Newron’s Experimental Therapy Sarizotan Fails in Rett Syndrome Study

Rare Daily Staff

Newron Pharmaceuticals said it will terminate development of its experimental Rett syndrome drug sarizotan after top-line results from its phase 2/3 study evaluating its experimental drug showed it failed to meet the primary or secondary efficacy endpoints.

Rett syndrome is a severe rare neurodevelopmental disorder primarily affecting females, that is characterized by a loss of acquired fine and gross motor skills and the development of neurological, cognitive and autonomic dysfunction, which lead to loss of ability to conduct daily life activities, walk or communicate. Rett syndrome also is associated with a reduced life expectancy. Approximately 25 percent of the deaths in patients with Rett syndrome are possibly related to multiple cardio-respiratory dysrhythmias that result from brain stem immaturity and autonomic failure. Episodes of apnea, hyperventilation and disordered breathing are found in approximately 70 percent of patients with Rett syndrome at some stage of their life. There are no approved therapies for the disorder.

The STARS (Sarizotan for the Treatment of Apneas in Rett Syndrome) clinical study qualified and enrolled 129 Rett syndrome patients in 14 centers throughout the world for the six-month clinical trial. Patients received treatment with daily doses of sarizotan or placebo. The primary endpoint of the STARS study was a percentage reduction in episodes of apnea during waking time compared with placebo.

Newron plans to work with the Rett research community and families to share learnings from the STARS clinical study as well as from the Rett Syndrome International Burden of Illness Survey, to further advance scientific and medical understanding of this disease.

“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,” said Ravi Anand, chief medical officer of Newron. “We are currently awaiting results of additional explanatory analyses and will continue to analyze the full data set from the study to understand more about the results.”

Newron, which is focused on therapies for diseases of the central and peripheral nervous system, will turn its focus to progressing the late-stage development of its experimental drug evenamide in schizophrenia, and other pipeline candidates.

Photo: Ravi Anand, chief medical officer of Newron