kiely - IR Daniel de Boer - Founder and CEO Gerard Platenburg - Chief Scientific Officer Conference Call Participants Jon Wolleben - Citizens JMP Steve Seedhouse - Raymond James Sarah Kiely Thank you, and good day, everyone. We appreciate you joining our event today. Today, we are pleased to highlight new preclinical proof-of-concept data for our AX-0810 program targeting NTCP, which was presented ...

Table of Contents Exhibit 99.1 PROQR THERAPEUTICS N.V. Index to Unaudited Condensed Consolidated Financial Statements | Unaudited Condensed Consolidated Statement of Financial Position at March 31, 2024 and December 31, 2023 | PAGE 1 | | --- | --- | | Unaudited Condensed Consolidated Statement of Profit or Loss and Other Comprehensive Income for the | | | Three Month Periods ended March 31, 2024 and 2023 | 2 | | Unaudited Condensed Consolidated Statement of Changes in Equity for the Three Month Periods Ende ...