About two and half years ago, back in May 2013, this blog reported that an experimental drug NNZ-2566 might be useful for protecting the brains of individuals with MECP2 duplication syndrome. Now there has been encouraging news from clinical trials using this drug with individuals with Rett syndrome.
In 2013, there was more encouraging news suggesting that soldiers who sustained head injuries might have fewer lasting symptoms of traumatic brain injury.
Now, Neuren, the company behind NNZ-2566, has released more encouraging results from clinical trials in Rett syndrome. They report:
- Achieved primary endpoint -bothe dose levels.
- Higher dose (70mg/kg twice daily) exceeded the pre-pecified criteria for improvement in core efficacy measures compared with placebo
- The clinical benefit in the trial encompassed core symptoms of Rett syndrome and was observed in both clinician and caregiver assessments
- Meeting with FDA expected in Q1 2015 to discuss further development
- in Rett syndrome
- Results will enable applications for both Orphan Drug and Breakthrough Therapy designation
The trial was supported by the International Rett Syndrome Foundation (IRSF).