A pre-clinical biotech company from Calgary, Marvel Biosciences Corporation, and its fully owned subsidiary Marvel Biotechnology, have shared new findings from their ongoing preclinical research on Rett syndrome. Conducted in partnership with the iBraiN Institute, these revelations offer encouraging pilot results that Marvel plans to bring to the attention of the US FDA.
In this study, the effectiveness of Marvel’s prominent compound, MB204, was compared against Trofinetide—the only treatment for Rett syndrome sanctioned by the US FDA and Health Canada. Over a two-week period, mice were administered either MB204 or Trofinetide, with their social behavior tracked post-treatment and weekly thereafter. Findings highlighted that MB204 presented sustained benefits after stopping the treatment, outperforming Trofinetide.
**Key Findings:**
– MB204 exhibited strong post-treatment effects on various social behavior parameters, lasting up to 21 days, suggesting a persistent influence that could act as a neuromodulator.
– Trofinetide’s effects were relatively short-lived, persisting only for 14 days.
– Comprehensive study results are anticipated by the first quarter of 2025.
Building on the successes seen in two autism models, Marvel aims to initiate discussions with the US FDA to secure orphan or rare disease designation for MB204 concerning Rett syndrome. Such recognition could offer benefits like market exclusivity, tax breaks, and expedited review processes.
Dr. Mark Williams, the Chief Science Officer at Marvel Biosciences, remarked, “MB204 consistently surpasses expectations in preclinical assessments with its enduring effects being notably inspiring.”
“In parallel with this endeavor, we’re pushing forward with preclinical studies concerning Fragile X syndrome and have recently been awarded a substantial grant for Alzheimer’s research,” commented Rod Matheson, CEO of Marvel Biosciences. “Securing orphan or rare disease designation for MB204 holds the potential to significantly enhance the value of our primary asset.”