Sarizotan is designed to treat the symptom of respiratory disturbances (apnoea) associated with Rett syndrome. Apnoea leads to low oxygen levels, affecting patient morbidity and perhaps accelerating disease progression. It is estimated ~70% of Rett patients experience clinically significant apnoea.
Sarizotan works by stimulating serotonin and dopamine receptors. By replacing some of the missing serotonin in the brain and spinal cord, it is expected that the drug might help restore normal breathing rhythm in patients with Rett syndrome.
The primary endpoint of Newron’s (likely pivotal) trial is the reduction in respiratory abnormality in patients with Rett syndrome, measured over 24 weeks. This will be assessed as the percentage reduction in the number of clinically significant (>10secs) apnoea episodes per hour while awake, measured using an ambulatory home monitoring system.
Sarizotan is an oral treatment, administered twice-daily.
Newron intends to commercialise sarizotan in the US by itself. It has yet to define its ROW commercialization strategy.
Trofinetide is designed to treat the core symptoms of Rett syndrome.
Trofinetide works by reducing neuroinflammation and supporting synaptic function. It has been designed to mimic the functions of glypromate (GPE), a product of IGF-1 breakdown but has more favorable pharmacological properties than GPE and remains for longer periods in the bloodstream. Both IGF-1 and GPE are important to maintain the biological balance required for normal activity in the developing brain and to respond to disease, stress, and injury in the mature brain.
Acadia/Neuren’s pivotal trial is investigating whether, over 12 weeks, trofinetide lessens Rett syndrome’s neurobehavioral symptoms, as measured by caregivers with the Rett Syndrome Behaviour Questionnaire and by clinicians using the Clinical Global Impression Scale-Improvement.
Trofinetide is an oral treatment, administered twice daily.
Trofinetide will be commercialized in North America by licensee, Acadia Pharmaceuticals. Neuren is seeking to outlicense trofinetide for ROW.
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