We now know what Rett is, what causes it, where it’s located. We have proven it’s reversible and for the very first time, we potentially have a treatment that could profoundly impact the disorder.
Crucially, improvements were shown after less than 2 months. Because of the way Trofinitide works, we could expect to see continued improvements over a longer period of time.
This will be the purpose of a phase 3 trial.
What is Trofinetide?
Trofinetide is designed to improve nerve connections in the brain. It modifies Rett biology which means it tackles more than one symptom at a time.
I previously wrote an article that explains Trofinetide and what it does in greater detail. I’ve put a link at the end of the blog.
What was the result of the phase 2 Trial?
The reason for trial was to prove that Trofinetide is safe for use in the younger Rett population (Paediatric).
Safety for older women was already proven in an earlier trial.
It also showed gains to the baseline which was measured by caregivers and clinicians. (Phase 3 will be an actual study of these improvements.)
It showed improvements for the largest dose, compared to the placebo, both statistically and clinically.
This improvement continued right through the treatment, to when it stopped. This suggests the may be more benefits with longer treatment times.
Caregivers and Clinicians spoke of gains from 15-16% above the baseline in 59 Days.
What happens now?
First we go through a Toxicity study (Safety Assessment), and in 2018 3rd phase trials start.
After that Neuren will apply for a New Drug Application with the FDA, which, hopefully, will be approved.
Those should be the hurdles to Trofinetide being distribution ready.
Trofinetide potentially offers measurable change to every person living with Rett syndrome.
My previous Trofinetide blog(which goes into greater detail about what it is) can be found here
Trofinetide is the lead product from Neuren Pharmaceuticals, a biopharmaceutical company developing new therapies for brain injury, neurodevelopmental and neurodegenerative disorders.
The Trofinetide trials are sponsored by Rettsyndrome.org