Marvel Announces the Second of Two Final Data Sets from Rett Syndrome Study: MB-204 Significantly Outperforms Trofinetide Following Cessation of Drug Treatment
Marvel Biosciences has announced significant results from its preclinical Rett syndrome study comparing MB-204 to Trofinetide. The study revealed that MB-204, administered at 10 mg/kg orally once daily, demonstrated superior and longer-lasting effects compared to Trofinetide (100 mg/kg injected).
Key findings show MB-204 maintained effectiveness for up to three weeks after treatment cessation, significantly outperforming Trofinetide in reversing social behavioral deficiencies. The drug showed remarkable results in multiple behavioral tests, including nose contacts, paw contacts, and following episodes (p<0.0001 vs control).
The research, conducted at the iBraiN Institute, will be presented at the International Rett Syndrome Foundation Scientific Meeting in Boston, June 2025. Scientists noted MB-204's potential as a novel treatment for both Rett syndrome and autism, highlighting its sustained effectiveness without reduction after chronic dosing.
Marvel Biosciences ha annunciato risultati significativi dal suo studio preclinico sulla sindrome di Rett, confrontando MB-204 con Trofinetide. Lo studio ha mostrato che MB-204, somministrato per via orale a 10 mg/kg una volta al giorno, ha dimostrato effetti superiori e più duraturi rispetto a Trofinetide (100 mg/kg per iniezione).
I risultati principali indicano che MB-204 ha mantenuto la sua efficacia fino a tre settimane dopo la sospensione del trattamento, superando significativamente Trofinetide nel migliorare i deficit comportamentali sociali. Il farmaco ha mostrato risultati notevoli in diversi test comportamentali, inclusi contatti con il naso, contatti con le zampe e episodi di inseguimento (p<0.0001 rispetto al controllo).
La ricerca, condotta presso l'iBraiN Institute, sarà presentata al International Rett Syndrome Foundation Scientific Meeting a Boston, giugno 2025. Gli scienziati hanno sottolineato il potenziale di MB-204 come nuovo trattamento per la sindrome di Rett e l'autismo, evidenziandone l'efficacia sostenuta senza diminuzione dopo somministrazioni croniche.
Marvel Biosciences ha anunciado resultados significativos de su estudio preclínico sobre el síndrome de Rett comparando MB-204 con Trofinetide. El estudio reveló que MB-204, administrado por vía oral a 10 mg/kg una vez al día, mostró efectos superiores y más duraderos en comparación con Trofinetide (100 mg/kg inyectado).
Los hallazgos clave indican que MB-204 mantuvo su efectividad hasta tres semanas después de suspender el tratamiento, superando significativamente a Trofinetide en la reversión de deficiencias en el comportamiento social. El medicamento mostró resultados notables en múltiples pruebas de comportamiento, incluyendo contactos con la nariz, contactos con las patas y episodios de seguimiento (p<0.0001 vs control).
La investigación, realizada en el Instituto iBraiN, será presentada en la Reunión Científica de la International Rett Syndrome Foundation en Boston, junio de 2025. Los científicos destacaron el potencial de MB-204 como un tratamiento novedoso tanto para el síndrome de Rett como para el autismo, resaltando su efectividad sostenida sin reducción tras dosis crónicas.
Marvel Biosciences는 MB-204와 Trofinetide를 비교한 레트 증후군 전임상 연구에서 중요한 결과를 발표했습니다. 연구에 따르면 MB-204는 10 mg/kg을 하루 한 번 경구 투여했을 때, Trofinetide(100 mg/kg 주사)보다 우수하고 지속적인 효과를 보였습니다.
주요 결과는 MB-204가 치료 중단 후 최대 3주까지 효과를 유지하며 사회적 행동 결함을 개선하는 데 있어 Trofinetide보다 현저히 뛰어났음을 보여줍니다. 이 약물은 코 접촉, 앞발 접촉, 추적 에피소드 등 여러 행동 테스트에서 뛰어난 결과를 나타냈습니다(p<0.0001 대조군 대비).
iBraiN 연구소에서 수행된 이 연구는 2025년 6월 보스턴에서 열리는 국제 레트 증후군 재단 과학 회의에서 발표될 예정입니다. 과학자들은 MB-204가 레트 증후군과 자폐증 모두에 대한 새로운 치료제로서의 가능성을 지니며, 만성 투여 후에도 효과가 감소하지 않는 점을 강조했습니다.
Marvel Biosciences a annoncé des résultats significatifs issus de son étude préclinique sur le syndrome de Rett, comparant MB-204 à Trofinetide. L'étude a révélé que MB-204, administré par voie orale à 10 mg/kg une fois par jour, a démontré des effets supérieurs et plus durables par rapport à Trofinetide (100 mg/kg injecté).
Les résultats clés montrent que MB-204 a maintenu son efficacité jusqu'à trois semaines après l'arrêt du traitement, surpassant significativement Trofinetide dans la correction des déficits comportementaux sociaux. Le médicament a obtenu des résultats remarquables dans plusieurs tests comportementaux, incluant les contacts nasaux, les contacts avec les pattes et les épisodes de suivi (p<0,0001 vs contrôle).
Cette recherche, menée à l'institut iBraiN, sera présentée lors du International Rett Syndrome Foundation Scientific Meeting à Boston en juin 2025. Les scientifiques ont souligné le potentiel de MB-204 comme nouveau traitement pour le syndrome de Rett et l'autisme, mettant en avant son efficacité soutenue sans diminution après une administration chronique.
Marvel Biosciences hat bedeutende Ergebnisse aus seiner präklinischen Studie zum Rett-Syndrom vorgestellt, in der MB-204 mit Trofinetide verglichen wurde. Die Studie zeigte, dass MB-204, oral einmal täglich mit 10 mg/kg verabreicht, überlegene und länger anhaltende Wirkungen im Vergleich zu Trofinetide (100 mg/kg injiziert) zeigte.
Wesentliche Erkenntnisse zeigen, dass MB-204 die Wirksamkeit bis zu drei Wochen nach Behandlungsende aufrechterhielt und Trofinetide bei der Umkehrung sozialer Verhaltensdefizite deutlich übertraf. Das Medikament erzielte bemerkenswerte Ergebnisse in mehreren Verhaltenstests, darunter Nasenkontakte, Pfotenkontakte und Verfolgungsepisoden (p<0,0001 vs. Kontrolle).
Die Forschung, durchgeführt am iBraiN-Institut, wird auf dem International Rett Syndrome Foundation Scientific Meeting im Juni 2025 in Boston vorgestellt. Wissenschaftler hoben das Potenzial von MB-204 als neuartige Behandlung sowohl für das Rett-Syndrom als auch für Autismus hervor und betonten die anhaltende Wirksamkeit ohne Abnahme nach chronischer Dosierung.
- MB-204 significantly outperformed FDA-approved Trofinetide in Rett syndrome treatment
- MB-204 showed sustained efficacy for 3 weeks after treatment cessation while Trofinetide showed no carry-over effect
- MB-204 demonstrated effectiveness at lower dosage (10mg/kg) compared to Trofinetide (100mg/kg)
- MB-204 reversed almost all social deficits in the test model with statistical significance (p<0.0001)
- Drug maintained effectiveness after chronic dosing, indicating no tolerance development
- None.
Calgary, Alberta--(Newsfile Corp. - April 30, 2025) - Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF), and its wholly owned subsidiary, Marvel Biotechnology Inc. (collectively the "Company" or "Marvel"), is pleased to share part of the second final data set from its preclinical Rett syndrome study conducted in collaboration with Dr. Julie Le Merrer and Dr. Jerome Becker at the iBraiN Institute.
The study evaluated MB-204, Marvel's lead compound (10 mg/kg oral once daily), in comparison to Trofinetide (100 mg/kg injected i.p. once daily), the only FDA and Health Canada approved treatment for Rett syndrome. Mecp2 mice were treated for approximately two weeks with either compound and then treatment ceased. Animals were monitored for up to three weeks post-treatment to study the carry-over effect of the drug.
Key Data Highlights:
- Week 1: MB-204 continued to reverse social behavioural deficiencies as measured by the number and duration of nose contacts, the number and duration of paw contacts, the number of following episodes and in the 3 Chamber Test (p<0.0001 vs control). Trofinetide only showed an improvement on following episode (p<0.05 vs control). MB-204 was significantly better than Trofinetide on the same endpoints (p<0.0001 except following episodes p<0.01). The 3 Chamber Test was conducted on day 10 post treatment.
- Week 2: MB-204 continued to maintain its positive effect on the number and duration of nose contacts, in addition to a continued effect on following episodes (p<0.0001 vs control), and outperformed Trofinetide (p<0.0001 and p<0.05 on the same respective endpoints). Trofinetide showed no discernible carry-over effect.
- Week 3: MB-204 continued to maintain its positive effect on nose contacts and following episodes (p<0.0001 vs control). The Trofinetide was not studied owing to the lack of any discernible carry-over effect in the previous week.
"These results are very encouraging," said Drs. Le Merrer and Becker. "MB-204 nearly reversed all the social deficits in the Mecp2 model while under treatment as we previously saw after a single dose in the Oprm1 mouse model of autism. This study suggests the drug does not reduce its effectiveness after chronic dosing, and we were pleasantly surprised by the continued carry-over effect seen at week 3 which suggests a profound sustained effect of MB-204. We look forward to seeing MB-204 advance as a novel treatment for Rett syndrome and autism in general".
The Company and its collaborators intend to present the data as an oral presentation at the International Rett Syndrome Foundation (IRSF) Scientific Meeting, taking place in Boston, June 9-11th, 2025.
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.
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FAQ
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