Marvel Biosciences Announces MB204 Outperforms Approved Drug Trofinetide in a Preclinical Model of Rett Syndrome
Marvel Biosciences (TSXV: MRVL, OTCQB: MBCOF) reported promising interim results from a preclinical study of MB204 as a potential treatment for Rett syndrome. Conducted by experts at the iBraiN Institute in France, the study showed that chronic oral dosing of MB204 restored social interaction behaviors to near-normal levels in a preclinical model of Rett syndrome. These improvements in cognitive function may represent a pivotal shift in therapy.
Led by Dr. Julie Le Merrer and Dr. Jerome Becker, the study found that 14 days of MB204 treatment (10 mg/kg oral dosing once daily) nearly reversed social and behavioral deficits. MB204 outperformed the approved drug Trofinetide (100 mg/kg via intraperitoneal injection once daily) on most studied endpoints and exhibited a distinct carry-over effect lasting at least 14 days after dosing cessation.
Marvel CSO Dr. Mark Williams noted that MB204 showed profound effects on restoring social behaviors, suggesting potential synaptic plasticity induction. Marvel CEO Rod Matheson emphasized the compelling nature of the data, hinting at improved therapies for Rett syndrome.
Marvel Biosciences (TSXV: MRVL, OTCQB: MBCOF) ha riportato risultati provvisori promettenti da uno studio preclinico su MB204 come trattamento potenziale per la sindrome di Rett. Condotto da esperti presso l'Istituto iBraiN in Francia, lo studio ha dimostrato che la somministrazione orale cronica di MB204 ha ripristinato comportamenti di interazione sociale a livelli quasi normali in un modello preclinico di sindrome di Rett. Questi miglioramenti nella funzione cognitiva potrebbero rappresentare un cambiamento fondamentale nella terapia.
Sotto la guida della Dr.ssa Julie Le Merrer e del Dr. Jerome Becker, lo studio ha scoperto che 14 giorni di trattamento con MB204 (10 mg/kg somministrati per via orale una volta al giorno) hanno quasi invertito le carenze sociali e comportamentali. MB204 ha superato il farmaco approvato Trofinetide (100 mg/kg tramite iniezione intraperitoneale una volta al giorno) su quasi tutti i punti finali studiati e ha mostrato un effetto duraturo distintivo che è durato almeno 14 giorni dopo la cessazione del trattamento.
Il CSO di Marvel, Dr. Mark Williams, ha notato che MB204 ha mostrato effetti profondi nel ripristinare i comportamenti sociali, suggerendo una potenziale induzione di plasticità sinaptica. Il CEO di Marvel, Rod Matheson, ha sottolineato la natura convincente dei dati, accennando a terapie migliorate per la sindrome di Rett.
Marvel Biosciences (TSXV: MRVL, OTCQB: MBCOF) reportó resultados interinos prometedores de un estudio preclínico sobre MB204 como un posible tratamiento para el síndrome de Rett. Realizado por expertos en el Instituto iBraiN en Francia, el estudio mostró que la dosificación oral crónica de MB204 restauró los comportamientos de interacción social a niveles casi normales en un modelo preclínico de síndrome de Rett. Estas mejoras en la función cognitiva podrían representar un cambio clave en la terapia.
Bajo la dirección de la Dra. Julie Le Merrer y el Dr. Jerome Becker, el estudio encontró que 14 días de tratamiento con MB204 (10 mg/kg administrados por vía oral una vez al día) casi invirtieron las deficiencias sociales y de comportamiento. MB204 superó al medicamento aprobado Trofinetide (100 mg/kg por inyección intraperitoneal una vez al día) en la mayoría de los puntos finales estudiados y mostró un efecto de persistencia distintivo que duró al menos 14 días después de la cesación del tratamiento.
El CSO de Marvel, Dr. Mark Williams, señaló que MB204 mostró efectos profundos en la restauración de los comportamientos sociales, sugiriendo una posible inducción de plasticidad sináptica. El CEO de Marvel, Rod Matheson, enfatizó la naturaleza convincente de los datos, insinuando mejoras en las terapias para el síndrome de Rett.
마블 바이오사이언스 (TSXV: MRVL, OTCQB: MBCOF)는 레트 증후군의 잠재적 치료제로서 MB204에 대한 전임상 연구에서 유망한 중간 결과를 보고했습니다. 프랑스에 있는 iBraiN 연구소의 전문가들이 수행한 이 연구는 MB204의 만성 경구 투여가 레트 증후군의 전임상 모델에서 사회적 상호작용 행동을 거의 정상 수준으로 회복시켰음을 보여주었습니다. 이러한 인지 기능의 개선은 치료의 중대한 전환점을 나타낼 수 있습니다.
Dr. Julie Le Merrer와 Dr. Jerome Becker가 이끄는 연구에서 14일 동안 MB204 치료(10 mg/kg 경구 투여 하루에 한 번)가 사회 및 행동 결핍을 거의 역전시켰습니다. MB204는 대부분의 연구된 최종 지표에서 승인된 약물인 트로피네타이드(100 mg/kg 복강 내 주사 하루에 한 번)를 초월했으며, 투여 중단 후 최소 14일 동안 지속되는 독특한 지속 효과를 보였습니다.
마블의 CSO인 Dr. Mark Williams는 MB204가 사회적 행동을 회복하는 데 깊은 영향을 미쳤다고 언급하며, 이는 시냅스 가소성 유도를 시사합니다. 마블의 CEO인 Rod Matheson은 데이터의 매력적인 성격을 강조하며 레트 증후군에 대한 개선된 치료법을 암시했습니다.
Marvel Biosciences (TSXV: MRVL, OTCQB: MBCOF) a rapporté des résultats intérimaires prometteurs d'une étude préclinique sur MB204 en tant que traitement potentiel pour le syndrome de Rett. Réalisée par des experts de l’Institut iBraiN en France, l’étude a montré que l'administration orale chronique de MB204 a restauré les comportements d'interaction sociale à des niveaux presque normaux dans un modèle préclinique du syndrome de Rett. Ces améliorations de la fonction cognitive pourraient représenter un tournant crucial dans la thérapie.
Dirigée par Dr. Julie Le Merrer et Dr. Jerome Becker, l'étude a révélé que 14 jours de traitement par MB204 (10 mg/kg par voie orale une fois par jour) avaient presque inversé les déficits sociaux et comportementaux. MB204 a surpassé le médicament approuvé Trofinetide (100 mg/kg par injection intrapéritonéale une fois par jour) sur la plupart des points finaux étudiés et a montré un effet persistant distinct qui a duré au moins 14 jours après l'arrêt du traitement.
Le CSO de Marvel, Dr. Mark Williams, a noté que MB204 montrait des effets profonds sur la restauration des comportements sociaux, suggérant une induction potentielle de plasticité synaptique. Le PDG de Marvel, Rod Matheson, a souligné la nature convaincante des données, laissant entendre des thérapies améliorées pour le syndrome de Rett.
Marvel Biosciences (TSXV: MRVL, OTCQB: MBCOF) berichtete über vielversprechende Zwischenresultate aus einer präklinischen Studie zu MB204 als potenzieller Behandlung für Rett-Syndrom. Die von Experten am iBraiN-Institut in Frankreich durchgeführte Studie zeigte, dass die chronische orale Verabreichung von MB204 das soziale Interaktionsverhalten in einem präklinischen Modell des Rett-Syndroms nahezu auf normale Werte wiederherstellte. Diese Verbesserungen der kognitiven Funktion könnten einen entscheidenden Wandel in der Therapie darstellen.
Unter der Leitung von Dr. Julie Le Merrer und Dr. Jerome Becker stellte die Studie fest, dass 14 Tage Behandlung mit MB204 (10 mg/kg orale Dosis einmal täglich) soziale und verhaltensbezogene Defizite nahezu umkehrte. MB204 übertraf das zugelassene Medikament Trofinetide (100 mg/kg intraperitoneale Injektion einmal täglich) in den meisten untersuchten Endpunkten und zeigte einen deutlichen Nachhangeffekt, der mindestens 14 Tage nach Beendigung der Behandlung anhielt.
Der CSO von Marvel, Dr. Mark Williams, bemerkte, dass MB204 tiefgreifende Auswirkungen auf die Wiederherstellung sozialer Verhaltensweisen zeigte, was auf eine mögliche Induktion von synaptischer Plastizität hindeutet. Der CEO von Marvel, Rod Matheson, betonte die überzeugende Natur der Daten und deutete auf verbesserte Therapien für das Rett-Syndrom hin.
- MB204 showed near reversal of social and behavioral deficits in a preclinical model of Rett syndrome.
- MB204 outperformed the approved drug Trofinetide on most studied endpoints.
- MB204 exhibited a lasting carry-over effect for at least 14 days after dosing cessation.
- The study results are preliminary and based on a preclinical model, not yet validated in human trials.
Calgary, Alberta--(Newsfile Corp. - November 7, 2024) - Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF), and its wholly owned subsidiary, Marvel Biotechnology Inc. (collectively the "Company" or "Marvel"), today reported promising interim results from its study on MB204, as a potential new treatment for Rett syndrome. The study, conducted by experts at the iBraiN Institute in Tours (France), demonstrated that chronic oral dosing of MB204 restored social interaction behaviors to near-normal levels in a preclinical model of Rett syndrome. These notable improvements in cognitive function could signify a pivotal shift in therapy.
The study, led by Dr. Julie Le Merrer and Dr. Jerome Becker, investigated the effects of 14 days of MB204 dosing on socialization, behavior, and cognition in the Rett syndrome model (Mecp2 knockout mouse). The approved drug Trofinetide was included as a positive control. Preliminary data highlights are:
- Near reversal of direct social and behavioral deficits after 2 weeks of MB204 treatment (10 mg/kg oral dosing once daily). MB204 outperformed Trofinetide (100 mg/kg via intraperitoneal injection once daily) in most studied endpoints.
- There was a distinct carry-over effect (lasting drug effect after dosing stopped) for MB204 after 7 days on most direct social behaviour endpoints which was not observed for Trofinetide. The carry-over effect continued for at least 14 days after cessation of MB204 dosing for select endpoints.
"This is the second preclinical model where we have seen a profound effect of MB204 on restoring many social behaviours," commented Marvel CSO Dr. Mark Williams. "We included the approved drug Trofinetide as a positive control in the study, but to dose it to clinically equivalent levels, Trofinetide had to be injected. Although early, the data clearly suggests that MB204 can improve social and behavioural functions more effectively with a much lower dose compared to Trofinetide. We are also very interested in the carry-over effect and suspect the drug could be inducing synaptic plasticity in brain structures. We intend to complete the balance of the study and publish the data."
"With the recent approval of Trofinetide by the FDA and Health Canada by Acadia Pharmaceuticals, there is renewed hope for people with Rett syndrome," said Marvel CEO Rod Matheson. "We believe these data with MB204 are very compelling and could result in even better therapies for individuals with Rett syndrome."
About Trofinetide:
Trofinetide, sold under the brand name Daybue® by Acadia Pharmaceuticals is a tripeptide fragment of insulin-like growth factor 1. The drug was approved by the FDA in 2023 and Health Canada in 2024. The drug is dosed between 5-12 grams twice daily. Acadia Pharmaceuticals reported US
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 that utilizes a "drug redevelopment" approach to drug development. Historically, when a new class of drug is developed, it is optimized for a particular target, but typically only approved for a specific disease. Often, a new disease is identified which involves the same target, however, pending the remaining patent life, the originally approved drug may not have sufficient time left for it to be commercially viable to be developed for the new disease indication. Marvel develops new synthetic chemical derivatives of the original approved drug for the new disease indication. Patent protection is sought, as the new potential asset is developed by the Company. The Company believes the business model results in significantly less risk, cost and time to develop its assets compared to traditional biotechnology companies.
Marvel Biotechnology Inc. has currently developed several new chemical entities, using synthetic chemical derivatives of known, off-patent drugs, that inhibit the A2a adenosine receptor with application to neurological diseases (depression & anxiety, Alzheimer's, ADHD), and the non-neurological conditions of cancer and non-alcoholic steatohepatitis. Marvel is also exploring additional undisclosed targets to expand its asset pipeline.
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
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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.
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