Apic Bio, Inc.

▎Armstrong 2024年10月7日，诺贝尔生理学或医学奖被授予给microRNA（miRNA）域做出突破性贡献的Victor Ambros教授和Gary Ruvkun教授，这两位学者发现了microRNA及其在转录后基因调控中的作用。 核酸药物领域，mRNA、siRNA、ASO等都已经成药并受到业界广泛关注，miRNA似乎还相对小众，那么这个赛道都有哪些主要的Biotech呢？ 截至目前，约有十几款miRNA药物（部分为靶向并抑制miRNA，另一部分则为miRNA类似物）进入临床阶段，适应症覆盖实体瘤、亨廷顿病、NASH、心衰、丙肝等， 递送系统也是miRNA药物开发的关键环节之一。 Abivax  Abivax为纳斯达克上市公司，目前市值为6亿美元。Abivax核心管线Obefazimod，可以选择性上调miR-124，用于治疗炎症性肠病。 Regulus Therapeutics Regulus Therapeutics为纳斯达克上市公司，目前市值1亿美元，首付管线为RGLS8429，靶向并抑制miR-17，用于治疗常染色体显性多囊肾（ADPKD），目前处于1b期临床阶段，正在筹备2/3期临床。 EnGeneIC EnGeneIC聚焦开发EDV技术，来递送siRNA或miRNA。 Cardior Pharmaceuticals Cardior Pharmaceuticals的核心管线CDR132L为一款阻断miR-132的基于寡核苷酸的ncRNA抑制剂，1b期临床中表现出心脏功能改善的效果，目前在进行HFrEF的二期临床。2024年3月，诺和诺德以11亿美元的价格收购Cardior Pharmaceuticals。 Transcode Therapeutics Transcode Therapeutics的首发管线为靶向miR-10b的TTX-MC138，用于治疗肿瘤，今年9月完成首例患者给药。 miRagen 原本以miRNA诊断为主，后被Viridian借壳上市。 miRNA Therapeutics miRNA Therapeutics的核心管线MRX-34为miR-34类似物，用于治疗肿瘤，因安全性问题终止开发。 此外，还有Arthex Biotech、Apic Bio、InteRNA Technologies，被韩国Nexturn Biosciences收购的RosVivo Therapeutics等。 总结 国内方面，觅瑞集团专注开发基于miRNA的液体活检，今年5月递交港股IPO申请。觅瑞集团于2023年7月完成5000万美元D轮融资，投后估值6亿美元。江苏命码生物聚焦开发miRNA诊断或治疗药物，并于2021年与金斯瑞合作开发KRAS为靶标基因的RNAi治疗药物和以miR-214为靶标基因的适应于多种抗肿瘤细胞逃逸的RNAi治疗药物。命码生物的创始人为南京大学张辰宇教授，一直致力于细胞外miRNA的研究。2021年，张辰宇又创立了艾玛生物，但管线以外泌体递送siRNA为主。 Armstrong技术全梳理系列 GPRC5D靶点全梳理； CD40靶点全梳理； CD47靶点全梳理； 补体靶向药物技术全梳理； 补体药物：眼科治疗的重要方向； Claudin 6靶点全梳理； Claudin 18.2靶点全梳理； 靶点冷暖，行业自知； 中国大分子新药研发格局； 被炮轰的“me too”； 佐剂百年史； 胰岛素百年传奇； CUSBEA：风雨四十载； 中国新药研发的焦虑； 中国生物医药企业的研发竞争； 中国双抗竞争格局； 中国ADC竞争格局； 中国双抗技术全梳理； 中国ADC技术全梳理； Ambrx技术全梳理； Vir Biotech技术全梳理； Immune-Onc技术全梳理； 亘喜生物技术全梳理； 康哲药业技术全梳理； 科济药业技术全梳理； 恺佧生物技术全梳理； 同宜医药技术全梳理； 百奥赛图技术全梳理； 腾盛博药技术全梳理； 创胜集团技术全梳理； 永泰生物技术全梳理； 中国抗体技术全梳理； 德琪医药技术全梳理； 德琪医药技术全梳理2.0； 和铂医药技术全梳理； 荣昌生物技术全梳理； 再鼎医药技术全梳理； 药明生物技术全梳理； 恒瑞医药技术全梳理； 豪森药业技术全梳理； 正大天晴技术全梳理； 吉凯基因技术全梳理； 基石药业技术全梳理； 百济神州技术全梳理； 百济神州技术全梳理第2版； 信达生物技术全梳理； 信达生物技术全梳理第2版； 中山康方技术全梳理； 复宏汉霖技术全梳理； 先声药业技术全梳理； 君实生物技术全梳理； 嘉和生物技术全梳理； 志道生物技术全梳理； 道尔生物技术全梳理； 尚健生物技术全梳理； 康宁杰瑞技术全梳理； 科望医药技术全梳理； 科望医药技术全梳理2.0； 岸迈生物技术全梳理； 礼进生物技术全梳理； 康桥资本技术全梳理； 余国良的抗体药布局； 荃信生物技术全梳理； 安源医药技术全梳理； 三生国健技术全梳理； 仁会生物技术全梳理； 乐普生物技术全梳理； 同润生物技术全梳理； 宜明昂科技术全梳理； 派格生物技术全梳理； 迈威生物技术全梳理； Momenta技术全梳理； NGM技术全梳理； 普米斯生物技术全梳理； 普米斯生物技术全梳理2.0； 三叶草生物技术全梳理； 贝达药业抗体药全梳理； 泽璟制药抗体药全梳理； 恒瑞医药抗体药全梳理； 齐鲁制药抗体药全梳理； 石药集团抗体药全梳理； 豪森药业抗体药全梳理； 华海药业抗体药全梳理； 科伦药业抗体药全梳理； 百奥泰技术全梳理； 凡恩世技术全梳理。

~ On track to initiate FDA interaction regarding AMT-130 in second quarter of 2024 and provide a clinical update from the Phase I/II trials in mid-2024 ~ ~ Clinical trial initiation for Fabry disease on track to begin in second quarter of 2024, followed by refractory mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024 ~ ~ Comprehensive review of operations and options to reduce expenses underway and expected to be completed in mid-2024 ~ LEXINGTON, Mass. and AMSTERDAM, May 07, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the first quarter of 2024 and highlighted recent progress across its business. “In the first quarter, we made solid progress across three key business priorities – clarifying the road ahead for AMT-130 in Huntington’s disease, initiating three new Phase I/II clinical trials, and prudently conserving our capital,” stated Matt Kapusta, chief executive officer of uniQure. “We are on track to initiate FDA interactions on AMT-130 this quarter and look forward to presenting longer-term follow-up data from our Phase I/II trials mid-year, which will include up to three years of patient follow-up on 29 treated patients. We have also made significant progress towards the initiation of new Phase I/II studies. We have obtained IRB approvals for initial sites, which are poised for imminent activation, and have also identified prospective patients. Enrollment for the Fabry disease study is on track to begin in the second quarter of 2024, with the SOD1-ALS and MTLE studies expected to begin in third quarter.” “In addition, we are actively engaged in a comprehensive review of uniQure’s operations and options to reduce expenses and increase shareholder value,” he added. “We believe deeply in the significant value of our assets, as well as the need to achieve a focused and sustainable level of investment in order to maximize this potential. We expect to complete this evaluation mid-2024.” Recent Updates Advancing AMT-130 for the treatment of Huntington’s disease The Company is on track to initiate interactions with the U.S. Food and Drug Administration in the second quarter of 2024 with the goal of defining the future clinical and regulatory pathway for AMT-130. These interactions, which are expected to continue throughout the year, are expected to include discussions of the interim data from the ongoing Phase I/II clinical trials and the potential to leverage natural history comparators alongside our long-term Phase I/II clinical data. The Company expects to provide an update on its regulatory plans as part of the next clinical update on AMT-130 mid-year and have greater clarity regarding a potential approval pathway for AMT-130 before the end of the year.In mid-2024, the Company expects to provide an update from its ongoing Phase I/II clinical trials of AMT-130. The data will include up to three years of follow-up data on 29 treated patients in both the low- and higher-dose cohorts, of which 21 patients will have a minimum of two years of follow-up.Patient dosing is ongoing in a third cohort of up to 12 patients to further evaluate both doses of AMT-130 in combination with perioperative immunosuppression, with a focus on evaluating near-term safety and tolerability. Enrollment in this third cohort is expected to be completed in the second half of 2024. Initiating new Phase I/II clinical studies AMT-191 for the treatment of Fabry disease - Patient enrollment in a Phase I/IIa clinical trial is expected to begin in the second quarter of 2024. The Phase I/IIa clinical trial will be a U.S.-based, multi-center, open-label trial consisting of two cohorts enrolling up to six adult male patients each. The study is designed to evaluate safety, tolerability, and early signs of efficacy. AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) – Patient enrollment in a Phase I/II clinical trial is expected to begin in the third quarter of 2024. The Phase I/II clinical trial will be a U.S.-based, multi-center, open-label trial consisting of three cohorts with up to four patients each receiving a one-time intrathecal infusion with immunosuppression. The study is designed to evaluate safety, tolerability, and early signs of efficacy. AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE) – Patient enrollment in a Phase I/IIa clinical trial is expected to begin in third quarter of 2024. The Phase I/II will be a U.S.-based consisting of two parts. The first part is a multicenter, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and first signs for efficacy of AMT-260 in patients with refractory MTLE. The second part is expected to be a randomized, controlled trial to generate proof of concept (POC) data. Upcoming Investor Events RBC Capital Markets 2024 Global Healthcare Conference, May 15th – New York, NY Financial Highlights Cash position: As of March 31, 2024, the Company held cash and cash equivalents and investment securities of $555.7 million, compared to $617.9 million as of December 31, 2023. The Company expects cash, cash equivalents and investment securities will fund operations into the second quarter of 2027. Revenues: Revenue for the three months ended March 31, 2024 was $8.5 million, compared to $5.3 million in the same period in 2023. The net increase of $3.2 million in revenue resulted from an increase of $2.9 million from collaboration revenue and an increase of $1.2 million in license revenue partially offset by a decrease of $0.9 million from contract manufacturing of HEMGENIX® for CSL. Cost of contract manufacturing revenues: Cost of contract manufacturing revenues were $9.1 million for the three months ended March 31, 2024, compared to $2.4 million for the same period in 2023. The increase primarily relates to expensing costs previously capitalized as inventory. R&D expenses: Research and development expenses were $40.7 million for the three months ended March 31, 2024, compared to $60.8 million during the same period in 2023. The $20.1 million decrease was primarily related to an $8.1 million decrease in external spend for the ALS SOD1 program, as prior year costs included a $10.0 million one-time cost for the acquisition of the program from Apic Bio. Additionally, there was a $7.0 million decrease in preclinical and other program spend, a $2.0 million decrease in costs incurred related to preclinical supplies and a $2.7 million decrease in employee related expenses. SG&A expenses: Selling, general and administrative expenses were $13.9 million for the three months ended March 31, 2024, compared to $17.8 million during the same period in 2023. The $3.9 million decrease was primarily related to a $1.7 million decrease in professional and intellectual property fees, as well as a reduction of expenses for information technology. Other non-operating items, net: Other non-operating items, net was an expense of $10.7 million for the three months ended March 31, 2024, compared to $4.3 million for the same period in 2023. The $6.5 million increase in other non-operating items, net was primarily related to an increase in non-cash interest expense of $12.5 million related to the royalty agreement that the Company entered into in May 2023, which partially was offset by an increase of $4.8 million in interest income earned on investment securities and cash on hand. Net loss:The net loss for the three months ended March 31, 2024, was $65.6 million, or $1.36 basic and diluted loss per ordinary share, compared to $77.2 million net loss for the same period in 2023, or $1.63 basic and diluted loss per ordinary share. About uniQure uniQure’s mission is to reimagine the future of medicine by delivering innovative cures that transform lives. The recent approvals of our gene therapy for hemophilia B – a historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. We are now leveraging our modular and validated technology and manufacturing platform to advance a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory mesial temporal lobe epilepsy, amyotrophic lateral sclerosis (ALS), Fabry disease, and other severe diseases. www.uniQure.com uniQure Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," “establish,” "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," “seek,” "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning the Company’s cash runway and its ability to fund its operations into the second quarter of 2027; the Company’s ongoing review of its operations and options to reduce expenses, the Company’s expectations that such review will lead to a strategy that, if effectively executed, will reduce operating expenses and increase shareholder value, and the expected timing of the completion of such review; the Company’s plans to announce additional follow-up data from its ongoing U.S. and European Phase I/II clinical studies of AMT-130; the Company’s plans to initiate interactions with the FDA regarding the further development of AMT-130, the timing of such interactions and expectations regarding regulatory clarity from such interactions; the Company’s plans regarding the third cohort in its AMT-130 clinical trial and the timing of enrollment for such cohort; and the Company’s plans to initiate patient enrollment for AMT-191 in the second quarter of 2024, and to initiate patient enrollment for AMT-260 and AMT-162 in the third quarter of 2024. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the clinical results and the development and timing of the Company’s programs; the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to approval; the Company’s ability to continue to build and maintain the company infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company’s ability to obtain, maintain and protect intellectual property; the Company’s ability to fund its operations and to raise additional capital as needed; and the impact of global economic uncertainty, rising inflation, rising interest rates or market disruptions on its business. These risks and uncertainties are more fully described under the heading "Risk Factors" in the Company’s periodic filings with the U.S. Securities & Exchange Commission (“SEC”), including its Annual Report on Form 10-K filed February 28, 2024 and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future. uniQure Contacts: FOR INVESTORS: FOR MEDIA: Maria E. CantorDirect: 339-970-7536Mobile: 617-680-9452m.cantor@uniQure.comChiara RussoDirect: 617-306-9137Mobile: 617-306-9137c.russo@uniQure.comTom MaloneDirect: 339-970-7558Mobile:339-223-8541t.malone@uniQure.com uniQure N.V.UNAUDITED CONSOLIDATED BALANCE SHEETS March 31, December 31, 2024 2023 (in thousands, except share and per share amounts)Current assets Cash and cash equivalents$243,062 $241,360Current investment securities 312,621 376,532Accounts receivable and contract asset 10,717 4,193Inventories 7,672 12,024Prepaid expenses 18,839 15,089Other current assets and receivables 3,092 2,655Total current assets 596,003 651,853Non-current assets Property, plant and equipment, net 44,554 46,548Operating lease right-of-use assets 27,695 28,789Intangible assets, net 59,111 60,481Goodwill 25,795 26,379Deferred tax assets, net 11,594 12,276Other non-current assets 5,298 5,363Total non-current assets 174,047 179,836Total assets$ 770,050 $ 831,689Current liabilities Accounts payable$5,231 $6,586Accrued expenses and other current liabilities 22,658 30,534Current portion of contingent consideration 27,587 28,211Current portion of operating lease liabilities 7,997 8,344Total current liabilities 63,473 73,675Non-current liabilities Long-term debt 102,120 101,749Liability from royalty financing agreement 405,398 394,241Operating lease liabilities, net of current portion 26,983 28,316Contingent consideration, net of current portion 14,625 14,795Deferred tax liability, net 7,376 7,543Other non-current liabilities 3,321 3,700Total non-current liabilities 559,823 550,344Total liabilities 623,296 624,019Shareholders' equity Total shareholders' equity 146,754 207,670Total liabilities and shareholders' equity$ 770,050 $ 831,689 uniQure N.V.UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS Three months ended March 31, 2024 2023 (in thousands, except share and per share amounts)Total revenues$ 8,485 $ 5,325 Operating expenses: Cost of license revenues (150) — Cost of contract manufacturing revenues (9,076) (2,435)Research and development expenses (40,692) (60,809)Selling, general and administrative expenses (13,937) (17,848)Total operating expenses (63,855) (81,092)Other income 1,376 1,811 Other expense (234) (216)Loss from operations (54,228) (74,172)Non-operating items, net (10,734) (4,262)Loss before income tax (expense) / benefit$ (64,962) $ (78,434)Income tax (expense) / benefit (656) 1,207 Net loss$ (65,618) $ (77,227) Basic and diluted net loss per ordinary share$(1.36) $(1.63)Weighted average shares used in computing basic and diluted net loss per ordinary share 48,384,510 47,436,335

~ Presented promising clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington’s disease; Up to three years of follow-up data to be presented in mid-2024; Regulatory interactions and clarity on potential strategies for clinical development expected in 2024 ~ ~ Announced FDA clearance of two Investigational New Drug (IND) applications; Initiation of Phase I/II clinical trials in mesial temporal lobe epilepsy (mTLE) and Fabry disease, in addition to SOD1-ALS, are expected in the first half of 2024 ~ ~ Announced strategic reorganization to advance multiple clinical-stage programs and deliver $180 million of cost savings over the next three years ~ ~ Cash position of approximately $618 million as of December 31, 2023 expected to fund operations into the second quarter of 2027 ~ LEXINGTON, Mass. and AMSTERDAM, Feb. 28, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the fourth quarter and full year 2023 and highlighted recent progress across its business. “We are pleased with the progress made across the company in 2023 and are now laser-focused on execution across multiple clinical programs,” stated Matt Kapusta, chief executive officer of uniQure. “Our top priorities are to engage with regulatory authorities to clarify the approval pathway for AMT-130 in Huntington’s disease and to initiate patient enrollment across three additional clinical trials as expeditiously as possible.” “While we enter 2024 with a strong balance sheet, we remain disciplined on the prudent and efficient allocation of our capital,” he continued. “Any decision to advance AMT-130 into late-stage development will necessitate a clear and timely approval pathway and financial feasibility, including through a partnership which we would secure before beginning any Phase III trial.” Recent Updates Advancing AMT-130 for the treatment of Huntington’s disease In December 2023, the Company announced updated clinical data from the ongoing U.S. and European Phase I/II studies of AMT-130 for the treatment of early-stage Huntington’s disease. Patients treated with AMT-130 continued to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to a non-concurrent criteria-matched natural history of the disease. When compared to the expected rate of decline from an inclusion criteria-matched natural history data set, AMT-130 showed favorable trends in composite Unified Huntington’s Disease Rating Scale (cUHDRS), Total Functional Capacity (TFC) and Total Motor Score (TMS) key clinical rating scales designed to assess disease progression. Further declines in neurofilament light chain (NfL), an exploratory biomarker for the measurement of neuronal degradation and disease progression were observed among patients treated with AMT-130. Mean NfL in the cerebrospinal fluid (CSL) for low-dose patients remained below baseline at 30 months of follow-up and high-dose patients were near baseline at 18 months. AMT-130 continued to be generally well-tolerated with a manageable safety profile across both doses. In the fourth quarter of 2023, the Company initiated patient dosing in a third cohort of up to 12 patients to further investigate both doses of AMT-130 in combination with perioperative immunosuppression, with a focus on evaluating near-term safety and tolerability. Enrollment in this cohort is expected to be completed in the second half of 2024. In the second quarter of 2024, the Company expects to initiate regulatory interactions with the U.S. Food and Drug Administration (FDA) to discuss data from the ongoing Phase I/II studies and potential strategies for the further development of AMT-130. By the end of 2024, the Company expects to have greater clarity regarding a potential approval pathway for AMT-130.In mid-2024, the Company expects to provide an interim update from the ongoing Phase I/II studies of AMT-130, including up to 24- and 36-month follow-up data from all treated patients in the U.S. and European trials. Advancing additional programs into the clinic AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (rMTLE) – In September 2023, the Company announced the clearance of an IND for the Phase I/IIa clinical study of AMT-260. Site initiation is underway, and patient enrollment is expected to begin in the first half of 2024.AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) - In January 2023, the Company entered into a global licensing agreement with Apic Bio for ABP-102, now known as AMT-162, for the treatment of superoxide dismutase 1 (SOD1) ALS, a rare, genetic form of ALS. Patient enrollment in a Phase I/II clinical trial is expected to begin in the first half of 2024.AMT-191 for the treatment of Fabry disease – In November 2023, the Company announced the clearance of an IND for the Phase I/IIa clinical study of AMT-191. Patient enrollment is expected to begin in the first half of 2024. Generating value from the commercial launch of HEMGENIX® The Company continues to provide commercial supply of etranacogene dezaparvovec (HEMGENIX®) to its partner CSL Behring and manufactures the product at its cGMP facility in Lexington, MA. The Company is one of a select number of gene therapy companies with a qualified facility producing routine commercial manufacturing for the market. Upcoming Investor Events 44th Annual Cowen Health Care Conference, March 5, 2024 – Boston, MALeerink Global Biopharma Conference 2024, March 12, 2024 – Miami, FLUBS Virtual CNS Day, March 18, 2024 Financial Highlights Cash position: As of December 31, 2023, the Company held cash and cash equivalents and investment securities of $617.9 million, compared to $392.8 million as of December 31, 2022. The Company entered into a royalty agreement in May 2023 and received an upfront payment of $375.0 million, and collected $100.0 million in July 2023 for a milestone due from CSL following the first sale of HEMGENIX® in the U.S. The Company expects cash, cash equivalents and investment securities will fund operations into the second quarter of 2027. Revenues: Revenue for the year ended December 31, 2023 was $15.8 million, compared to $106.5 million in the same period in 2022. Revenues in 2022 included $100.0 million of license revenue related to the U.S. first sale milestone payment of HEMGENIX® that the Company expected to receive in 2023. Revenue from contract manufacturing HEMGENIX® for CSL increased $9.1 million in 2023 compared to 2022. Cost of contract manufacturing revenues: Cost of contract manufacturing revenues were $13.6 million for the year ended December 31, 2023, compared to $2.1 million for the same period in 2022. The increase relates to an increase in activities related to contract manufacturing HEMGENIX® for CSL. R&D expenses: Research and development expenses were $214.9 million for the year ended December 31, 2023, compared to $197.6 million during the same period in 2022. The $17.2 million increase was primarily related to a $5.9 million net increase of external research and development expenses including a $10.0 million payment made to Apic Bio to acquire AMT-162, an $8.8 million non-cash increase related to the fair value of contingent consideration associated with the Company’s acquisition of Corlieve Therapeutics in 2021 as well as a $1.4 million non-cash impairment loss related to our Lexington research facility. SG&A expenses: Selling, general and administrative expenses were $74.6 million for the year ended December 31, 2023, compared to $55.1 million during the same period in 2022. The $19.5 million increase was primarily related to a $9.7 million increase of professional, financial advisory and intellectual property fees, a $3.7 million increase in information technology expenses as well as a $3.7 million increase in personnel and contractor-related expenses. Other non-operating items, net: Other non-operating items, net was an expense of $23.7 million for the year ended December 31, 2023, compared to net income of $14.9 million for the same period in 2022. The $38.6 million decrease in other non-operating items, net was primarily related to a decrease in foreign currency gains, net of $24.9 million and an increase in non-cash interest expense of $26.9 million related to the royalty agreement that the Company entered into in May 2023, which partially was offset by an increase of $19.0 million in interest income earned on investment securities and cash on hand. Net loss:The net loss for the year ended December 31, 2023, was $308.5 million, or $6.47 basic and diluted loss per ordinary share, compared to $126.8 million net loss for the same period in 2022, or $2.71 basic and diluted loss per ordinary share. About uniQure uniQure’s mission is to reimagine the future of medicine by delivering innovative cures that transform lives. The recent approvals of our gene therapy for hemophilia B – a historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. We are now leveraging our modular and validated technology and manufacturing platform to advance a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory mesial temporal lobe epilepsy, amyotrophic lateral sclerosis (ALS), Fabry disease, and other severe diseases. www.uniQure.com uniQure Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," “establish,” "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," “seek,” "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning the Company’s cash runway and its ability to fund its operations into the second quarter of 2027; the Company’s expected cost savings related to its strategic reorganization; the Company’s plans to announce additional follow up data from the ongoing U.S. and European Phase I/II clinical studies of AMT-130; the Company’s plans to initiate interactions with regulatory authorities regarding the further development of AMT-130 and the timing of regulatory clarity from such interactions; and the Company’s plans to initiate patient enrollment for AMT-260, AMT-162 and AMT-191 in the first half of 2024. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the clinical results and the development and timing of the Company’s programs; the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to approval; the Company’s ability to continue to build and maintain the company infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company’s ability to obtain, maintain and protect intellectual property; the Company’s ability to fund its operations and to raise additional capital as needed; and the impact of global economic uncertainty, rising inflation, rising interest rates or market disruptions on its business. These risks and uncertainties are more fully described under the heading "Risk Factors" in the Company’s periodic filings with the U.S. Securities & Exchange Commission (“SEC”), including its Annual Report on Form 10-K filed February 28, 2024 and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future. uniQure Contacts: FOR INVESTORS: FOR MEDIA: Maria E. CantorChiara RussoTom MaloneDirect: 339-970-7536Direct: 617-306-9137Direct: 339-970-7558Mobile: 617-680-9452Mobile: 617-306-9137Mobile: 339-223-8541m.cantor@uniQure.comc.russo@uniQure.comt.malone@uniQure.com uniQure N.V.UNAUDITED CONSOLIDATED BALANCE SHEETS December 31, December 31, 2023 2022 (in thousands, except share and per share amounts) Current assets Cash and cash equivalents$241,360 $228,012 Current investment securities 376,532 124,831 Accounts receivable and contract asset 4,193 102,376 Inventories, net 12,024 6,924 Prepaid expenses 15,089 11,817 Other current assets and receivables 2,655 2,814 Total current assets 651,853 476,774 Non-current assets Property, plant and equipment, net 46,548 50,532 Non-current investment securities — 39,984 Operating lease right-of-use assets 28,789 32,726 Intangible assets, net 60,481 58,778 Goodwill 26,379 25,581 Deferred tax assets, net 12,276 14,528 Other non-current assets 5,363 6,061 Total non-current assets 179,836 228,190 Total assets$ 831,689 $ 704,964 Current liabilities Accounts payable$6,586 $10,984 Accrued expenses and other current liabilities$30,534 $30,571 Current portion of contingent consideration 28,211 25,982 Current portion of operating lease liabilities 8,344 8,382 Total current liabilities 73,675 75,919 Non-current liabilities Long-term debt 101,749 102,791 Liability from royalty financing agreement 394,241 — Operating lease liabilities, net of current portion 28,316 31,719 Contingent consideration, net of current portion 14,795 9,334 Deferred tax liability, net 7,543 8,257 Other non-current liabilities 3,700 935 Total non-current liabilities 550,344 153,036 Total liabilities 624,019 228,955 Shareholders' equity Total shareholders' equity 207,670 476,009 Total liabilities and shareholders' equity$ 831,689 $ 704,964 Balancing check - - uniQure N.V.UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS Years ended December 31, 2023 2022 2021 (in thousands, except share and per share amounts)License revenues$2,758 $100,000 $517,400 Contract manufacturing revenues 10,835 1,717 - Collaboration revenues 2,250 4,766 6,602 Total revenues 15,843 106,483 524,002 Operating expenses: Cost of license revenues (65) (1,254) (24,976)Cost of contract manufacturing revenues (13,563) (2,089) - Research and development expenses (214,864) (197,591) (143,548)Selling, general and administrative expenses (74,591) (55,059) (56,290)Total operating expenses (303,083) (255,993) (224,814)Other income 6,059 7,171 12,306 Other expense (1,690) (820) (876)(Loss) / income from operations (282,871) (143,159) 310,618 Non-operating items, net (23,686) 14,900 22,188 (Loss) / income before income tax (expense) / benefit$ (306,557) $ (128,259) $ 332,806 Income tax (expense) / benefit (1,921) 1,470 (3,217)Net (loss) / income$ (308,478) $ (126,789) $ 329,589 Earnings per ordinary share - basic Basic net (loss) / income per ordinary share$(6.47) $(2.71) $7.17 Earnings per ordinary share - diluted Diluted net (loss) / income per ordinary share$(6.47) $(2.71) $7.04 Weighted average shares - basic 47,670,986 46,735,045 45,986,467 Weighted average shares - diluted 47,670,986 46,735,045 46,840,972