Etranacogene dezaparvovec

~ Achieved alignment with the FDA on the AMT-130 statistical analysis plan and CMC requirements to support a planned BLA submission in the first quarter of 2026; ~ Company expects to present AMT-130 topline three-year data in September 2025 ~ ~ Presented case study from first participant treated with AMT-260 for refractory mesial temporal lobe epilepsy showing 92% seizure reduction with no serious adverse events through first five months of follow up ~ ~ Appointment of Kylie O’Keefe as Chief Customer and Strategy Officer ~ ~ Cash, cash equivalents and current investment securities of approximately $377.0 million as of June 30, 2025 are expected to fund operations into second half of 2027 ~ ~ uniQure to host earnings call at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, July 29, 2025 (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 second quarter of 2025 and highlighted recent progress across its business. “We delivered tremendous progress across our business in the first half of 2025, setting the stage for a transformative period ahead for uniQure,” said Matt Kapusta, chief executive officer of uniQure. “With alignment from the FDA on a biological license application (BLA) pathway and pivotal topline data expected in September, AMT-130 is well-positioned to potentially become the first disease-modifying therapy for people living with Huntington’s disease.” “At the same time, we’re encouraged by early clinical signals from the first patient treated with AMT-260 in refractory mesial temporal lobe epilepsy and the continued advancement of our Fabry and ALS programs,” continued Mr. Kapusta. “Backed by a strong balance sheet and an exceptional team, we believe that we are well prepared to execute on the opportunities ahead, including the planned commercialization of AMT-130, and delivering lasting value to patients and shareholders.” Recent Company Developments and Updates Alignment with FDA continues to support Accelerated Approval pathway for AMT-130 In June 2025, the Company announced its alignment with the FDA on the statistical analysis plan (SAP) and Chemistry, Manufacturing and Controls (CMC) requirements to support a planned Biologics License Application (BLA) submission for AMT-130 in the first quarter of 2026. The primary efficacy analysis will evaluate the three-year change in the composite Unified Huntington’s Disease Rating Scale (cUHDRS) in patients receiving the high dose of AMT-130 compared to patients in a propensity score-matched external control arm derived from the ENROLL-HD data set. Certain sensitivity analyses, including one using a propensity score-weighted methodology, will also be provided. The FDA also agreed to uniQure’s approach of leveraging prior knowledge and manufacturing experience from the HEMGENIX® process, supplemented with additional full-scale Good Manufacturing Practices (GMP) batches and a single Process Performance Qualification (PPQ) run.In July 2025, the Company submitted the final SAP to the FDA. Additionally, the Company initiated its PPQ campaign to support the planned BLA submission.In May 2025, the Company announced key safety observations from the third cohort of the Phase I/II study evaluating the impact of prophylactic immunosuppression in 12 patients receiving AMT-130. AMT-130 was generally well-tolerated at both doses with no treatment-related serious adverse events. Three serious adverse events related to immunosuppression were observed and resolved with supportive care. The Company expects a short-term steroid regimen will be incorporated as a prophylactic immunosuppression strategy.In April 2025, the FDA granted Breakthrough Therapy designation to AMT-130 based on clinical evidence from Phase I/II trials showing the potential for slowing progression of Huntington’s disease.The Company continues to prepare for a BLA submission of AMT-130 in the treatment of Huntington’s disease. Certain next steps and expected timing include: September 2025: Present topline, three-year data from the ongoing Phase I/II trials.Q3 2025: Initiate a fourth cohort evaluating high-dose AMT-130 in six patients with lower striatal volumes compared to those of patients enrolled in previous cohorts.Q4 2025: Hold pre-BLA meeting with FDA and complete PPQ campaign.Q1 2026: Submit BLA with a request for priority review designation. Advancing additional clinical programs to proof-of-concept AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE) – In May 2025, the Company presented initial safety and exploratory efficacy data from the first treated patient showing a 92% reduction in seizure frequency observed through the first five months of follow up with no serious safety events. The 14 clinical sites in the U.S. continue to screen patients for this study. AMT-191 for the treatment of Fabry disease – The Company continues to enroll patients in the Phase I/IIa study and expects to present initial safety and exploratory efficacy data at the 15th ICIEM 2025 in Kyoto, Japan on Friday, September 5, 2025.AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) – The Company continues to enroll patients in the Phase I/II EPISOD1 study and expects to present initial data in the first half of 2026. Strengthening the leadership team In June 2025, Kylie O’Keefe was appointed as Chief Customer and Strategy Officer, responsible for leading all commercial and medical affairs at uniQure. Ms. O’Keefe brings decades of commercial experience to the company having most recently served as Chief Commercial Officer at PTC Therapeutics, where she led global commercial strategy, operations and portfolio management for multiple rare neurology and metabolic commercial products across more than 50 countries. Strong financial position As of June 30, 2025, the Company had cash, cash equivalents and investment securities of $377.0 million. The Company expects that cash, cash equivalents and investment securities will be sufficient to fund operations into the second half of 2027, including the launch of AMT-130. Financial Highlights Cash position: As of June 30, 2025, the Company held cash, cash equivalents and current investment securities of $377.0 million, compared to $367.5 million as of December 31, 2024. The net increase in cash was primarily related to net proceeds of $80.5 million from the follow-on offering in January and February 2025. Based on the Company’s current operating plan, including the planned U.S. launch of AMT-130, the Company expects cash, cash equivalents and current investment securities will be sufficient to fund operations into the second half of 2027. Revenues: Revenue for the three months ended June 30, 2025 was $5.3 million, compared to $11.1 million in the same period in 2024. The decrease of $5.8 million in revenue resulted from a $3.4 million increase in license revenue, a decrease of $7.1 million from collaboration revenue, and a decrease of $2.1 million from contract manufacturing of HEMGENIX® for CSL Behring. Following the divestment of the Lexington facility in July 2024, revenue from contract manufacturing is recorded net of cost within other expenses. Cost of contract manufacturing revenues: Cost of contract manufacturing revenues were nil for the three months ended June 30, 2025, compared to $7.2 million for the same period in 2024. Following the divestment of the Lexington facility in July 2024, cost of contract manufacturing is recorded net of revenue within other expenses. R&D expenses: Research and development expenses were $35.4 million for the three months ended June 30, 2025, compared to $33.7 million during the same period in 2024. The $1.7 million increase was related to a net increase of $6.3 million in external program spend and $4.0 million higher expenses related to an increase in the fair value of contingent consideration. This was offset by a decrease of $4.7 million in employee-related expenses, a decrease of $2.1 million in facility expenses and a $1.8 million decrease in costs related to preclinical supplies and other expenses. SG&A expenses: Selling, general and administrative expenses were $13.5 million for the three months ended June 30, 2025, compared to $15.8 million during the same period in 2024. The $2.3 million decrease was primarily related to a $1.6 million decrease in employee-related expenses and a $0.6 million decrease in professional fees compared to the prior year period. Other expense: Other expense was $2.2 million for the three months ended June 30, 2025, compared to $0.2 million during the same period in 2024. The increase was primarily related to $1.4 million of non-cash expense recognized to amortize the right to purchase HEMGENIX® from Genezen on favorable terms. Other non-operating items, net: Other non-operating items, net was an income of $6.6 million for the three months ended June 30, 2025, compared to an expense of $11.3 million for the same period in 2024. The $17.9 million increase in other non-operating items, net was primarily related to an increase in net foreign currency gains of $19.6 million, a decrease in interest income of $2.3 million and a reduction of interest expense of $0.6 million due to the $50.0 million repayment of Hercules debt in July 2024. Net loss: The net loss for the three months ending June 30, 2025, was $37.7 million, or $0.69 basic and diluted loss per ordinary share, compared to a $56.3 million net loss for the same period in 2024, or $1.16 basic and diluted loss per ordinary share. Investor Conference Call and Webcast Information uniQure management will host an investor conference call and webcast today, Tuesday, July 29 at 8:30 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure’s website at https://www.uniqure.com/investors-media/events-presentations, and following the event a replay will be archived for 90 days. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. If you are joining the conference call, please dial in 15 minutes before the start time. About uniQure uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure’s gene therapy for hemophilia B – an 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. uniQure is now advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, 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 half of 2027; the timing and outcome of regulatory interactions with respect to the AMT-130 program, FDA’s support for the program and the Company’s plans to provide further regulatory updates, including the timing of its planned BLA submission and anticipated request for priority review designation; the Company’s plans to incorporate a course of steroids as an immunosuppression strategy for patients receiving AMT-130; the Company’s plans to announce AMT-130 topline three-year data in September 2025; the initiation of a fourth cohort evaluating high-dose AMT-130; the Company’s commercialization plans, including with respect to AMT-130; and the Company’s plans for further clinical updates and plans to announce initial data in its AMT-191 and AMT-162 programs. 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 and timing for regulatory 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; and the Company’s ability to fund its operations and to raise additional capital as needed. 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 with the SEC on February 27, 2025, its Quarterly Report on Form 10-Q filed with the SEC on May 9, 2025, 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: Chiara Russo Tom MaloneDirect: 781-491-4371 Direct: 339-970-7558Mobile: 617-306-9137Mobile:339-223-8541 c.russo@uniQure.com t.malone@uniQure.com uniQure N.V.UNAUDITED CONSOLIDATED BALANCE SHEETS June 30, December 31, 2025 2024 (in thousands, except share and per share amounts)Current assets Cash and cash equivalents $253,778 $158,930 Current investment securities 123,196 208,591 Accounts receivable 5,606 5,881 Prepaid expenses 18,538 9,281 Other current assets and receivables 7,455 7,606 Total current assets 408,573 390,289 Non-current assets Property, plant and equipment, net 17,492 20,424 Other investments 29,735 27,464 Operating lease right-of-use assets 13,635 13,647 Intangible assets, net 75,604 71,043 Goodwill 25,340 22,414 Deferred tax assets, net 9,017 9,856 Other non-current assets 5,494 1,399 Total non-current assets 176,317 166,247 Total assets $ 584,890 $ 556,536 Current liabilities Accounts payable $5,541 $7,227 Accrued expenses and other current liabilities 31,479 29,225 Current portion of operating lease liabilities 3,909 3,601 Total current liabilities 40,929 40,053 Non-current liabilities Long-term debt 51,842 51,324 Liability from royalty financing agreement 455,259 434,930 Operating lease liabilities, net of current portion 10,946 11,136 Contingent consideration, net of current portion 15,931 10,860 Deferred tax liability, net 7,963 7,043 Other non-current liabilities 6,023 7,942 Total non-current liabilities 547,964 523,235 Total liabilities 588,893 563,288 Shareholders' (deficit) / equity Total shareholders' (deficit) / equity (4,003) (6,752)Total liabilities and shareholders' (deficit) / equity $ 584,890 $ 556,536 uniQure N.V. UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS Three months ended June 30, 2025 2024 (in thousands, except share and per share amounts) Total revenues $ 5,262 $ 11,126 Operating expenses: Cost of license revenues (656) (234)Cost of contract manufacturing revenues - (7,227)Research and development expenses (35,383) (33,655)Selling, general and administrative expenses (13,500) (15,767)Total operating expenses (49,539) (56,883)Other income 2,597 1,983 Other expense (2,185) (236)Loss from operations (43,865) (44,010)Non-operating items, net 6,571 (11,341)Loss before income tax (expense) / benefit $ (37,294) $ (55,351)Income tax (expense) / benefit (425) (948)Net loss $ (37,719) $ (56,299) Basic and diluted net loss per ordinary share $(0.69) $(1.16)Weighted average shares used in computing basic and diluted net loss per ordinary share 54,807,967 48,622,440

1、新闻事件：2、定价和市场预测（国内及国际类似产品市场表现类比）3、观点1、新闻消息：2025年7月23日，多家媒体报道，国产创新药公司信念医药的AAV基因治疗药物波哌达可基注射液（商品名：信玖凝，研发代号：BBM-H901）定价出炉，每瓶定价9.3万元。根据说明书，该基因疗法的具体用药量，将按照患者体重计算，例如70kg体重的处方用量是44瓶，总治疗费用高达409.2万元。目前，信念医药已经发起慈善援助项目，在该项目支持下，患者用药如果超过30瓶，则只需要支付30瓶的费用，超过30瓶的部分可以获得免费赠药。但是即使如此，患者的自费部分仍然需要279万人民币。2、关于定价和销售预测关于定价：在产品上市时，增进做过预测，预计亲民点BBM-H901定价在70-80万/年，加上医保或者赠药等，价格应该差不多合理。这样每年最终能覆盖到500人，每年能有个3-5亿收入，应该是很不错了。5年一个循环（估计应该能持续有效5年吧），要是持续到8年，那就很不好意思了，5年后说不定就没人用了（有钱的都用了，还在有效期，剩下的就是没钱的）~~~这个价格比当时预测的高了4倍。CAR-T ，复星凯特120万定价，每年160张左右处方。罗氏的艾美赛珠单抗在国内费用是130万/年，2023年国内销售额只有2000万左右，BBM-H901会有多少？10张、50张，还是100张？再看看国际上其他产品表现：其他已经上市的三款血友病基因治疗均是惨淡经营。但是标价确实全球最贵：Roctavian标价290万美元，Hemgenix和Beqvez标价均为350万美元。如此高的标价下CSL的Hemgenix，2022年上市，销售额一直是个谜，在网上看到几个数据，年销售额超过了10亿美元（无法证明真伪，就简单一说）。Roctavian也是2022年上市，2023年买了360万美元，在2024年卖了2600万美元，这个和当初的预测相差甚远。根据标价推算，意味着24年只有十个患者使用了该药；23年，那只有可怜1个人；至于，Beqvez在临床试验外，实在是没有患者使用。2025年初，辉瑞宣布停止血友病B基因疗法Beqvez的全球开发和商业化，彻底放弃了该产品~。其他有意思的血友病产品可以看看下面这篇文章信念 血友病B基因治疗药物申报上市，定价预测，代表产品简述3、观点这个定价是超出了杂评君自己的预测，生产成本应该是一个不可忽视的因素。血友病B换着在国内 登记人数估计四五千人的样子。很关键一点事，血友病患者，觉大多是原生家庭条件都不好，因为这个病会影响患者的就业和生活，对家庭拖累很大。所以，这个价格应该是更有象征性意义，不会真的依靠中国市场就能赚到多少钱~出海估计也难，在美国的几个产品表现也不尽人意~但是从另一方面，还是比美国的同类药物还是便宜很多很多在海南这个地方，或者其他地方，吸引美国患者来国内治疗呢~有没有机会？这里不太熟悉跨国医保或者保险的问题，有熟悉的可以留言讨论。关于血友病介绍，下面这篇文章有一些综述，有兴趣的可以点开看看信念血友病基因治疗药物上市，狂欢之后，市场表现会如何？信念 血友病B基因治疗药物申报上市，定价预测，代表产品简述血友病领域研发全景-行业调研anti-TFPI血友病新药国内报上市，简述TFPI差异化作用机制及血友病也可以关注下知识星球，有分享一篇综述，不过是大概一年前整理的了下面是我的咨询业务，欢迎多来勾搭~~