uniQure Announces Completion of Enrollment in the First Cohort and Favorable Recommendation from the Independent Data Monitoring Committee for its Phase I/IIa Clinical Trial of AMT-191 for the Treatment of Fabry Disease
uniQure (NASDAQ: QURE) has announced the completion of enrollment in the first cohort of its Phase I/IIa trial for AMT-191, a gene therapy treatment for Fabry disease. The Independent Data Monitoring Committee (IDMC) reviewed safety data from the initial two patients and found no significant safety concerns, recommending progression to the second cohort.
The company plans to begin enrollment for the second dose cohort in Q1 2025. AMT-191 is an AAV5-based gene therapy utilizing a proprietary promoter to deliver a galactosidase alpha (GLA) transgene targeting the liver to produce GLA protein. This one-time intravenous treatment aims to address Fabry disease, a condition where GLA gene variants cause α-galactosidase A enzyme deficiency, leading to lipid accumulation in multiple cell types and resulting in a multi-system disorder.
uniQure (NASDAQ: QURE) ha annunciato il completamento dell'arruolamento nella prima coorte del suo trial di Fase I/IIa per AMT-191, un trattamento di terapia genica per la malattia di Fabry. Il Comitato Indipendente di Monitoraggio dei Dati (IDMC) ha esaminato i dati di sicurezza dei primi due pazienti e non ha riscontrato preoccupazioni significative per quanto riguarda la sicurezza, raccomandando di procedere con la seconda coorte.
L'azienda prevede di iniziare l'arruolamento per la seconda coorte di dosi nel primo trimestre del 2025. AMT-191 è una terapia genica basata su AAV5 che utilizza un promotore proprietario per consegnare un trasgene di galattosidasi alfa (GLA) mirato al fegato per produrre la proteina GLA. Questo trattamento endovenoso somministrato una sola volta mira a trattare la malattia di Fabry, una condizione in cui le varianti del gene GLA causano una carenza dell'enzima α-galattosidasi A, portando all'accumulo di lipidi in più tipi cellulari e risultando in un disturbo multi-sistemico.
uniQure (NASDAQ: QURE) ha anunciado la finalización de la inscripción en la primera cohorte de su ensayo de Fase I/IIa para AMT-191, un tratamiento de terapia génica para la enfermedad de Fabry. El Comité Independiente de Monitoreo de Datos (IDMC) revisó los datos de seguridad de los dos primeros pacientes y no encontró preocupaciones significativas de seguridad, recomendando avanzar a la segunda cohorte.
La compañía planea comenzar la inscripción para la segunda cohorte de dosis en el primer trimestre de 2025. AMT-191 es una terapia génica basada en AAV5 que utiliza un promotor propietario para entregar un transgén de galactosidasa alfa (GLA) dirigido al hígado para producir la proteína GLA. Este tratamiento intravenoso de una sola vez tiene como objetivo abordar la enfermedad de Fabry, una condición en la que las variantes del gen GLA causan una deficiencia de la enzima α-galactosidasa A, lo que lleva a la acumulación de lípidos en múltiples tipos de células y resulta en un trastorno multisistémico.
uniQure (NASDAQ: QURE)는 AMT-191의 1상/2a 시험의 첫 번째 코호트 등록 완료를 발표했습니다. 이 치료는 파브리병에 대한 유전자 요법입니다. 독립 데이터 모니터링 위원회(IDMC)는 처음 두 환자의 안전성 데이터를 검토하고 심각한 안전성 문제가 없음을 발견하여 두 번째 코호트로의 진행을 추천했습니다.
회사는 2025년 1분기 중 두 번째 용량 코호트 등록을 시작할 계획입니다. AMT-191은 간에서 GLA 단백질을 생산하기 위해 GLA(α-galactosidase A) 유전자 치료법을 AAV5 기반의 독점 프로모터를 활용하여 전달합니다. 이 단회 정맥 주사 치료는 GLA 유전자 변이가 α-galactosidase A 효소 결핍을 초래하여 여러 세포 유형에서 지방축적이 발생하고 결과적으로 다기관 장애를 유발하는 파브리병을 해결하는 것을 목표로 합니다.
uniQure (NASDAQ: QURE) a annoncé l'achèvement de l'inscription dans la première cohorte de son essai de Phase I/IIa pour AMT-191, un traitement de thérapie génique pour la maladie de Fabry. Le Comité Indépendant de Surveillance des Données (IDMC) a examiné les données de sécurité des deux premiers patients et n'a trouvé aucune préoccupation significative en matière de sécurité, recommandant de passer à la deuxième cohorte.
L'entreprise prévoit de commencer l'inscription pour la deuxième cohorte de doses au premier trimestre de 2025. AMT-191 est une thérapie génique basée sur AAV5 utilisant un promoteur propriétaire pour délivrer un transgène de galactosidase alpha (GLA) ciblant le foie pour produire la protéine GLA. Ce traitement intraveineux unique vise à traiter la maladie de Fabry, une condition dans laquelle les variantes du gène GLA provoquent une carence en enzyme α-galactosidase A, entraînant une accumulation de lipides dans plusieurs types de cellules et résultant en un trouble multisystémique.
uniQure (NASDAQ: QURE) hat den Abschluss der Rekrutierung der ersten Kohorte seiner Phase I/IIa-Studie für AMT-191, eine Gentherapie zur Behandlung der Fabry-Krankheit, bekannt gegeben. Das Unabhängige Datenüberwachungskomitee (IDMC) hat die Sicherheitsdaten der ersten beiden Patienten überprüft und keine signifikanten Sicherheitsbedenken festgestellt, und empfiehlt die Fortsetzung zur zweiten Kohorte.
Das Unternehmen plant, die Rekrutierung für die zweite Dosis-Kohorte im ersten Quartal 2025 zu beginnen. AMT-191 ist eine gentherapeutische Behandlung auf Basis von AAV5, die einen proprietären Promotor verwendet, um ein Transgen für die Galaktosidase Alpha (GLA) zu liefern, das auf die Leber abzielt, um das GLA-Protein zu produzieren. Diese einmalige intravenöse Behandlung zielt darauf ab, die Fabry-Krankheit zu behandeln, eine Erkrankung, bei der GLA-Genvarianten zu einem Mangel des Enzyms α-Galaktosidase A führen, was zu einer Lipidakkumulation in mehreren Zelltypen und somit zu einer systemischen Erkrankung führt.
- IDMC review found no significant safety concerns in first cohort
- Cleared to proceed with second cohort enrollment
- Initial pharmacodynamics and biomarkers show encouraging results
- None.
Insights
The completion of first cohort enrollment and favorable IDMC recommendation for uniQure's AMT-191 trial represents a significant de-risking event in the development of their Fabry disease gene therapy program. The IDMC's positive safety assessment is particularly noteworthy given the scrutiny on AAV-based gene therapies' safety profiles.
The trial's progression carries strategic importance for several reasons: First, Fabry disease represents a $2+ billion market opportunity, currently dominated by enzyme replacement therapies that require regular infusions. A one-time gene therapy could be transformative for both patients and uniQure's market position. Second, the successful advancement validates uniQure's proprietary promoter technology and AAV5 platform, which has broader implications for their gene therapy pipeline.
The planned initiation of the second cohort in Q1 2025 maintains the development timeline, suggesting potential preliminary efficacy data could emerge in late 2025. This timing is important as several competitors are also developing gene therapies for Fabry disease. The liver-directed approach using AAV5 differentiates AMT-191 from other candidates and could potentially offer better targeting and reduced immunogenicity.
For investors, this progress demonstrates uniQure's ability to advance complex gene therapy programs through clinical development while maintaining safety standards. The company's focused execution in rare diseases, combined with their validated gene therapy platform, positions them well in the competitive landscape of genetic medicine.
~ Independent Data Monitoring Committee recommends proceeding with dosing of second cohort after planned safety assessment ~
~ Company expects to initiate enrollment of second dose cohort in the first quarter of 2025 ~
LEXINGTON, Mass. and AMSTERDAM, Feb. 03, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the completion of enrollment in the first cohort of the Phase I/IIa trial of AMT-191, an investigational gene therapy for the treatment of Fabry disease. Additionally, the Independent Data Monitoring Committee (IDMC) reviewed safety data from the initial two patients enrolled in the first cohort. The IDMC’s review identified no significant safety concerns and recommended proceeding with enrollment in the second cohort.
“Fabry is a debilitating disease that continues to represent a significant unmet medical need,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “We are encouraged by the initial pharmacodynamics, biomarkers and safety profile observed to date for AMT-191 as well as the positive outcome of the IDMC review. This strengthens our confidence in the potential of AMT-191 to make a meaningful difference in the lives of patients with Fabry disease. We look forward to advancing to the second cohort in this important clinical program.”
AMT-191 is an investigational AAV5-based gene therapy that uses a proprietary, highly potent promoter to deliver a galactosidase alpha (GLA) transgene designed to target the liver to produce GLA protein. In patients with Fabry disease, a pathogenic variant in the GLA gene leads to α-galactosidase A (aGAL-A) enzyme deficiency, which in turn results in a progressive accumulation of lipids in multiple cell types, including kidney and heart cells, eventually resulting in a multi-system disorder. AMT-191 may offer a novel one-time intravenously administered approach to treating Fabry disease.
About the Phase I/IIa Clinical Program of AMT-191
The Phase I/IIa clinical trial of AMT-191 is a multi-center, open-label trial being conducted in the United States consisting of two dosing cohorts of up to six adult male patients each receiving an intravenous infusion of AMT-191. Patients will continue to receive their regular enzyme replacement therapy until the criteria for withdrawal is met and will be followed for a period of 24 months. The trial will explore the safety, tolerability, and early signs of efficacy by measuring the expression of lysosomal enzyme aGLA-A. Additional details are available on www.clinicaltrials.gov (NCT06270316).
AMT-162 has been granted both Orphan Drug status and Fast Track designation by the U.S. Food and Drug Administration.
About Fabry Disease
Fabry disease is an X-linked genetic disorder resulting from a deficiency of GLA. Based on a 2020 study published in the Journal of Therapeutics and Clinical Risk Management, the prevalence is estimated to be between one in 40,000 and one in 117,000 individuals. The current standard of care for Fabry disease is bi-weekly infusions of enzyme replacement therapy, a treatment with limited effectiveness in many patients due to poor cross-correction, with inefficient clearance of substrates in the target organs, in particular the kidney and the heart.
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 regarding the Company’s plans to initiate enrollment of the second dose cohort of the AMT-191 study in the first quarter of 2025; the potential of AMT-191 to make a meaningful difference in the lives of patients and as a novel one-time intravenously administered approach to treating Fabry disease. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, without limitation, 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; risks associated with the implementation of the Company’s restructuring; 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 February 28, 2024, its Quarterly Report on Form 10-Q filed November 5, 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: | |
| Chiara Russo Direct: 617-306-9137 Mobile: 617-306-9137 c.russo@uniQure.com | Tom Malone Direct: 339-970-7558 Mobile:339-223-8541 t.malone@uniQure.com |
FAQ
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