Marvel Biosciences Announces Promising New Data from Rett Syndrome Study and Plans to Discuss Orphan and Rare Disease Status with the FDA

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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.