UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands (State or other jurisdi ...

Financial Performance - Total revenues for Q1 2021 were $454,000, a decrease of 56.3% compared to $1,040,000 in Q1 2020[17] - Net loss for Q1 2021 was $41,556,000, compared to a net loss of $27,999,000 in Q1 2020, representing a 48.3% increase in losses[17] - Basic and diluted net loss per ordinary share for Q1 2021 was $0.91, compared to $0.63 in Q1 2020[17] - Total revenues for the three months ended March 31, 2021, were $454,000, compared to $104,000 for the same period in 2020, representing a 336% increase[127] - Collaboration revenue increased to $454,000 in Q1 2021 from $57,000 in Q1 2020, a growth of 696%[128] - The net loss for the three months ended March 31, 2021, was $41.6 million, up from a net loss of $28.0 million in the same period of 2020, indicating higher operational costs[107] Expenses - Research and development expenses increased to $32,656,000 in Q1 2021, up 25.5% from $26,013,000 in Q1 2020[17] - Selling, general and administrative expenses increased to $12.4 million in Q1 2021 from $9.1 million in Q1 2020, reflecting a 36.3% rise[139] - Personnel and contractor-related expenses increased to $11.6 million in Q1 2021 from $9.3 million in Q1 2020, a rise of 24.6%[142] - The company incurred $2.7 million in share-based compensation expenses in Q1 2021, compared to $2.4 million in Q1 2020, marking a 12.5% increase[142] - Share-based compensation expense for the three months ended March 31, 2021 was $5.754 million, an increase from $4.340 million in the same period in 2020[62] Cash and Liquidity - Cash and cash equivalents at the end of Q1 2021 were $260,813,000, an increase of 6.3% from $244,932,000 at the end of 2020[14] - As of March 31, 2021, cash and cash equivalents totaled $260.8 million, an increase from $244.9 million as of December 31, 2020[107] - The company expects cash and cash equivalents, along with a $100 million facility, to fund operations until the end of 2022, with sufficient funds projected into the second half of 2024 following a $450 million payment received on May 7, 2021[146] - The company expects to fund its operating expenses and capital expenditure requirements into the second half of 2024 with existing cash and the $100.0 million 2021 Amended Facility[163] Assets and Liabilities - Total assets as of March 31, 2021, were $363,853,000, up 6.9% from $340,395,000 at the end of 2020[14] - Total liabilities increased to $135,163,000 as of March 31, 2021, compared to $96,490,000 at the end of 2020, marking an increase of 40.1%[14] - Shareholders' equity decreased to $228,690,000 as of March 31, 2021, down 6.2% from $243,905,000 at the end of 2020[14] - Total contractual obligations as of March 31, 2021, amounted to $144.1 million, including debt obligations and operating lease obligations[165] Clinical Trials and Development - The pivotal HOPE-B trial for the lead gene therapy candidate, Etranacogene dezaparvovec, showed a mean FIX activity increase from ≤ 2% to 37.2% at 26 weeks, meeting the primary endpoint[90] - During the 26-week follow-up, there was an 83% reduction in total bleeding events compared to the observational lead-in phase, with only three spontaneous bleeds requiring treatment[92] - The Phase 1b/II clinical trial for AMT-130 in Europe is expected to begin enrolling patients in the second half of 2021, aiming to establish safety and optimal dosing[99] - The company is responsible for completing the HOPE-B clinical trial and manufacturing process validation for the product under the CSL Behring Agreement[36] - The company plans to incorporate FIX activity and bleeding rates at 52 weeks as additional co-primary endpoints in the HOPE-B study following interactions with the FDA and EMA[92] Regulatory and Collaboration Agreements - The company entered into a collaboration agreement with CSL Behring, receiving an upfront cash payment of $450 million and potential additional payments of up to $1.6 billion based on regulatory and commercial milestones[35] - The company did not recognize any revenue related to the CSL Behring License Revenue due to contingent payments not being fulfilled[38] - The collaboration with Bristol-Myers Squibb (BMS) allows the company to receive up to $217 million in milestone payments for each of the four Collaboration Targets[42] - The company did not record any License Revenue during the three months ended March 31, 2021, due to significant uncertainty surrounding the development of gene-therapy product candidates[46] Risks and Challenges - The ongoing COVID-19 pandemic has materially affected the company's operations, causing disruptions in raw material supply and clinical trial processes[186] - None of the company's product candidates have been approved for commercial sale, and significant revenue generation is not expected before 2022[188] - Clinical trials are subject to delays and may not demonstrate the safety and efficacy of product candidates, impacting future development[191] - The company may need to raise additional funding, which could be difficult to obtain on acceptable terms, impacting product development efforts[182] - The company may face challenges in enrolling eligible patients for clinical trials due to the rarity of the diseases targeted, potentially delaying or abandoning trials[197]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands (Jurisdiction of incorpor ...

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| --- | --- | --- | --- | --- | |-------------------------------------------|-------|-------|-------|-------| | | | | | | | | | | | | | | | | | | | | | | | | | Global License Agreement in Hemophilia B | | | | | | June 24, 2020 | | | | | | | | | | | | | | | | | Forward-looking Statements A G L O B A L L E A D E R I N G E N E T H E R A P Y J A N U A R Y 2 0 2 0 | 2 This presentation contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, whi ...

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uniQure Delivering Gene Therapy to Patients Corporate Presentation November 2019 D E L I V E R I N G G E N E T H E R A P Y T O P A T I E N T S N O V E M B E R 2 0 1 9 | 2 Forward-looking Statements This presentation 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," "estimate," "expect," "goal," "intend," "look forward to," "may," "plan," "potential," "predic ...