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Press Release

Newron Pharmaceuticals Announces Advisory Board Recommendations and Rett Congress Findings

Newron Pharmaceuticals
Posted on: 07 Nov 17

-       Advisory Board Characterizes Illness and Treatment Patterns

-       Rett Congress Reviews Multi-Organ Impact of Single Symptoms


Milan, Italy & Morristown, N.J. – November 7, 2017 - 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, today announced recommendations from its advisory board, who are  evaluating the burden of disease experienced by patients with Rett syndrome and their families, as well as an update from the recent European Rett Syndrome Congress in Berlin, Germany. Newron is currently developing sarizotan, the first new chemical entity (NCE) in a late Phase 3 clinical trial in Rett syndrome.


At the Rett Congress, Newron’s Burden of Disease advisory board reached a consensus on the concept elicitation interviews which are being used to develop a survey that will be distributed internationally to caregivers and allied healthcare professionals. The study assesses the impact of symptoms on daily activities while also capturing the frequency of clinical events and resource utilization as reported by caregivers for Rett syndrome patients.  


At the advisory board meeting, all significant symptoms of Rett syndrome were reviewed including breathing abnormalities, mobility, communication, sleep disturbances, bone health, and other symptoms of this multi-organ disease. Speakers agreed that in Rett syndrome patients, one symptom can have a significant impact on other symptoms. Girish Sharma, MD, Pulmonologist, Rush University Medical Center, commented, “Ongoing chronic respiratory abnormalities, including acute episodes such as hyperventilation, may result in biochemical and metabolic changes with an impact on multiple systems and stress levels in the patient and caregiver. We look forward to utilizing the Burden of Disease study to help understand gaps in managing a patient’s condition throughout the whole body.”

The well attended European Rett Syndrome Congress also included an oral presentation entitled “Important symptoms of Rett syndrome and their impact: the perspective of parents and healthcare providers.” During the podium session, Dr. Kate Williams, Senior Consultant, Clinical Outcomes Assessment at PAREXEL International, presented an update on the findings from the concept elicitation interviews with caregivers of individuals with Rett syndrome and healthcare providers. The findings from the interviews demonstrated that some symptoms, for example respiratory abnormalities, impact other symptoms such as gastrointestinal gas and subsequent pain. Other symptoms, such as mobility and muscle tone, were found to impact each other bi-directionally. 

Separately, Newron hosted a roundtable discussion on the need for new tools to assess the health economic value of orphan drugs treating rare disease that impact multiple organ systems. “Although only a small number of patients suffer from each rare disease, collectively rare diseases are a significant medical and social issue,” said Dennis Dionne, VP of Commercial Affairs at Newron. “The advent of improved methods for assessing the value of orphan drugs will enable more and better therapies for rare diseases to be made available to patients.”


About Rett Syndrome

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. 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


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 and the USA, 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


For more information, please contact



Stefan Weber – CEO

+39 02 6103 46 26



Julia Phillips, FTI Consulting

+44 20 3727 1000



Martin Meier-Pfister, IRF Communications

+41 43 244 81 40



Anne Hennecke, MC Services

+49 211 52925222



Beth Kurth, LaVoieHealthScience

+1 617 374 8800, Ext. 106


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 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. 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. Newron does not intend to register any of its securities in the United States or to conduct a public offering of its securities in the United States. 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.

Editor's Details

Mike Wood

Last updated on: 07/11/2017

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