uniQure Announces Dosing of First Patient in Phase I/II Clinical Trial of AMT-162 for the Treatment SOD1-ALS
uniQure N.V. (NASDAQ: QURE) has announced the dosing of the first patient in the Phase I/II clinical trial of AMT-162 for the treatment of SOD1-ALS, a rare inherited form of amyotrophic lateral sclerosis. The EPISOD1 trial is a multi-center, open-label study with three dose-escalating cohorts, designed to assess the safety, tolerability, and exploratory efficacy of AMT-162.
AMT-162 is an AAVrh10-based gene therapy that aims to knock down the expression of mutated SOD1 protein, which is toxic to motor neurons. The therapy, administered intrathecally as a one-time treatment, has received Orphan Drug status and Fast Track designation from the FDA.
The trial will be conducted in the United States, with four active sites and plans to activate seven more by Q1 2025. It will measure neurofilament light chain and SOD1 protein levels as exploratory efficacy markers.
uniQure N.V. (NASDAQ: QURE) ha annunciato la somministrazione del primo paziente nel trial clinico di Fase I/II di AMT-162 per il trattamento della SOD1-ALS, una rara forma ereditaria di sclerosi laterale amiotrofica. Il trial EPISOD1 è uno studio multicentrico, in aperto, con tre coorti di dosaggio in aumento, progettato per valutare la sicurezza, la tollerabilità e l'efficacia esplorativa di AMT-162.
AMT-162 è una terapia genica basata su AAVrh10 che mira a ridurre l'espressione della proteina SOD1 mutata, tossica per i neuroni motori. La terapia, somministrata intratecalmente come trattamento una tantum, ha ricevuto la designazione di Farmaco Orfano e Fast Track dalla FDA.
Il trial sarà condotto negli Stati Uniti, con quattro siti attivi e piani per attivarne sette di più entro il primo trimestre del 2025. Misurerà i livelli della catena leggera di neurofilamento e della proteina SOD1 come indicatori di efficacia esplorativa.
uniQure N.V. (NASDAQ: QURE) ha anunciado la dosificación del primer paciente en el ensayo clínico de Fase I/II de AMT-162 para el tratamiento de SOD1-ALS, una rara forma hereditaria de esclerosis lateral amiotrófica. El ensayo EPISOD1 es un estudio multicéntrico, abierto, con tres cohortes de dosis en escalada, diseñado para evaluar la seguridad, la tolerabilidad y la eficacia exploratoria de AMT-162.
AMT-162 es una terapia génica basada en AAVrh10 que tiene como objetivo reducir la expresión de la proteína SOD1 mutada, que es tóxica para las neuronas motoras. La terapia, administrada intratecalmente como un tratamiento único, ha recibido el estatus de Medicamento Huérfano y Designación de Vía Rápida de la FDA.
El ensayo se llevará a cabo en los Estados Unidos, con cuatro sitios activos y planea activar siete más para el primer trimestre de 2025. Medirá los niveles de la cadena ligera de neurofilamentos y de la proteína SOD1 como indicadores de eficacia exploratoria.
uniQure N.V. (NASDAQ: QURE)는 SOD1-ALS 치료를 위한 AMT-162의 1상/2상 임상 시험에서 첫 번째 환자의 용량 투여를 발표했습니다. EPISOD1 시험은 안전성, 내약성 및 AMT-162의 탐색적 효능을 평가하기 위해 설계된 3개의 용량 증량 코호트와 함께 진행되는 다기관 공개 연구입니다.
AMT-162는 운동 뉴런에 독성이 있는 변이 SOD1 단백질의 발현을 억제하려는 AAVrh10 기반 유전자 요법입니다. 이 요법은 단회 치료로 경막외로 투여되며, FDA로부터 오르판 약 지정 및 신속 심사 지정을 받았습니다.
이 시험은 미국에서 진행되며, 4개의 활성 사이트를 가지고 있으며 2025년 1분기까지 7개 더 활성화할 계획입니다. 탐색적 효능 지표로서 신경 필라멘트 경 질량 체인 및 SOD1 단백질 수준을 측정할 것입니다.
uniQure N.V. (NASDAQ: QURE) a annoncé la dosimétrie du premier patient dans l'essai clinique de Phase I/II de AMT-162 pour le traitement de SOD1-ALS, une forme rare héréditaire de sclérose latérale amyotrophique. L'essai EPISOD1 est une étude multicentrique et ouverte avec trois cohortes de dose croissante, conçue pour évaluer la sécurité, la tolérabilité et l'efficacité exploratoire d'AMT-162.
AMT-162 est une thérapie génique basée sur AAVrh10 qui vise à réduire l'expression de la protéine SOD1 mutante, qui est toxique pour les neurones moteurs. La thérapie, administrée par voie intrathécale comme un traitement unique, a reçu le statut de médicament orphelin et la désignation Fast Track de la FDA.
L'essai sera mené aux États-Unis, avec quatre sites actifs et des projets d'en activer sept supplémentaires d'ici le premier trimestre 2025. Il mesurera les niveaux de chaînes légères de neurofilaments et de protéines SOD1 en tant que marqueurs d'efficacité exploratoires.
uniQure N.V. (NASDAQ: QURE) hat die Dosierung des ersten Patienten in der Phase I/II klinischen Studie zu AMT-162 zur Behandlung von SOD1-ALS, einer seltenen erblichen Form der amyotrophen Lateralsklerose, bekannt gegeben. Die EPISOD1 Studie ist eine multizentrische, offen durchgeführte Studie mit drei dosis steigenden Kohorten, die darauf abzielt, die Sicherheit, Verträglichkeit und explorative Wirksamkeit von AMT-162 zu bewerten.
AMT-162 ist eine auf AAVrh10 basierende Gentherapie, die darauf abzielt, die Expression des mutierten SOD1-Proteins zu reduzieren, das für motorische Neuronen toxisch ist. Die Therapie, die einmalig intrathekal verabreicht wird, hat von der FDA den Status eines Arzneimittels für seltene Krankheiten und eine Fast-Track-Zulassung erhalten.
Die Studie wird in den Vereinigten Staaten durchgeführt, mit vier aktiven Standorten und der Absicht, bis zum ersten Quartal 2025 sieben weitere zu aktivieren. Dabei werden die Werte von Neurofilament-Leichtketten und SOD1-Protein als explorative Wirksamkeitsmarker gemessen.
- First patient dosed in Phase I/II clinical trial for AMT-162
- AMT-162 granted Orphan Drug status and Fast Track designation by FDA
- Potential for one-time dosing with differentiated efficacy profile
- Expansion to 11 trial sites planned by Q1 2025
- None.
Insights
The initiation of the Phase I/II clinical trial for AMT-162 in SOD1-ALS is a significant milestone for uniQure. This gene therapy approach targets a specific genetic form of ALS, which affects about
Key points to consider:
- AMT-162's one-time intrathecal administration could offer a significant advantage over chronic treatments if proven effective.
- The use of neurofilament light chain as a biomarker is promising, as it's increasingly recognized as a valuable indicator of neuronal damage in ALS.
- The Fast Track designation from the FDA could accelerate the development process if early results are positive.
- With only four active sites initially and plans for seven more, patient recruitment might be a challenge, potentially affecting the timeline for data readouts.
While this news is encouraging, investors should remain cautious. Early-stage trials often face hurdles and the small patient population for SOD1-ALS may limit the market potential even if successful.
This clinical trial initiation, while scientifically significant, has immediate financial impact on uniQure. The company's market cap of
Key financial considerations:
- R&D expenses are likely to increase with this new trial, potentially impacting near-term cash burn.
- The orphan drug status could provide tax credits and market exclusivity if AMT-162 reaches approval.
- As a rare disease treatment, AMT-162 could command premium pricing if successful, but the small patient population may limit revenue potential.
- Investors should monitor cash runway and any potential need for additional financing to support this and other pipeline programs.
While positive trial results could boost investor confidence and stock price, the long development timeline for gene therapies means any significant revenue impact is likely years away. The company's overall financial health and progress of other pipeline candidates remain important for near-term valuation.
LEXINGTON, Mass. and AMSTERDAM, Oct. 15, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the first patient has been dosed in the Phase I/II clinical trial of AMT-162 for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1), a rare, inherited and progressive motor neuron disease. EPISOD1 is a Phase I/II multi-center, open-label trial being conducted in the United States with three dose-escalating cohorts assessing the safety, tolerability and exploratory signs of efficacy of AMT-162 in individuals with SOD1-ALS.
“We are pleased to announce the first patient dosing of AMT-162, our investigational gene therapy for the treatment of SOD1-ALS, a debilitating, degenerative and fatal disease,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “The start of this trial marks the advancement of our third gene therapy program into the clinic with this trial design, continuing our goal of rapidly generating proof-of-concept data using well-established biomarkers in order to bring treatments to patients as quickly as possible. We believe our novel AAV-based gene therapy candidate can deliver on the convenience of one-time dosing with the potential for a differentiated efficacy profile that is needed for such a devasting disease.”
AMT-162 is an investigational AAVrh10-based gene therapy that expresses a miRNA designed to knock down the expression of the mutated SOD1 protein. Patients with SOD1-ALS express a misfolded SOD1 protein. This misfolded protein is toxic to motor neurons causing degeneration which, over time leads to muscle weakness, loss of function and eventually death. AMT-162 may offer a novel one-time, intrathecally administered approach to slowing or halting the progression of SOD1-ALS. AMT-162 has been granted both Orphan Drug status and Fast Track designation by the U.S. Food and Drug Administration.
The EPISOD1 Phase I/II clinical trial of AMT-162 will be conducted in the United States. The multicenter, open-label trial consists of three cohorts with up to four patients each receiving a short course of immunosuppression prior to and after an intrathecal infusion of AMT-162. The trial will explore the safety and tolerability of AMT-162 and will assess exploratory signs of efficacy by measuring neurofilament light chain, a biomarker of neuronal damage, and SOD1 protein. There are currently four active sites in the U.S. with plans to activate seven additional sites by the first quarter of 2025. Additional details are available on www.clinicaltrials.gov (NCT06100276).
About Amyotrophic Lateral Sclerosis (ALS) caused by mutations in Superoxide Dismutase 1 (SOD1)
SOD1-ALS is a rare, progressive and fatal neurodegenerative disease that results in the loss of motor neurons in the brain and spinal cord. As ALS progresses, individuals experience muscle weakness and atrophy causing them to lose the ability to move their arms and legs, speak, swallow and eventually breathe, causing respiratory failure. Based on a 2021 study in Neuroepidemiology, there are an estimated ~170,000 individuals with ALS globally with SOD1 mutations representing
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 availability of proof-of-concept data in the Company’s open-label U.S. Phase I/II clinical trial for SOD1-ALS; the effectiveness of the EPISOD1 trial design; and the potential efficacy profile of AMT-162 through one-time administration with the ability to slow or halt the progression of SOD1-ALS. 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; 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 its 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 Reports on Form 10-Q filed May 7, 2024 and August 1, 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 | Tom Malone |
| Direct: 617-306-9137 | Direct: 339-970-7558 |
| Mobile: 617-306-9137 | Mobile: 339-223-8541 |
| c.russo@uniQure.com | t.malone@uniQure.com |
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FAQ
What is the purpose of uniQure's AMT-162 clinical trial for SOD1-ALS?
How is AMT-162 administered to patients with SOD1-ALS?
What biomarkers will be measured in the EPISOD1 trial of AMT-162?
How many sites are currently active for uniQure's AMT-162 clinical trial?
