Marvel Biosciences Announces MB204 Outperforms Approved Drug Trofinetide in a Preclinical Model of Rett Syndrome

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 $251.7 million in net product sales of Daybue® for their nine month period ended September 30, 2024.

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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