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

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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.