MILAN–(BUSINESS WIRE)–June 25, 2015–
Newron Pharmaceuticals S.p.A. (“Newron”), a research and development company focused on novel CNS and pain therapies, announces that it has received a positive opinion for its New Chemical Entity Sarizotan for the treatment of Rett Syndrome (RTT) from the Committee for Orphan Medicinal Products (COMP) from the European Medicines Agency (EMA). An application for Orphan Designation has also been filed in the US.
Rett Syndrome is a severe neurodevelopmental disorder primarily affecting females with an estimated prevalence ranging from 1:10-20,000 females. There are no approved treatments available. RTT is characterized by a loss of acquired fine and gross motor skills, development of neurological, cognitive and autonomic dysfunction leading to loss of ability to conduct daily life activities, ambulate or communicate. RTT is associated with a reduced life expectancy. Approximately 25% of the deaths in patients with RTT are possibly related to multiple cardio-/respiratory dysrhythmias that result from brain stem immaturity and autonomic failure. More than 95% of the patients present with a mutation in the MeCP2 gene.
Prevalence of episodes of apnea, hyperventilation, disordered breathing, etc. are found in approximately 70% of the patients at some stage of their life. Sarizotan, a 5HT1A agonist, and D2 agonist/antagonist has been associated with a 70-85% reduction of apneas and hyperventilation episodes in a genetic (MeCP2) knockout model, both after acute and chronic dosing. Sarizotan has been fully characterized in preclinical studies evaluating its toxicological effects and metabolic profile, without any significant safety findings.
Ravi Anand, Newron’s CMO, stated: “Newron plans to demonstrate the reduction of episodes of apnea and hyperventilation in patients with RTT by Sarizotan. This reduction in respiratory symptoms is likely to improve quality of life of patients, caregivers and in long term treatment may reduce secondary complications and increase longevity. Newron is currently in advanced discussions with regulatory authorities in Europe, the US and Canada on the proposed clinical development program.”
An orphan designation allows a pharmaceutical company to benefit from incentives from the European Union to develop a medicine for a rare disease, such as reduced fees and protection from competition once the medicine is placed on the market. Applications for orphan designation are examined by the COMP, which adopts an opinion that is forwarded to the European Commission.
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. Marketing authorization in the EU for Xadago® (safinamide) was granted by the EU Commission in February 2015, following the recommendation by the Committee for Medicinal Products for Human Use (CHMP) to approve the compound in the EU on Dec. 19, 2014. The New Drug Application NDA to the U.S. FDA, as informed early March, has been accepted for filing, after being re-submitted by Newron on Dec. 26, 2014. In March 2014, Zambon, a partner of Newron, submitted a MAA to Swissmedic. Zambon has the rights to develop and commercialize safinamide globally, excluding Japan and other key Asian territories where Meiji Seika has the rights to develop and commercialize the compound. Newron’s additional projects are based on highly promising treatments for rare disease patients and are at various stages of clinical development, including sarizotan for patients with Rett syndrome, sNN0031 for patients with Parkinson’s disease, non-responsive to oral drug treatments, sNN0029 for patients with ALS 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.
This document contains forward-looking statements, including (without limitation) about (1) Newron’s ability to develop and expand its business, successfully complete development of its current product candidates and current and future collaborations for the development and commercialisation of its product candidates and reduce costs (including staff costs), (2) the market for drugs to treat CNS diseases and pain conditions, (3) Newron’s anticipated future revenues, capital expenditures and financial resources, and (4) assumptions underlying any such statements. In some cases these statements and assumptions can be identified by the fact that they use words such as “will”, “anticipate”, “estimate”, “expect”, “project”, “intend”, “plan”, “believe”, “target”, and other words and terms of similar meaning. All statements, other than historical facts, contained herein regarding Newron’s strategy, goals, plans, future financial position, projected revenues and costs and prospects are forward-looking statements.
By their very nature, such statements and assumptions involve inherent risks and uncertainties, both general and specific, and risks exist that predictions, forecasts, projections and other outcomes described, assumed or implied therein will not be achieved. Future events and actual results could differ materially from those set out in, contemplated by or underlying the forward-looking statements due to a number of important factors. These factors include (without limitation) (1) uncertainties in the discovery, development or marketing of products, including without limitation negative results of clinical trials or research projects or unexpected side effects, (2) delay or inability in obtaining regulatory approvals or bringing products to market, (3) future market acceptance of products, (4) loss of or inability to obtain adequate protection for intellectual property rights, (5) inability to raise additional funds, (6) success of existing and entry into future collaborations and licensing agreements, (7) litigation, (8) loss of key executive or other employees, (9) adverse publicity and news coverage, and (10) competition, regulatory, legislative and judicial developments or changes in market and/or overall economic conditions.
Newron may not actually achieve the plans, intentions or expectations disclosed in forward-looking statements and assumptions underlying any such statements may prove wrong. Investors should therefore not place undue reliance on them. There can be no assurance that actual results of Newron’s research programmes, development activities, commercialisation plans, collaborations and operations will not differ materially from the expectations set out in such forward-looking statements or underlying assumptions.
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Stefan Weber, CEO, +39 02 6103 46 26
Julia Phillips, +44 (0)20 3727 1000
Martin Meier-Pfister, +41 43 244 81 40
David Connolly, +1-617-374-8800, Ext. 108
Investors and analysts
Stefan Weber, CEO, +39 02 6103 46 30
Kristina Coppola, +1-617-374-8800, Ext. 105
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