ProQR(PRQR) - 2024 Q4 - Annual Report

ProQR Year End 2024 Operating and Financial Results Business Update & Recent Progress ProQR advances its Axiomer RNA editing platform, anticipating multiple clinical data readouts by 2026, supported by financing - The company is entering a pivotal growth phase, expecting up to four clinical trial data readouts in 2025 and 2026, with the first topline data for its NTCP program (AX-0810) anticipated in Q4 2025[3][5] - In October 2024, ProQR raised approximately $82.1 million in gross proceeds through a public offering and a concurrent private placement with Eli Lilly, extending its cash runway into mid-2027[3][8] - Strategic partnerships are a key focus, highlighted by a $3.9 billion collaboration with Eli Lilly and an expanded collaboration with the Rett Syndrome Research Trust, which secured up to $9.2 million in total funding[3][8] - In December 2024, Dr. Peter A. Beal, a leading expert in ADAR and RNA biology, was appointed as Chief ADAR Scientist to drive the development of the Axiomer platform[8] Anticipated Upcoming Events & Pipeline Milestones ProQR outlines key pipeline milestones, with lead candidate AX-0810 targeting initial clinical data in Q4 2025 Pipeline Development Milestones | Program | Target Indication | Anticipated Milestone | | :--- | :--- | :--- | | AX-0810 | NTCP for Cholestatic diseases | Q2 2025: CTA submission
Q4 2025: First clinical data | | AX-2402 | MECP2 (R270X) for Rett Syndrome | 2025: Clinical candidate selection
2026: Clinical trial initiation & topline data | | AX-2911 | PNPLA3 for MASH | 2025: Clinical candidate selection
2026: Clinical trial initiation & topline data | | AX-1412 | B4GALT1 for Cardiovascular diseases | mid-2025: Update on optimization for GalNAc delivery | - The partnership with Lilly may be expanded to a total of 15 targets, which would trigger a $50 million opt-in payment to ProQR[8] Year End 2024 Financial Highlights ProQR ended 2024 with a strong cash position of €149.4 million, extending its financial runway into mid-2027 Key Financial Metrics (Year Ended Dec 31) | Financial Metric | 2024 (€ million) | 2023 (€ million) | Change (€ million) | | :--- | :--- | :--- | :--- | | Cash and cash equivalents | €149.4 | €118.9 | +€30.5 | | Net cash (used in)/generated by operating activities | (€36.4) | €21.5 | -€57.9 | | Research and development costs | €36.4 | €25.1 | +€11.3 | | General and administrative costs | €13.7 | €16.2 | -€2.5 | | Net loss | €27.8 | €27.7 | +€0.1 | | Net loss per diluted share | €0.32 | €0.35 | -€0.03 | - The net positive cash flow from operations in 2023 was primarily due to the receipt of a $60 million upfront payment from Eli Lilly[7] - The increase in R&D costs in 2024 was driven by increased investments in the Axiomer platform and advancing pipeline programs[9] Company & Technology Overview ProQR focuses on its proprietary Axiomer RNA editing technology, utilizing the cell's own ADAR machinery for precise RNA edits - ProQR's core technology is Axiomer, a next-generation RNA base editing platform[11][12] - The technology utilizes a patient's own cellular machinery, specifically ADAR (Adenosine Deaminase Acting on RNA) enzymes, to edit RNA[11] - The mechanism involves using Editing Oligonucleotides (EONs) to direct ADAR to convert a specific Adenosine (A) to an Inosine (I), which the cell translates as a Guanosine (G), thereby correcting mutations or modulating protein function[11] Forward Looking Statements This section serves as a legal disclaimer, cautioning that the report contains forward-looking statements subject to significant risks - The press release includes forward-looking statements regarding clinical trial timelines, data readouts, CTA filings, the potential of the Axiomer platform, and financial position[14] - Key risks that could affect results include the timing and cost of clinical trials, reliance on contract research organizations and manufacturers, unpredictability of regulatory review, and general economic and geopolitical instability[14] - The company advises against placing undue reliance on these forward-looking statements and assumes no obligation to update them[14]