Newron Pharmaceuticals announced that top-line results from its phase 3 STARS clinical study evaluating sarizotan in patients with Rett syndrome did not demonstrate evidence of efficacy on the primary or secondary efficacy variables. As a result, Newron has decided to terminate this development program.
The STARS (Sarizotan for the Treatment of Apneas in Rett Syndrome) clinical study enrolled 129 Rett syndrome patients throughout the US, Europe, Asia and Australia for the six-month clinical trial. Patients received treatment with daily doses of 10 mg and 20 mg of sarizotan or placebo. The primary endpoint was a percentage reduction in episodes of apnea during waking time compared with placebo.
Newron has not yet released specific details but did indicate it plans to work with the Rett research community and families to share results from the trial, as well as from the Rett Syndrome International Burden of Illness Survey.
“We are very disappointed that the top-line results in the STARS study did not meet the study endpoints”, said Ravi Anand, M.D., Chief Medical Officer of Newron.” 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. 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 is planning to focus on the Phase III clinical program evaluating Evenamide in schizophrenia as well as additional pipeline candidates focused on diseases of the central and peripheral nervous system.
Sarizotan is a highly selective compound for specific serotonin or dopamine receptors that modulates the activity of these neurotransmitters in the brain. In preclinical evaluation studies, the full agonist at the serotonergic 5HT1A receptor demonstrated dramatic improvement of respiration in a number of genetic mouse models of Rett syndrome.
About Rett Syndrome
Rett syndrome is a severe neurodevelopmental disorder primarily affecting females, with an estimated prevalence of one in 10,000 females. The syndrome is characterized 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. 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. . More than 95 percent of these patients have a random mutation in the MeCP2 gene.