Newron Supports Global Rare Disease Day® 2019

Feb. 28, 2019 12:00 UTC

Sponsoring international study to examine the burden of Rett syndrome on patients and caregivers, featured at Findacure’s Drug Repurposing for Rare Diseases Conference in the UK

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 and peripheral nervous system, is pleased to support this year’s Rare Disease Day®. Observed today, February 28, Rare Disease Day helps to raise awareness of rare diseases, and in turn improve access to treatments.

As part of its commitment to the rare disease patient community, Newron has partnered with the global Rett community in conducting the ongoing, first International Burden of Disease study. Rett syndrome is a severe neuro-developmental orphan disease with no approved treatment options and overwhelmingly affects girls starting at a very young age. Apneas are an unfortunate feature of Rett syndrome, present in approximately 70 percent of patients, and contribute significantly to other co-morbidities as well as to a reduced quality of life. The study aims to deliver data and analytics to quantify the physical, emotional and financial challenges of Rett syndrome. The results will help identify and guide improved intervention programs and services designed to complement the Rett care pathway.

Newron is also strongly supportive of the role that drug repurposing can play in accelerating the development of rare disease treatments. Newron’s sarizotan, a new chemical entity, which was initially studied in another indication, has since been repurposed and is currently being studied in the STARS (Sarizotan for the Treatment of Apneas in Rett Syndrome) clinical trial.

Findacure, a UK charity supporting the rare disease community to drive research and develop treatments, yesterday held the Drug Repurposing for Rare Diseases Conference in London, UK. At the conference, Rachael Stevenson, CEO of Reverse Rett, and Becky Jenner, CEO of Rett UK, two leading advocates for the Rett patient community, discussed the potential for improving the lives of children and adults with Rett syndrome in the UK and also shared their experiences of their daughters who participated in Newron’s STARS trial in the UK.

Dr. Richard Thompson, CEO of Findacure, said, “Drug repurposing can make use of our existing knowledge about a drug and its interactions with humans to potentially accelerate the pace of drug discovery. There is an enormous unmet need, as more than 7,000 rare diseases have been identified but only 400 have an approved treatment. Effective drug repurposing can have significant potential to bring treatments to patients with little hope of ever receiving pharmaceutical intervention.”

“Through our commitment to the Rett community, we work with caregivers, advocacy groups, physicians and government agencies to help deliver what patients need,” said Dennis Dionne, Newron Pharmaceutical’s Vice President of Commercial Affairs. “We are proud to engage in a meaningful way with Rare Disease Day, and to support the advocacy organizations that are helping to improve the quality of life for patients and their families.”

About Rare Disease Day®
Rare Disease Day takes place on the last day of February each year, a month known as having a ‘rare’ number of days, thus on February 28, 2019. The main objective of Rare Disease Day is to raise awareness among the general public and decision-makers about rare diseases and their impact on patients' lives. The campaign targets primarily the general public and seeks to raise awareness among policy makers, public authorities, industry representatives, researchers, health professionals and anyone who has a genuine interest in rare diseases. The campaign started as a European event and has progressively become a world phenomenon, with the USA joining in 2009 and participants in over 90 countries throughout the world in both 2017 and 2018. On you can find information about the thousands of events happening around the world on the last day of February.

About Rett Syndrome
Rett syndrome is a severe neuro-developmental 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. 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

Newron has successfully completed patient enrollment in the Sarizotan Treatment of Apneas in Rett Syndrome (STARS) study, a clinical study to 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 person body; they can have a marked effect on biochemistry, influence emotions, circulation and digestive function as well as musculoskeletal structures in the respiratory process.

About Newron Pharmaceuticals
Newron (SIX: NWRN) is a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central and peripheral nervous system. 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, Switzerland, the USA, Australia and Canada, and is commercialized by Newron’s partner Zambon. US WorldMeds holds the commercialization rights in the USA. 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 Evenamide as the potential first add-on therapy for the treatment of patients with positive symptoms of schizophrenia. For more information, please visit:

Important Notices
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 commercialization of its product candidates and reduce costs (including staff costs), (2) the market for drugs to treat diseases of the central nervous system 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, commercialization plans, collaborations and operations will not differ materially from the expectations set out in such forward-looking statements or underlying assumptions. Newron does not undertake any obligation to publicly update or revise forward-looking statements except as may be required by applicable regulations of the SIX Swiss Exchange, where the shares of Newron are listed. This announcement is not an offer for sale of securities in the United States, Canada, Australia or Japan or any other jurisdiction where such an offer or solicitation would otherwise be unlawful. The securities referred to herein may not be sold in the United States absent registration or an exemption from registration under the U.S. Securities Act of 1933, as amended. This document does not contain or constitute an offer or invitation to purchase or subscribe for any securities of Newron and no part of this document shall form the basis of or be relied upon in connection with any contract or commitment whatsoever.


Stefan Weber – CEO
+39 02 6103 46 26

Julia Phillips / Natalie Garland-Collins, FTI Consulting
+44 20 3727 1000

Martin Meier-Pfister, IRF
+41 43 244 81 40

Anne Hennecke, MC Services
+49 211 52925222

Paul Sagan, LaVoieHealthScience
+1 617 374 8800, Ext. 112


Source: Newron Pharmaceuticals S.p.A.

Back to news