MILAN & MORRISTOWN, N.J.--(BUSINESS WIRE)--Newron Pharmaceuticals S.p.A. (“Newron”) (SIX: NWRN), a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central nervous system (CNS) and pain, announced today that its Investigational New Drug (IND) application for the evaluation of sarizotan for the treatment of patients with Rett syndrome was approved by the U.S. Food and Drug Administration (FDA). Newron expects to initiate the potentially pivotal STARS (Sarizotan Treatment of Apneas in Rett Syndrome) in the third quarter of this year.
STARS is a randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome with respiratory symptoms. It will be conducted at centers of excellence in the United States, with similar centers to be added in other countries. The study protocol was designed following extensive discussions with regulatory authorities in the United States, Europe and Canada.
"The FDA approval of the STARS trial is a major step in the development of sarizotan for the treatment of cardinal abnormal respiratory symptoms of Rett syndrome, such as episodes of apnea, hyperventilation and breathing dysrhythmia, that are not only alarming and incapacitating for the patient, but also may contribute to morbidity and mortality long-term,” said Ravi Anand, M.D., Chief Medical Officer at Newron. “Newron greatly appreciates the many leading Rett syndrome researchers, experts and advocacy group leaders who provided us with significant input on the trial design for STARS, which is the first pivotal study to be performed in patients with Rett syndrome.”
Patients with Rett syndrome experience respiratory symptoms, such as episodes of apnea and hyperventilation, whose onset is as early as 3 years of age and that may persist for 10-15 years. These apneic episodes may occur as frequently as 10-60 times / hour during waking time. Regulatory endorsement of the study endpoints represents a growing recognition of the importance and contribution of respiratory symptoms.
Sarizotan has shown consistent, dramatic and statistically significant reduction in apneic episodes in genetic knock-out murine models of Rett’s syndrome that exhibit the characteristic phenotypic features of the disease. These effects were achieved with plasma levels of sarizotan that approximate those that will be achieved at the doses evaluated in the STARS study.
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 treatment of Rett Syndrome patients. Newron intends to commercialise Sarizotan directly.
About Rett Syndrome
Rett syndrome is a severe neurodevelopmental disorder primarily affecting females, with an estimated prevalence ranging from one in 10,000 females. There are no approved treatments available. Rett syndrome 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. 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. Episodes of apnea, hyperventilation and disordered breathing are found in approximately 70 percent of patients with Rett syndrome at some stage of their life.
For more information on Rett Syndrome, visit www.rettsyndrome.org
About Newron Pharmaceuticals
Newron (SIX: NWRN) is a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central nervous system (CNS) and pain. The Company is headquartered in Bresso near Milan, Italy. Xadago® (Safinamide) has received marketing authorization for the treatment of Parkinson’s Disease in the European Union and Switzerland and is commercialized by Newron’s Partner Zambon. US WorldMeds holds the commercialization rights in the US. Meiji Seika has the rights to develop and commercialize the compound in Japan and other key Asian territories. In addition to Xadago® for Parkinson’s disease, Newron has a strong pipeline of promising treatments for rare disease patients at various stages of clinical development, including sarizotan for patients with Rett syndrome and ralfinamide for patients with specific rare pain indications. Newron is also developing NW-3509 as the potential first add-on therapy for the treatment of patients with positive symptoms of schizophrenia.
For more information, please visit: www.newron.com
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