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Newron terminates sarizotan development in Rett syndrome

Newron Pharmaceuticals S.p.A.‘s Sarizotan failed to meet efficacy endpoints in the Phase III STARS study.

While Newron Pharmaceuticals S.p.A.’ agonist of the serotonergic 5HT1A receptor significantly reduced episodes of apnea during waking time vs placebo in mice, sarizotan did not in patients with respiratory disturbances enroled in the pivotal Phase III STARS (Sarizotan Treatment of Apneas in Rett Syndrome) study. The Milan-based company, which had licenced the programme from German Merck, lost 70% in value after it announced the termination of the clinical programme.

The STARS study qualified and enrolled 129 Rett syndrome patients 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 of the STARS study was a percentage reduction in episodes of apnea, which occurs together with hyperventilation and disordered breathingin approximately 70% of patients with Rett syndrome at some stage of their life, during waking time vs placebo.

Rett syndrome is a severe neurodevelopmental disorder primarily affecting females, with an estimated prevalence of one in 10,000 females. There are no approved treatments available. Rett 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. More than 95 percent of these patients have a random mutation in the MeCP2 gene.

Newron decided to priorize development of evenamide as the potential first add-on therapy for the treatment of patients with schizophrenia symptoms, who   are resistant to clozapine therapy.