Marvel Biosciences Announces Promising New Data from Rett Syndrome Study and Plans to Discuss Orphan and Rare Disease Status with the FDA
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF) has reported promising new data from its preclinical Rett syndrome study of MB204, conducted with the iBraiN Institute. The study compared MB204 to Trofinetide, the only FDA and Health Canada approved Rett syndrome treatment. Key findings show MB204 demonstrated sustained post-treatment benefits lasting 21 days, outperforming Trofinetide's 14-day effects. The company plans to pursue FDA Orphan and rare disease designation for MB204, which could provide market exclusivity and other benefits. Full study results are expected in Q1 2025.
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF) ha riportato nuovi dati promettenti dallo studio preclinico sulla sindrome di Rett riguardante MB204, condotto in collaborazione con l'Istituto iBraiN. Lo studio ha confrontato MB204 con Trofinetide, l'unico trattamento per la sindrome di Rett approvato dalla FDA e da Health Canada. Risultati chiave mostrano che MB204 ha dimostrato benefici sostenuti post-trattamento per 21 giorni, superando gli effetti di 14 giorni di Trofinetide. L'azienda prevede di richiedere la designazione di farmaco orfano e malattia rara per MB204, che potrebbe fornire esclusività di mercato e altri vantaggi. I risultati completi dello studio sono attesi nel primo trimestre del 2025.
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF) ha reportado datos prometedores de su estudio preclínico sobre el síndrome de Rett relacionado con MB204, realizado con el Instituto iBraiN. El estudio comparó MB204 con Trofinetide, el único tratamiento para el síndrome de Rett aprobado por la FDA y Health Canada. Hallazgos clave muestran que MB204 demostró beneficios sostenidos después del tratamiento que duran 21 días, superando los efectos de 14 días de Trofinetide. La empresa planea buscar la designación de enfermedad huérfana y rara de la FDA para MB204, lo que podría proporcionar exclusividad en el mercado y otros beneficios. Se esperan los resultados completos del estudio en el primer trimestre de 2025.
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF)는 iBraiN 연구소와 함께 수행된 MB204에 대한 전임상 레트 증후군 연구에서 유망한 새로운 데이터를 보고했습니다. 이 연구는 MB204와 FDA 및 캐나다 보건부에서 승인한 유일한 레트 증후군 치료제인 트로피네타이드(Trofinetide)를 비교했습니다. 주요 발견에 따르면, MB204는 21일 동안 지속적인 치료 후 이점을 보여주었으며, 이는 트로피네타이드의 14일 효과를 초과하는 결과입니다. 이 회사는 MB204에 대한 FDA의 고아약 및 희귀 질환 지정을 추구할 계획이며, 이는 시장 독점 및 기타 혜택을 제공할 수 있습니다. 전체 연구 결과는 2025년 1분기에 발표될 예정입니다.
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF) a rapporté de nouvelles données prometteuses de son étude préclinique sur le syndrome de Rett portant sur MB204, réalisée en collaboration avec l'Institut iBraiN. L'étude a comparé MB204 à Trofinetide, le seul traitement pour le syndrome de Rett approuvé par la FDA et Santé Canada. Résultats clés montrent que MB204 a démontré des bénéfices durables après traitement durant 21 jours, surpassant les effets de 14 jours de Trofinetide. L'entreprise prévoit de demander à la FDA la désignation de médicament orphelin et de maladie rare pour MB204, ce qui pourrait offrir une exclusivité sur le marché et d'autres avantages. Les résultats complets de l'étude sont attendus au premier trimestre 2025.
Marvel Biosciences (TSXV: MRVL) (OTCQB: MBCOF) hat vielversprechende neue Daten aus seiner präklinischen Studie zur Rett-Syndrom mit MB204, die in Zusammenarbeit mit dem iBraiN-Institut durchgeführt wurde, veröffentlicht. Die Studie verglich MB204 mit Trofinetide, der einzigen von der FDA und Health Canada zugelassenen Behandlung für das Rett-Syndrom. Wesentliche Ergebnisse zeigen, dass MB204 nachhaltige Vorteile nach der Behandlung für 21 Tage demonstrierte, was die 14-tägigen Effekte von Trofinetide übertrifft. Das Unternehmen plant, eine Orphan- und seltene Krankheits-Kennzeichnung der FDA für MB204 zu beantragen, die Marktexklusivität und andere Vorteile bieten könnte. Die vollständigen Studiendaten werden im ersten Quartal 2025 erwartet.
- MB204 showed superior durability with 21-day post-treatment benefits versus Trofinetide's 14 days
- Potential for market exclusivity through Orphan/rare disease designation
- Expanding research pipeline with additional studies in Fragile X syndrome and Alzheimer's disease
- Secured major grant for Alzheimer's disease research
- Results are still in preclinical stage, requiring further clinical trials
- Full study results not available until Q1 2025
Calgary, Alberta--(Newsfile Corp. - November 26, 2024) - Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF), and its wholly owned subsidiary, Marvel Biotechnology Inc. (collectively the "Company" or "Marvel"), are pleased to share additional data from its ongoing preclinical Rett syndrome study conducted in collaboration with the iBraiN Institute. Marvel also announces plans to engage with the FDA to discuss these promising pilot results.
The study evaluated MB204, Marvel's lead compound, in comparison to Trofinetide, the only FDA and Health Canada approved treatment for Rett syndrome. Mice were treated for two weeks with either compound, and social behaviour endpoints were assessed when the treatment was ceased and weekly thereafter. New data from our collaborations reveal the MB204 exhibited sustained post-treatment benefits, surpassing Trofinetide.
Key Data Highlights:
- MB204 demonstrated strong carry-over effects on multiple social behaviour endpoints, persisting for 21 days post-treatment. This new data suggests a durable post-treatment and potential neuromodulator effect.
- In contrast, Trofinetide showed minimal carry-over effects, lasting only 14 days.
- Full study results are expected in Q1 of 2025.
Building on these results in two models of autism, Marvel intends to begin the process with the FDA of obtaining Orphan and/or rare disease designation for MB204 in Rett Syndrome. Such designation could provide market exclusivity, tax incentives, and priority review waivers.
"MB204 continues to exceed our expectations in pre-clinical studies," said Dr. Mark Williams, Chief Science Officer of Marvel Biosciences. "MB204's long-last effects are particularly exciting and encouraging."
"Alongside this program, we are advancing preclinical studies in Fragile X syndrome and recently secured a major grant to support preclinical research in Alzheimer's disease," added Rod Matheson, CEO of Marvel Biosciences. "Achieving orphan or rare disease designation for MB204 could also add significant value to our lead asset."
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
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/231338
FAQ
What are the key findings of Marvel Biosciences' (MBCOF) Rett syndrome study?
When will Marvel Biosciences (MBCOF) release full Rett syndrome study results?
What regulatory benefits could Marvel Biosciences (MBCOF) receive for MB204?
