MeiraGTx Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for AAV2-hAQP1 for the Treatment of Grade 2/3 Radiation-Induced Xerostomia
MeiraGTx has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for AAV2-hAQP1, targeting Grade 2/3 radiation-induced xerostomia (RIX). This designation, granted to less than half of applicants, includes benefits of Fast Track and Breakthrough Therapy designations, enabling increased FDA interaction and potential accelerated approval.
The therapy showed promising results in the Phase 1 AQUAx clinical trial, demonstrating significant improvements in patient-reported outcomes and saliva production, with no treatment-related serious adverse events. The ongoing Phase 2 AQUAx2 study is a randomized, double-blind, placebo-controlled trial across multiple sites in the U.S., Canada, and U.K., which could potentially support a BLA filing.
MeiraGTx ha ricevuto la designazione FDA Regenerative Medicine Advanced Therapy (RMAT) per AAV2-hAQP1, che mira a trattare la xerostomia indotta da radiazioni di Grado 2/3 (RIX). Questa designazione, concessa a meno della metà dei richiedenti, include i vantaggi delle designazioni Fast Track e Breakthrough Therapy, permettendo un maggiore interazione con la FDA e la potenziale accelerazione dell'approvazione.
La terapia ha mostrato risultati promettenti nel trial clinico di Fase 1 AQUAx, dimostrando significativi miglioramenti nei risultati riportati dai pazienti e nella produzione di saliva, senza eventi avversi seri correlati al trattamento. Lo studio in corso di Fase 2 AQUAx2 è uno studio randomizzato, in doppio cieco e controllato con placebo che si svolge in più sedi negli Stati Uniti, in Canada e nel Regno Unito, che potrebbe potenzialmente supportare una richiesta di BLA.
MeiraGTx ha recibido la designación de la FDA Regenerative Medicine Advanced Therapy (RMAT) para AAV2-hAQP1, dirigida a la xerostomía inducida por radiación de Grado 2/3 (RIX). Esta designación, otorgada a menos de la mitad de los solicitantes, incluye los beneficios de las designaciones Fast Track y Breakthrough Therapy, permitiendo una mayor interacción con la FDA y una posible aceleración en la aprobación.
La terapia mostró resultados prometedores en el ensayo clínico de Fase 1 AQUAx, demostrando mejoras significativas en los resultados reportados por los pacientes y en la producción de saliva, sin eventos adversos graves relacionados con el tratamiento. El estudio en curso de Fase 2 AQUAx2 es un ensayo aleatorizado, doble ciego y controlado con placebo que se lleva a cabo en múltiples sitios en EE.UU., Canadá y Reino Unido, el cual podría potencialmente respaldar una solicitud de BLA.
MeiraGTx는 FDA로부터 AAV2-hAQP1에 대한 재생의학 고급 치료(RMAT) 지정을 받았습니다. 이는 2/3 등급의 방사선 유도 구강 건조증(RIX)을 목표로 하고 있습니다. 이 지명은 신청자의 절반도 안 되는 수에 부여되며, 빠른 심사(Fast Track) 및 혁신 치료(Breakthrough Therapy) 지명의 이점을 포함하여 FDA와의 상호작용을 증가시키고 잠재적인 신속 승인 가능성을 제공합니다.
이 치료법은 1상 AQUAx 임상시험에서 유망한 결과를 보여주었으며, 환자가 보고한 결과 및 침의 생산에서 상당한 개선을 나타냈으며, 치료와 관련된 심각한 부작용은 없었습니다. 진행 중인 2상 AQUAx2 연구는 미국, 캐나다 및 영국의 여러 사이트에서 이루어지는 무작위 이중 맹검 위약 대조 연구로, BLA 제출을 지원할 가능성이 있습니다.
MeiraGTx a reçu la désignation de la FDA pour la Therapie Avancée en Médecine Régénérative (RMAT) pour AAV2-hAQP1, ciblant la xérostomie induite par les radiations de Grade 2/3 (RIX). Cette désignation, accordée à moins de la moitié des demandeurs, inclut les avantages des désignations Fast Track et Breakthrough Therapy, permettant une interaction accrue avec la FDA et une approbation potentiellement accélérée.
La thérapie a montré des résultats prometteurs dans l'essai clinique de Phase 1 AQUAx, démontrant des améliorations significatives des résultats signalés par les patients et de la production de salive, sans événements indésirables graves liés au traitement. L'étude en cours de Phase 2 AQUAx2 est un essai randomisé, en double aveugle et contrôlé par placebo mené sur plusieurs sites aux États-Unis, au Canada et au Royaume-Uni, qui pourrait potentiellement soutenir une demande de BLA.
MeiraGTx hat von der FDA die Regenerative Medicine Advanced Therapy (RMAT)-Auszeichnung für AAV2-hAQP1 erhalten, das auf Grad 2/3 strahleninduzierte Xerostomie (RIX) abzielt. Diese Auszeichnung, die weniger als der Hälfte der Antragsteller erhält, umfasst Vorteile der Fast-Track- und Durchbruchs-Therapie-Auszeichnungen, was eine erhöhte Interaktion mit der FDA und eine potenzielle beschleunigte Genehmigung ermöglicht.
Die Therapie zeigte vielversprechende Ergebnisse in der Phase-1-AQUAx-Studie, wobei signifikante Verbesserungen in den von den Patienten berichteten Ergebnissen und der Speichelproduktion beobachtet wurden, ohne schwerwiegende behandlungsbezogene Nebenwirkungen. Die laufende Phase-2-AQUAx2-Studie ist eine randomisierte, doppelblinde, placebo-kontrollierte Studie an mehreren Standorten in den USA, Kanada und Großbritannien, die möglicherweise eine BLA-Einreichung unterstützen könnte.
- Received RMAT designation, enabling accelerated development and review process
- Phase 1 trial showed significant improvements in patient outcomes
- No treatment-related serious adverse events reported in clinical trials
- FDA alignment on Phase 2 trial as potential pivotal study for BLA filing
- In-house manufacturing capabilities established for production
- None.
Insights
The FDA's RMAT designation for MeiraGTx's AAV2-hAQP1 gene therapy represents a significant regulatory milestone. This designation is particularly noteworthy as <b>less than 50% of RMAT applications are approved</b>, indicating strong preliminary clinical evidence. The therapy targets radiation-induced xerostomia, a severe condition affecting cancer survivors with no current effective treatments.
The Phase 1 AQUAx trial results demonstrated meaningful improvements in both subjective outcomes and objective saliva production measurements, with a favorable safety profile. The ongoing Phase 2 AQUAx2 trial has already received FDA agreement to serve as a pivotal study for BLA submission, which could significantly accelerate the path to market.
The combination of RMAT, Fast Track and potential Priority Review status substantially reduces regulatory hurdles and development timeline, potentially bringing this first-in-class treatment to market years earlier than traditional pathways.
This regulatory milestone significantly enhances MeiraGTx's market position and development timeline. The RMAT designation, combined with previous Orphan Drug status, provides multiple strategic advantages including expedited review processes and increased FDA interaction. For a company with a <money>470M</money> market cap, this regulatory win could attract institutional investors and potential partners.
The agreement for Phase 2 AQUAx2 to serve as a pivotal trial represents substantial cost savings and timeline acceleration. MeiraGTx's vertical integration with proprietary manufacturing capabilities further strengthens their position, reducing reliance on third-party manufacturers and potentially improving margins. The combination of these factors positions the company favorably for either commercial success or potential acquisition interest from larger pharmaceutical companies.
- RMAT designation recognizes the preliminary clinical evidence of the potential benefit of AAV2-hAQP1 as a one-time treatment for this debilitating condition
- RMAT designation includes the benefits of the Fast Track and Breakthrough Therapy designations, allows frequent regulatory interactions with the FDA, and potential routes to accelerated approval and Priority Review
LONDON and NEW YORK, Dec. 09, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV2-hAQP1 for the treatment of Grade 2/3 radiation-induced xerostomia (RIX).
“This RMAT designation underscores the strength of our data indicating the potential of our AAV2-hAQP1 therapy to significantly improve the lives of patients who suffer from xerostomia following radiation treatment. The requirements for receiving an RMAT designation include that the drug candidate is an advanced regenerative medicine, in this case a gene therapy; that the therapy is targeting a serious condition, in this case, Grade 2 and Grade 3 late xerostomia caused by radiotherapy for cancers of the upper aerodigestive tract; and that the applicant has presented preliminary clinical evidence demonstrating that the drug candidate has the potential to address an unmet need in the serious condition. The RMAT requirement for clinical data supporting a benefit in an unmet need is a high hurdle, with less than half of all RMAT designation applications granted. We are therefore very excited to have been awarded this designation for our AAV-hAQP1 program and we look forward to working closely with the FDA to bring this potential life changing therapy to these patients with no alternative treatments as quickly as possible,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx.
The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising Regenerative Medicine therapeutic candidates, including human gene therapies, that treat, modify, reverse or cure serious or life-threatening diseases. Similar to Breakthrough Therapy designation, RMAT designation allows for increased interaction with the FDA and immediate multidisciplinary comprehensive discussions of the ongoing product development program, clinical trials and plans for expediting the manufacturing development strategy. RMAT designation includes the benefits of Fast Track and Breakthrough Therapy designations with rolling review and potential Priority Review of a product’s biologics license application (BLA).
AAV2-hAQP1 was previously granted Orphan Drug Designation by the FDA.
AAV2-hAQP1 for the Treatment of Xerostomia:
- Data from the Company’s Phase 1 AQUAx clinical trial were presented in an oral session at the American Academy of Oral Medicine (AAOM) 2024 annual meeting in April 2024, demonstrating that treatment with AAV2-hAQP1 resulted in significant improvements across three different patient-reported outcomes and in saliva production, with no treatment-related serious adverse events or dose-limiting toxicities reported. These data underpinned this successful RMAT designation.
- The Phase 2 AQUAx2 (NCT05926765) randomized, double-blind, placebo-controlled study continues to enroll and dose participants at multiple sites in the U.S., Canada and the U.K.
- The Company has gained alignment with the FDA on requirements for the ongoing Phase 2 AQUAx2 clinical trial for Grade 2/3 radiation-induced xerostomia to be considered a pivotal trial in support of a potential BLA filing based on the use of material manufactured using MeiraGTx’s proprietary production process and in-house manufacturing facilities.
- The RMAT designation will allow the Company to benefit from increased interactions with the FDA to further accelerate the development pathway and BLA approval.
About AAV2-hAQP1
Grade 2/3 radiation-induced xerostomia (RIX) is a severely debilitating consequence of radiation treatment for head and neck cancer that affects approximately 30
About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of late-stage clinical programs supported by end-to-end manufacturing capabilities. MeiraGTx has internal plasmid production for GMP, two GMP viral vector production facilities as well as an in-house Quality Control hub for stability and release, all fit for IND through commercial supply. In addition, MeiraGTx has developed a proprietary manufacturing platform with leading yield and quality aspects and commercial readiness, core capabilities in viral vector design and optimization and a transformative riboswitch gene regulation platform technology that allows for the precise, dose-responsive control of gene expression by oral small molecules. MeiraGTx is focusing the riboswitch platform on the delivery of metabolic peptides, including GLP-1, GIP, Glucagon, and PYY, using oral small molecules, as well as cell therapy for oncology and autoimmune diseases. MeiraGTx has developed the technology to apply genetic medicine to more common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high.
For more information, please visit www.meiragtx.com
Forward Looking Statement
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FAQ
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