Newron Pharmaceuticals, a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the CNS and pain, announced that the Sarizotan Treatment of Apneas in Rett Syndrome (STARS) study has been expanded to include Rett syndrome patients as young as six years of age. An amendment to allow the inclusion of the patients under the age of 13 was approved by the FDA after Newron submitted results from juvenile toxicology studies. Newron is also submitting the amendment to Health Authorities and Institutional Review Boards in other countries.

Ravi Anand, M.D., chief medical officer at Newron, commented: “Rett syndrome is a devastating condition that manifests in early childhood. This decision by the FDA to lower the age limit for inclusion of patients from six years old will allow sarizotan’s benefits to be evaluated in these younger patients before the disease has significantly progressed. Newron believes that earlier onset of treatment in Rett’s patients may be associated with less deterioration of respiratory and neurological symptoms.”

STARS is a randomized, double blind, placebo-controlled study, expected to enroll 129 patients (three groups of 43), in up to 15 centers of excellence in the USA, the UK, Italy, Australia and India. The potentially pivotal clinical study will evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome suffering from respiratory symptoms. Among the core symptoms of Rett, breathing disturbances may affect the whole body; they can have a marked effect on biochemistry, influence emotions, circulation and digestive function as well as musculoskeletal structures in the respiratory process.

The primary endpoint for the STARS study is reduction in episodes of clinically significant apneas (>10 sec) during waking time by 20 percent. During this 6-month study, patients will receive treatment with daily doses of 10 and 20 mg of sarizotan, or placebo. The Company expects to have top-line results available for release in 2018.

Sarizotan received Orphan Drug Designation for the treatment of Rett syndrome from both the European Commission and the FDA in 2015. It could become the first therapy approved for the treatment of Rett syndrome patients. Newron intends to commercialize sarizotan directly.

Besides the STARS efficacy study, Newron, as part of its commitment to the rare disease patient community, 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. These learnings can help identify improved intervention programs and services designed to complement the Rett care pathway.