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Potential to Become First Drug Ever Approved for Rett Syndrome

Sarizotan, a new chemical entity licensed from Merck KGaA, is a highly selective compound for specific serotonin or dopamine receptors that modulates the activity of these neurotransmitters in the brain. As Sarizotan was originally developed in another indication, the compound was licensed with an extensive safety and tolerability data package. Sarizotan could become the first drug approved for Rett syndrome, a debilitating genetic disorder with no specific treatment options. Sarizotan is targeting respiratory disturbances as primary efficacy outcome in an ongoing pivotal study. In preclinical evaluation studies, the full agonist at the serotonergic 5HT1A receptor has demonstrated dramatic improvement of respiration in  a number of genetic mouse model of Rett.

Newron is currently performing the “Sarizotan Treatment of Apneas in Rett Syndrome” (STARS) study, a potentially pivotal clinical study performed in study centers in the US, Europe, Asia and Australia to evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome suffering from respiratory symptoms. We hope that this study will prove successful, offering to Newron the opportunity to advance towards regulatory approval and to commercialize sarizotan for Rett syndrome directly in key markets.

As part of its commitment to the rare disease patient community, Newron is partnering with the global Rett community to work on the first Burden of Disease (BOD) study. The study aims to deliver data and analytics to quantify the physical, emotional and financial challenges of Rett syndrome. The learnings can help identify improved intervention programs and services designed to complement the Rett care pathway.