Newron drops development of sarizotan in Rett syndrome

Shares in Newron Pharmaceuticals have taken a massive hit from the failure of sarizotan to show a clear benefit in patients with Rett syndrome, a severe neurodevelopmental disorder primarily affecting females.

The company revealed top-line data from the pivotal Phase II/III STARS trial of the drug, an experimental 5-HT1A receptor agonist and D2 receptor antagonist, showing that it was not effective on primary or secondary endpoints.

The primary endpoint was a percentage reduction in episodes of apnoea during waking time versus placebo, while secondary goals related to respiratory symptoms and motor behaviour.

Newron confirmed that on the back of the data it will discontinue sarizotan's development programme.

Ravi Anand chief medical officer of Newron, said the company is “very disappointed” with the outcome of the trial.

“The results of this well designed and executed study, based on highly promising data from a genetic model of Rett syndrome in mice, indicate the difficulties inherent in translating effects in animal models to human clinical studies,” he noted.

“We are currently awaiting results of additional explanatory analyses and will continue to analyse the full data set from the study to understand more about the results”.

The news will come as a particular blow to the Rett community, as there are currently no approved treatments for the condition, which is characterised by a loss of acquired fine and gross motor skills and the development of neurological, cognitive and autonomic dysfunction, which leads to loss of ability to conduct daily life activities, walk or communicate.

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