Marvel Announces First of Two Final Data Sets from Rett Syndrome Study: MB-204 Significantly Outperforms Trofinetide

Core Insights - Marvel Biosciences Corp. has announced promising results from its preclinical study of MB-204, a treatment for Rett syndrome, which significantly outperformed Trofinetide, the only FDA-approved treatment for the condition [1][4]. Study Overview - The study compared MB-204 (10 mg/kg oral once daily) with Trofinetide (100 mg/kg injected once daily) in Mecp2 mice over approximately two weeks, assessing multiple endpoints [2]. Key Data Highlights - MB-204 demonstrated significant improvements in social behavioral deficiencies, with p-values less than 0.0001 compared to control, while Trofinetide showed no improvement [6]. - MB-204 also reversed impairments in following behavior (p<0.0001 vs control) and was significantly better than Trofinetide on this endpoint (p<0.001) [6]. - Both compounds reversed social preference behavior in the three-chamber test (p<0.0001 vs control), a common method for evaluating social behavior in autism models [6]. - MB-204 improved spatial working memory and repetitive behaviors as measured by the Y-maze test, with p-values less than 0.0001 compared to control, while Trofinetide showed no improvement [6]. Company Background - Marvel Biosciences Corp. is a Calgary-based preclinical stage pharmaceutical development biotechnology company focused on developing MB-204, a novel fluorinated derivative of Istradefylline, an approved anti-Parkinson's drug [5]. - The company is exploring the potential of MB-204 in treating various neurological diseases, including autism, depression, Alzheimer's Disease, and other neurodevelopmental disorders like Rett Syndrome and Fragile X Syndrome [5].