Marvel to File for Orphan Drug Designation with The U.S. FDA for MB-204 as a Treatment for Rett Syndrome
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF) has announced plans to file for Orphan Drug Designation (ODD) with the FDA for MB-204 as a potential treatment for Rett Syndrome. The decision follows promising preclinical data showing MB-204's superior performance compared to Trofinetide, the only FDA-approved treatment for the disease.
The preclinical study, conducted with the iBraiN Institute and led by Drs. Julie le Merrer and Jerome Becker, evaluated MB-204's efficacy in a Rett Syndrome mouse model, focusing on social interaction, memory, and repetitive behaviors.
ODD is a regulatory pathway supporting treatments for rare diseases affecting fewer than 200,000 individuals in the U.S. The designation offers several benefits including:
- Market exclusivity
- Tax credits
- Waived FDA user fees
- Access to expedited programs like fast-track and priority review
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF) ha annunciato piani per richiedere la Designazione di Farmaco Orfano (ODD) presso la FDA per MB-204 come potenziale trattamento per la Sindrome di Rett. La decisione segue dati preclinici promettenti che mostrano la superiore efficacia di MB-204 rispetto a Trofinetide, l'unico trattamento approvato dalla FDA per la malattia.
Lo studio preclinico, condotto con l'Istituto iBraiN e guidato dai Dott. Julie le Merrer e Jerome Becker, ha valutato l'efficacia di MB-204 in un modello murino della Sindrome di Rett, concentrandosi sull'interazione sociale, la memoria e i comportamenti ripetitivi.
L'ODD è un percorso normativo che supporta i trattamenti per malattie rare che colpiscono meno di 200.000 individui negli Stati Uniti. La designazione offre diversi vantaggi tra cui:
- Esclusività di mercato
- Crediti d'imposta
- Esenzione dalle tasse per l'uso della FDA
- Accesso a programmi accelerati come fast-track e revisione prioritaria
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF) ha anunciado planes para solicitar la Designación de Medicamento Huérfano (ODD) ante la FDA para MB-204 como un posible tratamiento para el Síndrome de Rett. La decisión sigue a datos preclínicos prometedores que muestran el rendimiento superior de MB-204 en comparación con Trofinetide, el único tratamiento aprobado por la FDA para la enfermedad.
El estudio preclínico, realizado con el Instituto iBraiN y dirigido por los Drs. Julie le Merrer y Jerome Becker, evaluó la eficacia de MB-204 en un modelo de ratón del Síndrome de Rett, centrándose en la interacción social, la memoria y los comportamientos repetitivos.
La ODD es una vía regulatoria que apoya tratamientos para enfermedades raras que afectan a menos de 200,000 individuos en EE. UU. La designación ofrece varios beneficios, incluyendo:
- Exclusividad en el mercado
- Créditos fiscales
- Exención de tarifas de usuario de la FDA
- Acceso a programas acelerados como fast-track y revisión prioritaria
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF)는 FDA에 MB-204를 레트 증후군의 잠재적 치료제로서 고아약 지정 (ODD)을 신청할 계획을 발표했습니다. 이 결정은 MB-204가 이 질병에 대해 FDA 승인을 받은 유일한 치료제인 트로피네타이드에 비해 우수한 성능을 보여주는 유망한 전임상 데이터를 바탕으로 합니다.
iBraiN 연구소와 협력하여 Drs. Julie le Merrer와 Jerome Becker가 이끄는 전임상 연구는 레트 증후군 쥐 모델에서 MB-204의 효능을 평가하며, 사회적 상호작용, 기억력 및 반복 행동에 중점을 두었습니다.
ODD는 미국에서 200,000명 미만의 개인에게 영향을 미치는 희귀 질환 치료를 지원하는 규제 경로입니다. 이 지정은 다음과 같은 여러 가지 혜택을 제공합니다:
- 시장 독점권
- 세금 공제
- FDA 사용자 수수료 면제
- 패스트트랙 및 우선 심사와 같은 신속 프로그램에 대한 접근
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF) a annoncé son intention de demander la Désignation de Médicament Orphelin (ODD) auprès de la FDA pour MB-204 en tant que traitement potentiel du Syndrome de Rett. Cette décision fait suite à des données précliniques prometteuses montrant la performance supérieure de MB-204 par rapport à Trofinetide, le seul traitement approuvé par la FDA pour cette maladie.
L'étude préclinique, réalisée en collaboration avec l'Institut iBraiN et dirigée par les Drs. Julie le Merrer et Jerome Becker, a évalué l'efficacité de MB-204 dans un modèle murin du Syndrome de Rett, en se concentrant sur l'interaction sociale, la mémoire et les comportements répétitifs.
L'ODD est une voie réglementaire qui soutient les traitements pour les maladies rares touchant moins de 200 000 individus aux États-Unis. La désignation offre plusieurs avantages, notamment :
- Exclusivité sur le marché
- Crédits d'impôt
- Exonération des frais d'utilisation de la FDA
- Accès à des programmes accélérés tels que le fast-track et l'examen prioritaire
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF) hat Pläne angekündigt, bei der FDA eine Waisenarzneimittel-Zulassung (ODD) für MB-204 als potenzielle Behandlung des Rett-Syndroms zu beantragen. Die Entscheidung folgt auf vielversprechende präklinische Daten, die die überlegene Leistung von MB-204 im Vergleich zu Trofinetide, dem einzigen von der FDA zugelassenen Medikament für die Krankheit, zeigen.
Die präklinische Studie, die in Zusammenarbeit mit dem iBraiN Institut und unter der Leitung von Dr. Julie le Merrer und Jerome Becker durchgeführt wurde, bewertete die Wirksamkeit von MB-204 in einem Mausmodell des Rett-Syndroms und konzentrierte sich auf soziale Interaktion, Gedächtnis und repetitive Verhaltensweisen.
ODD ist ein regulatorischer Weg, der Behandlungen für seltene Krankheiten unterstützt, die weniger als 200.000 Personen in den USA betreffen. Die Zulassung bietet mehrere Vorteile, darunter:
- Marktexklusivität
- Steuergutschriften
- Gebührenbefreiung für FDA-Nutzer
- Zugang zu beschleunigten Programmen wie Fast-Track und Prioritätsprüfung
- Preclinical data shows MB-204 outperforms Trofinetide, the only FDA-approved treatment
- Potential to receive Orphan Drug Designation benefits including market exclusivity
- Targeting an underserved market with treatment options
- Still in preclinical stage, requiring extensive clinical trials before potential approval
- Success in mouse models doesn't guarantee efficacy in human trials
Calgary, Alberta--(Newsfile Corp. - March 17, 2025) - Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF), and its wholly owned subsidiary, Marvel Biotechnology Inc. (collectively the "Company" or "Marvel"), announced plans to file for Orphan Drug Designation (ODD) with the U.S. Food and Drug Administration (FDA) for its lead compound, MB-204, as a potential treatment for Rett Syndrome.
This decision follows strong preclinical data showing MB-204's sustained benefits, outperforming Trofinetide-the only FDA-approved treatment for the disease. Marvel aims to leverage ODD benefits to accelerate MB-204's clinical development and provide new hope to patients and families.
Marvel's preclinical study, conducted in collaboration with the iBraiN Institute, evaluated MB-204's efficacy using Trofinetide as a benchmark. Led by Drs. Julie le Merrer and Jerome Becker, the study assessed key behavioral outcomes in a Rett Syndrome mouse model (Mecp2 +/-), including social interaction, memory, and repetitive behaviors.
"We are very pleased with these results and excited to take the next step in seeking Orphan Drug Designation," said Dr. Mark Williams, Chief Science Officer of Marvel Biosciences. "Trofinetide, which received FDA approval in 2023 for Rett Syndrome, also holds Orphan Drug Status. Given the encouraging data from our study, we see a strong case for MB-204 to follow a similar regulatory path."
Orphan Drug Designation is a critical regulatory pathway that supports the development of treatments for rare diseases affecting fewer than 200,000 individuals in the U.S. Rett Syndrome, a severe neurodevelopmental disorder primarily affecting girls, has limited treatment options, making ODD an important tool for advancing new therapies. The designation provides key benefits, including market exclusivity, financial support through tax credits, waived FDA user fees, and access to expedited programs such as fast-track and priority review to accelerate the approval process.
"Orphan Drug Designation will help validate MB-204's potential in Rett Syndrome and reinforce Marvel's position as a leader in rare neurological therapies," said Rod Matheson, CEO of Marvel Biosciences. "This designation would provide essential support as we work to bring this much-needed treatment to patients."
About Marvel Biosciences Corp.
Marvel Biosciences Corp., and its wholly owned subsidiary, Marvel Biotechnology Inc., is a Calgary-based pre-clinical stage pharmaceutical development biotechnology company. The Company is developing MB-204, a novel fluorinated derivative of the approved anti-Parkinson's drug Istradefylline, the only clinically approved adenosine A2a antagonist. A significant and growing body of scientific evidence suggests drugs that block the adenosine A2a receptor, such as MB-204, could be useful in treating other neurological diseases such as autism, depression and Alzheimer's Disease. The Company is actively investigating its potential in addressing other neurodevelopmental disorders, such as Rett Syndrome and Fragile X Syndrome, to expand its therapeutic reach.
Contact Information:
Marvel Biosciences Corp.
J. Roderick (Rod) Matheson, Chief Executive Officer or
Dr. Mark Williams, President and Chief Science Officer
Tel: 403 770 2469
Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSXV) accepts responsibility for the adequacy or accuracy of this press release. All information contained in this news release with respect to the Company and its subsidiary, (collectively, the "Parties") were supplied by Marvel, respectively, for inclusion herein and each parties' directors and officers have relied on each other for any information concerning such Party.
This news release may contain forward-looking statements and other statements that are not historical facts. Forward-looking statements are often identified by terms such as "will", "may", "should", "anticipate", "expects" and similar expressions. All statements other than statements of historical fact, included in this release, including, without limitation, statements regarding the future plans and objectives of the Company are forward-looking statements that involve risks and uncertainties. There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those anticipated in such statements. Important factors that could cause actual results to differ materially from the expectations of the Company and include other risks detailed from time to time in the filings made by the Company under securities regulations.
The reader is cautioned that assumptions used in the preparation of any forward-looking information may prove to be incorrect. Events or circumstances may cause actual results to differ materially from those predicted, as a result of numerous known and unknown risks, uncertainties, and other factors, many of which are beyond the control of the Company. As a result, the Company cannot guarantee that the above events on the terms will occur and within the time disclosed herein or at all. The reader is cautioned not to place undue reliance on any forward-looking information. Such information, although considered reasonable by management at the time of preparation, may prove to be incorrect and actual results may differ materially from those anticipated. Forward-looking statements contained in this news release are expressly qualified by this cautionary statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company will update or revise publicly any of the included forward-looking statements as expressly required by Canadian securities law.
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/244579
FAQ
What are the key findings of Marvel Biosciences' (MBCOF) preclinical study for MB-204 in Rett Syndrome?
What benefits would Orphan Drug Designation provide for Marvel Biosciences' (MBCOF) MB-204?
How does Marvel Biosciences' (MBCOF) MB-204 compare to existing Rett Syndrome treatments?
What is the target patient population for Marvel Biosciences' (MBCOF) MB-204 Rett Syndrome treatment?
