LEXINGTON, Mass. and AMSTERDAM, June 02, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today provided a regulatory update on AMT-130, its investigational gene therapy for the treatment of Huntington’s disease.

列克星敦，马萨诸塞州和阿姆斯特丹，2025年6月2日（GLOBE NEWSWIRE）-- uniQure N.V.（纳斯达克股票代码：QURE），一家领先的基因治疗公司，致力于为有严重医疗需求的患者推进变革性疗法，今天提供了关于其研究性基因疗法AMT-130用于治疗亨廷顿病的监管更新。

Following recent Type B meetings and further guidance from the U.S. Food and Drug Administration (FDA), the Company has reached alignment with the FDA on several key components of the statistical analysis plan and Chemistry, Manufacturing and Controls (CMC) information that will support a Biologics License Application (BLA) submission expected in the first quarter of 2026..

在最近的B类会议和美国食品药品监督管理局（FDA）的进一步指导下，公司已与FDA就统计分析计划和化学、制造与控制（CMC）信息的几个关键部分达成一致，这些内容将支持预计于2026年第一季度提交的生物制品许可申请（BLA）。

“We are very pleased with our continued, productive engagement with the FDA and the progress we’ve made toward a planned BLA submission for AMT-130 in the first quarter of 2026,” said Walid Abi-Saab, M.D., chief medical officer of uniQure. “We are pursuing an accelerated approval pathway supported by multiple years of clinical data – a rigorous and differentiated approach that reflects the urgent need in Huntington’s disease and our commitment to delivering the first disease-modifying treatment for people affected by this devastating disease.

“我们与FDA持续且富有成效的合作感到非常高兴，并且我们为计划在2026年第一季度提交AMT-130的BLA申请所取得的进展感到满意，”uniQure首席医学官Walid Abi-Saab博士表示。“我们正在寻求一条加速审批路径，该路径得到了多年临床数据的支持——这是一种严谨且独特的方法，反映了亨廷顿病的迫切需求，以及我们致力于为受这种毁灭性疾病影响的人群提供首个疾病修饰治疗方案的承诺。”

We are grateful to the FDA for their continued engagement and look forward to sharing three-year top-line data in the third quarter of 2025.” .

我们感谢 FDA 的持续参与，并期待在 2025 年第三季度分享三年期的顶级数据。

Statistical Analysis Plan

统计分析计划

In the second quarter of 2025, the Company held a Type B meeting with the FDA to discuss the proposed use of external control data and the prospectively defined statistical analysis plan (SAP) in support of the planned BLA submission for AMT-130. The FDA continued to support its prior agreement that the composite Unified Huntington’s Disease Rating Scale (cUHDRS) may serve as an acceptable registrational, intermediate clinical endpoint for accelerated approval.

2025年第二季度，公司与FDA举行了一次B类会议，讨论拟议的外部对照数据使用及前瞻性定义的统计分析计划（SAP），以支持计划中的AMT-130的BLA提交。FDA继续支持其先前的共识，即复合统一亨廷顿病评定量表（cUHDRS）可作为加速批准的可接受注册性中间临床终点。

The FDA agreed that the primary efficacy analysis for the BLA will evaluate the 3-year change in cUHDRS in high-dose AMT-130 patients compared to a propensity score-adjusted external control arm. The Company plans to use propensity score-weighted external control derived from the ENROLL-HD dataset for the primary analysis and to submit certain sensitivity analyses, including one using a propensity score-matched external control, as additional support..

FDA同意，针对BLA的主要疗效分析将评估高剂量AMT-130患者与经倾向评分调整的外部对照组相比，cUHDRS在三年内的变化。公司计划使用基于ENROLL-HD数据集的倾向评分加权外部对照进行主要分析，并提交某些敏感性分析作为补充支持，其中包括使用倾向评分匹配的外部对照分析。

The FDA also agreed that ENROLL-HD – a large, prospective, longitudinal, natural history study of patients with Huntington’s disease – may be acceptable as the external control dataset for the primary analysis of the trial data along with additional sensitivity analyses using the TRACK-HD/TRACK-ON and PREDICT-HD datasets.

FDA 还同意，ENROLL-HD——一项大规模、前瞻性、纵向的亨廷顿病患者自然史研究——可以作为试验数据主要分析的外部对照数据集，并结合使用 TRACK-HD/TRACK-ON 和 PREDICT-HD 数据集进行额外的敏感性分析。

To date, approximately 33,000 patients have enrolled in ENROLL-HD. Compared to previously used natural history studies like TRACK-HD and PREDICT-HD, ENROLL-HD offers a substantially larger sample size, lower attrition rates, and longer average patient follow-up. The Company expects the larger sample size to reduce variability in the external control data and enhance the robustness of the SAP..

迄今为止，已有大约33,000名患者注册了ENROLL-HD。与之前使用的自然史研究（如TRACK-HD和PREDICT-HD）相比，ENROLL-HD提供了更大的样本量、更低的流失率以及更长的平均患者随访时间。公司预计更大的样本量将减少外部对照数据的变异性，并增强SAP的稳健性。

The Company plans to submit an updated SAP consistent with discussions from the recently held Type B meeting to the FDA in the second quarter of 2025.

公司计划在2025年第二季度向FDA提交一份与最近举行的B类会议讨论一致的更新版SAP。

Chemistry, Manufacturing and Controls (CMC)

化学、制造和控制 (CMC)

In the first quarter of 2025, the Company held a Type B meeting with the FDA to discuss CMC requirements in support of the planned BLA submission for AMT-130. The FDA agreed that validation of the AMT-130 manufacturing process should be possible using experience and prior knowledge from the etranacogene dezaparvovec-drlb (HEMGENIX.

2025年第一季度，公司与FDA举行了B类会议，讨论支持AMT-130计划BLA提交的CMC要求。FDA同意，应可通过etranacogene dezaparvovec-drlb（HEMGENIX）的经验和已有知识来验证AMT-130生产工艺。

®

®

) process, complemented with additional full-scale AMT-130 GMP batches and a single Process Performance Qualification (PPQ) batch.

）过程，辅以额外的全规模AMT-130 GMP批次和单个工艺性能确认（PPQ）批次。

The FDA also agreed with the Company’s proposed drug product release testing plan, including the proposed potency assay, pending completion of qualification and specification setting activities.

FDA 还同意公司提出的药品放行检测计划，包括拟议的效价测定，等待资格确认和规范设定活动的完成。

Next Steps for the Planned BLA Submission

计划中的BLA提交的下一步

Based on these recent Type B meetings with the FDA, the Company continues to prepare for a BLA submission for AMT-130 in the treatment of Huntington’s disease. Certain key next steps and expected timing include:

基于近期与FDA召开的B类会议，公司将继续准备就AMT-130治疗亨廷顿病提交BLA。某些关键的后续步骤和预期时间包括：

About the Phase I/II Clinical Program of AMT-130

关于AMT-130的I/II期临床项目

uniQure is conducting two multi-center, dose-escalating, Phase I/II clinical studies to explore the safety, tolerability, and exploratory efficacy signals of AMT-130 for the treatment of Huntington’s disease. In the U.S. study, a total of 26 patients with early manifest Huntington’s disease were randomized to treatment (n=6 low dose; n=10 high dose) or an imitation (sham) surgical procedure (n=10).

uniQure正在进行两项多中心、剂量递增的I/II期临床研究，以探索AMT-130治疗亨廷顿病的安全性、耐受性和探索性疗效信号。在美国的研究中，共有26名早期显性亨廷顿病患者被随机分配到治疗组（n=6低剂量；n=10高剂量）或模拟（假）手术程序组（n=10）。

Treated patients received a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). The study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of treated patients for five years.

接受治疗的患者通过MRI引导、对流增强的立体定向神经外科手术，将AMT-130单次直接注入纹状体（尾状核和壳核）。该研究包括一个为期12个月的盲法核心研究阶段，随后对治疗患者进行为期五年的非盲长期随访。

An additional four control patients crossed over to treatment..

另外四名对照组患者转为接受治疗。

The European open-label Phase Ib/II study of AMT-130 enrolled 13 patients with early manifest Huntington’s disease (n=6 low dose; n=7 high dose).

欧洲开放标签的AMT-130 Ib/II期研究招募了13名早期显性亨廷顿病患者（低剂量组n=6；高剂量组n=7）。

A third cohort enrolled an additional 12 patients across sites in the U.S. and EU. This cohort was randomized to explore both doses of AMT-130 in combination with immunosuppression, using the current, established stereotactic administration procedure.

第三组队列在美国和欧盟的多个研究中心额外招募了12名患者。该队列被随机分配，以探索AMT-130的两种剂量与免疫抑制剂联合使用的效果，同时采用当前已确立的立体定向给药程序。

Additional details are available on www.clinicaltrials.gov (NCT0543017, NCT04120493)

更多详细信息可访问 www.clinicaltrials.gov (NCT0543017, NCT04120493)。

AMT-130 has been granted the FDA’s Regenerative Medicine Advance Therapy (RMAT) designation and Breakthrough Therapy designation, the first therapy for Huntington’s disease to receive an RMAT designation.

AMT-130 已获得 FDA 的再生医学先进疗法（RMAT）认定和突破性疗法认定，是首个获得 RMAT 认定的亨廷顿病疗法。

About Huntington’s Disease

关于亨廷顿病

Huntington’s disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain.

亨廷顿病是一种罕见的遗传性神经退行性疾病，会导致包括舞蹈症、行为异常和认知能力下降在内的运动症状，进而引发身体和精神的进行性恶化。该疾病为常染色体显性遗传，由亨廷顿基因第一个外显子中的CAG重复扩展引起，导致脑内异常蛋白的产生和聚集。

According to 2021 study in Neuroepidemiology, approximately 70,000 people have been diagnosed with Huntington’s disease in the U.S. and Europe, with hundreds of thousands of others at risk of inheriting the disease. Despite the clear etiology of Huntington’s disease, there are currently no approved therapies to delay the onset or to slow the disease’s progression..

根据《神经流行病学》2021年的一项研究，美国和欧洲约有7万人被诊断出患有亨廷顿病，还有数十万人面临遗传该病的风险。尽管亨廷顿病的病因明确，但目前尚无获批的疗法能够延缓其发病或减缓疾病进展。

About uniQure

关于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正在兑现基因治疗的承诺——单次治疗可能带来治愈效果。 uniQure针对B型血友病的基因疗法获得批准，这一历史性成就是基于十多年的研究和临床开发，代表了基因组医学领域的重大里程碑，并为血友病患者带来了全新的治疗方式。

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目前正在推进一系列针对亨廷顿病、难治性颞叶癫痫、ALS、法布里病和其他严重疾病的专有基因疗法的管线。www.uniQure.com。

uniQure Forward-Looking Statements

uniQure 前瞻性声明

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. 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 and the negatives of those terms.

本新闻稿包含经修订的1933年《证券法》第27A条和经修订的1934年《证券交易法》第21E条所指的前瞻性陈述。除历史事实陈述外，所有其他陈述均为前瞻性陈述，通常可以通过诸如“预期”、“相信”、“可能”、“建立”、“估计”、“期望”、“目标”、“期待”、“或许”、“计划”、“潜在”、“预测”、“预计”、“寻求”、“应该”、“将”、“会”等词语及其否定形式来识别。

Forward-looking statements are based on management's beliefs and assumptions and on information available to management as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning: the availability of accelerated approval pathways and the need for additional pre-approval studies for AMT-130; the Company’s anticipated timing of the BLA submission; the Company’s plans to submit a revised SAP and CMC information to the FDA; the Company’s ability to deliver a potentially life-changing therapy to people living with Huntington’s disease and related timeline for doing so; the potential clinical and functional effects of AMT-130; the Company’s plans to continue clinical development of AMT-130; the Company’s plans to share clinical data of AMT-130 in the third quarter of 2025; and the utility of the ENROLL-HD patient dataset with respect to Phase I/II study.

前瞻性声明基于管理层的信念和假设，以及管理层在本新闻稿发布之日可获得的信息。这些前瞻性声明的例子包括但不限于以下内容的声明：AMT-130加速审批途径的可用性及需要额外的批准前研究；公司预计提交BLA的时间；公司计划向FDA提交修订的SAP和CMC信息；公司为亨廷顿病患者提供可能改变生命的治疗方案及其相关时间表的能力；AMT-130潜在的临床和功能效果；公司继续AMT-130临床开发的计划；公司计划在2025年第三季度分享AMT-130的临床数据；以及ENROLL-HD患者数据集在I/II期研究中的效用。

The Company may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements. Furthermore, the Company’s actual results could differ materially from the plans, intentions and expectations anticipated in these forward-looking statements f.

公司可能无法实际实现这些前瞻性陈述中披露的计划、意图或预期。此外，公司的实际结果可能与这些前瞻性陈述中预期的计划、意图和期望有很大差异。

later developments with the FDA and other regulators that could be inconsistent with the feedback received to date; and whether regulatory authorities will accept the Company’s approach as a basis for accelerated approval; risks related to the Company’s use of nominal p values as a basis for its statistical analyses; whether the measurements that the Company is evaluating continue to be viewed as robust and sensitive measurements of disease progression; whether RMAT designation or any accelerated pathway, will lead to regulatory approval; the Company’s ability to continue to build and maintain the infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the continued development and acceptance of gene therapies; the Company’s ability to obtain, maintain and protect its intellectual property; and the Company’s ability to fund its operations and to raise additional capital as needed and on acceptable terms.

与FDA及其他监管机构的后续发展可能与迄今为止收到的反馈不一致；监管机构是否会接受公司作为加速审批依据的方法；与公司使用名义p值作为统计分析基础相关的风险；公司正在评估的测量方法是否继续被视为疾病进展的稳健且敏感的测量标准；RMAT指定或任何加速途径是否将导致监管批准；公司能否持续建设和维护实现其目标所需的基础设施和人员；公司在管理当前和未来临床试验及监管流程方面的有效性；公司在临床试验中证明其基因治疗候选产品治疗益处的能力；基因疗法的持续开发和接受度；公司获取、维护和保护其知识产权的能力；以及公司为其运营提供资金并在需要时以可接受的条件筹集额外资本的能力。

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 Reports on Form 10-Q filed May 9, 2025, and in other filings that the Company makes with the SEC from time to time.

这些风险和不确定性在公司向美国证券交易委员会（SEC）提交的定期报告中“风险因素”标题下有更详细的描述，包括其于2025年2月27日向SEC提交的年度报告Form 10-K、2025年5月9日提交的季度报告Form 10-Q，以及公司不时向SEC提交的其他文件。

Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements and, except as required by law, the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in.

鉴于这些风险、不确定性和其他因素，您不应过分依赖这些前瞻性陈述，并且，除非法律要求，即使新信息可用，公司也不承担更新这些前瞻性陈述的义务。

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