Apic Bio, Inc.

~ 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

~ Announced FDA clearance of Investigational New Drug (IND) application for AMT-260 in refractory mesial temporal lobe epilepsy ~ ~ On track to provide clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington’s disease later in the fourth quarter of 2023 ~ ~ uniQure and CSL win 2023 Prix Galien USA award for HEMGENIX® ~ ~ Announced strategic reorganization to focus on advancing multiple clinical-stage programs and expect to deliver $180 million of cost savings over the next three years ~ ~ Strong cash position of approximately $660 million as of September 30, 2023 expected to fund operations into the second quarter of 2027 ~ LEXINGTON, Mass. and AMSTERDAM, Nov. 07, 2023 (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 third quarter of 2023 and highlighted recent progress across its business. “In the third quarter we made significant progress towards our goal of advancing multiple clinical-stage programs and focusing resources to generate value from these important investments,” stated Matt Kapusta, chief executive officer of uniQure. “We are very pleased to have achieved FDA clearance of the IND for AMT-260 in refractory mesial temporal lobe epilepsy and look forward to initiating patient enrollment in the Phase I/IIa study as soon as possible. In addition, we are making good progress toward initiating patient screening for a Phase I/II trial of AMT-162 in SOD1-ALS and the submission of an IND for AMT-191 in Fabry disease. We also look forward to providing another clinical update later this quarter from our ongoing Phase I/II studies of AMT-130 in Huntington’s disease, which will include up to 2.5 years of patient follow-up.” “As we prepare for 2024, we remain laser focused on execution across our portfolio of gene therapy product candidates with the goal of achieving clinical proof-of-concept as rapidly as possible. We also remain committed to carefully managing costs, prudently allocating capital, and thoughtfully evaluating strategies that can enhance value for shareholders in an increasingly challenging market environment.” Recent Updates Advancing AMT-130 for the treatment of Huntington’s disease Later in the fourth quarter of 2023, the Company plans to provide a clinical update from the U.S. and European Phase I/II studies of AMT-130, including 18- and 30-month follow-up data from the treated patients in the high- and low-dose U.S. cohorts, respectively. The data update will include safety and tolerability, biomarker, imaging and functional data across both high and low-dose cohorts.A total of 33 patients have now been treated with AMT-130 across two dose cohorts in the U.S. and EU clinical trials. The Company recently initiated patient screening for a third cohort in up to 12 patients to further investigate both doses in combination with perioperative immunosuppression, with a focus on evaluating near-term safety and tolerability.In the first quarter of 2024, the Company plans to request regulatory interactions with the Food and Drug Administration (FDA) to discuss the U.S. and EU data and potential late-stage development pathways for AMT-130. Advancing additional programs into the clinic AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (rMTLE) – In the third quarter of 2023, the Company announced the clearance of an investigational new drug (IND) for the Phase I/IIa clinical study of AMT-260. Screening and patient enrollment is expected to begin in the fourth quarter of 2023 with first patient dosing planned to occur in the first quarter 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 AMT-162 for the treatment of superoxide dismutase 1 (SOD1) ALS, a rare, genetic form of ALS. The Company expects to initiate patient screening in the fourth quarter of 2023 with first patient dosing planned to occur in the first quarter of 2024.AMT-191 for the treatment of Fabry disease – The Company expects to submit an IND in the fourth quarter of 2023 and to begin patient dosing in 2024. Supporting the commercial launch of HEMGENIX® in the United States and European Union In October 2023, uniQure and CSL Behring LLC (“CSL”) announced that the companies received the 2023 Prix Galien USA Award in the category of Best Product for Rare/Orphan Diseases for HEMGENIX®. HEMGENIX® was one of several product nominations in this category. Created in 1970, the Prix Galien Awards recognizes outstanding innovation and scientific advancement and is regarded as the equivalent of the Nobel Prize for the life science industry.In July 2023, uniQure received a $100 million milestone payment from CSL associated with the first commercial sale of HEMGENIX® in the United States. Implementing strategic reorganization focused on advancing multiple clinical-stage programs, achieving cost savings, and extending cash runway In October 2023, the Company announced a strategic reorganization plan which included a reduction of 28% of the workforce not related to manufacturing HEMGENIX®, the discontinuation of more than half of the research and technology projects, and a focus on executing across four clinical-stage programs. The Company expects a total cost savings of approximately $180 million over the next three years, which is expected to extend cash runway into the second quarter of 2027.As part of the strategic reorganization and the significant reduction in research activities, Ricardo Dolmetsch, Ph.D., the Company’s former chief scientific officer, departed the Company and will remain as a scientific consultant through the end of the year. Richard Porter, Ph.D., assumed responsibilities for research, as well as non-clinical and vector development in his new role as Chief Business and Scientific Officer. Dr. Porter has more than 25 years of neuroscience leadership in the biopharma industry and joined uniQure in June 2021 through the acquisition of uniQure France SAS (formerly, Corlieve Therapeutics), where he was founder and chief executive officer. Upcoming Investor Events Stifel 2023 Healthcare Conference, November 14, 2023 – New York, NY Financial Highlights Cash position: As of September 30, 2023, the Company held cash and cash equivalents and investment securities of $658.9 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. Revenues: Revenues for the three months ended September 30, 2023 were $1.4 million, compared to $1.4 million in the same period in 2022. The current period included an increase in license revenues of $0.5 million and contract manufacturing revenues of $0.4 million related to contract manufacturing HEMGENIX® for CSL, and a decrease of $0.9 million in collaboration revenues. R&D expenses: Research and development expenses were $65.4 million for the three months ended September 30, 2023, compared to $48.1 million in the same period in 2022. The change was primarily related to a non-cash, $8.8 million increase in the fair value of contingent consideration associated with the Company’s acquisition of Corlieve Therapeutics in 2021. SG&A expenses: Selling, general and administrative expenses were $18.1 million for the three months ended September 30, 2023, compared to $13.3 million in the same period in 2022. The increase was primarily related to an increase in personnel and contractor-related expenses, and in legal fees associated with various corporate initiatives. Other non-operating items, net: Other non-operating items, net was an expense of $7.8 million for the three months ended September 30, 2023, compared to net income of $11.3 million for the same period in 2022. The decrease in other non-operating items, net was primarily related to a decrease in foreign currency gains, net of $14.2 million and an increase in interest expense of $12.4 million primarily related to the royalty agreement that the Company entered into in May 2023, which partially was offset by an increase of $7.5 million in interest income earned on investment securities and cash on hand. Net loss:The net loss for the three months ended September 30, 2023, was $89.6 million, or $1.88 basic and diluted loss per ordinary share, compared to $47.9 million net loss for the same period in 2022, or $1.02 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. These forward-looking statements include, but are not limited to, the Company’s statements about its cash runway and its ability to fund operations into 2027, the Company’s cost savings related to the recently-announced strategic organization, whether there will be continued progress on the commercialization of HEMGENIX®, the Company’s plans to announce follow up data from the U.S. and European Phase I/II clinical study of AMT-130 in 2023, whether that data will help to further guide our ongoing clinical development of AMT-130, the Company’s expectation to submit an investigational new drug application for AMT-191 in Fabry disease in 2023 and begin patient dosing in 2024, the Company’s expectation to initiate patient screening in AMT-162 and AMT-260 in the fourth quarter of 2023 and begin patient dosing in each in the first quarter of 2024, and whether the Company will begin interactions with regulatory agencies in the first quarter of 2024 with respect to AMT-130. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the regulatory approval and commercial launch of HEMGENIX®, material changes to our interim or preliminary data, our clinical trial for Huntington’s disease, the impact of financial and geopolitical events on our Company and the wider economy and health care system, the Company’s ability to raise additional capital to support late stage development of the Company’s clinical program(s) if supported by future data, our Commercialization and License Agreement with CSL Behring, our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims and ongoing litigation, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in the Company’s periodic securities filings, including its Annual Report on Form 10-K filed February 27, 2023 and the Quarterly Report on Form 10-Q filed November 7, 2023. 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 September 30, December 31, 2023 2022 (in thousands, except share and per share amounts)Current assets Cash and cash equivalents $229,484 $228,012 Current investment securities 429,428 124,831 Accounts receivable and contract asset 1,644 102,376 Inventories 15,654 6,924 Prepaid expenses 14,884 11,817 Other current assets and receivables 2,532 2,814 Total current assets 693,626 476,774 Non-current assets Property, plant and equipment, net 45,946 50,532 Non-current investment securities — 39,984 Operating lease right-of-use assets 30,360 32,726 Intangible assets, net 57,976 58,778 Goodwill 25,273 25,581 Deferred tax assets, net 12,351 14,528 Other non-current assets 6,018 6,061 Total non-current assets 177,924 228,190 Total assets $871,550 $704,964 Current liabilities Accounts payable $5,584 $10,984 Accrued expenses and other current liabilities 28,427 30,571 Current portion of contingent consideration 26,708 25,982 Current portion of operating lease liabilities 7,888 8,382 Total current liabilities 68,607 75,919 Non-current liabilities Long-term debt 101,431 102,791 Liability from royalty financing agreement 383,711 — Operating lease liabilities, net of current portion 28,977 31,719 Contingent consideration, net of current portion 14,030 9,334 Deferred tax liability, net 4,917 8,257 Other non-current liabilities 1,093 935 Total non-current liabilities 534,159 153,036 Total liabilities 602,766 228,955 Shareholders' equity Total shareholders' equity 268,784 476,009 Total liabilities and shareholders' equity $871,550 $704,964 uniQure N.V.UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS Three months ended September 30, 2023 2022 (in thousands, except share and per share amounts)Total revenues $1,407 $1,449 Operating expenses: Cost of contract manufacturing revenues (1,006) (861)Research and development expenses (65,400) (48,068)Selling, general and administrative expenses (18,074) (13,324)Total operating expenses (84,480) (62,253)Other income 1,424 1,485 Other expense (228) (199)Loss from operations (81,877) (59,518)Non-operating items, net (7,763) 11,332 Loss before income tax (expense) / benefit $(89,640) $(48,186)Income tax (expense) / benefit 69 329 Net loss $(89,571) $(47,857) Basic and diluted net loss per ordinary share $(1.88) $(1.02)Weighted average shares used in computing basic and diluted net loss per ordinary share 47,770,101 46,772,430