Newron to Evaluate Burden of Disease for Rett Syndrome Patients and Families
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, today announced that it is sponsoring a
study to evaluate the burden of disease experienced by patients with Rett syndrome and their families.
The announcement was made during the international 2016 Rettsyndrome.org Research Symposium, held in Itasca, Illinois, USA, from
June 22–24, 2016. The expert advisory panel governing the study is comprised of preeminent Rett syndrome specialists,
Rettsyndrome.org advocacy leaders and patient families.
“This will be the first international study to characterize the disease, including disease - related quality of life, frequency
of clinical events and resource utilization that will evaluate the burden and impact of Rett syndrome on patients and families. The
study will provide detailed information currently unavailable to help guide physicians, caregivers and policy experts about the
human and financial cost of this devastating disease,” said Alan K. Percy, M.D., Professor of Pediatric Neurology, Department of
Neurobiology at the University of Alabama (UAB) School of Medicine, Birmingham, Alabama, USA., Director of the UAB Sparks Clinics
Rett Syndrome Research Program and former Director of the American Board of Psychiatry and Neurology.
Rett syndrome is characterized by severe neurological, respiratory, cognitive, and behavioral symptoms. The rare disease,
primarily affecting approximately one in 10,000 females, leads to severe physical and psychological disabilities and many patients
require lifelong 24-hour care. However, the impact of the disease on the amount and quality of care required for the management of
these patients and the burden of care imposed on the families is currently unknown.
The study will be comprised of two global surveys, one to be completed by 500 caregivers and the other by 50 physicians. Both
will examine patient burden, with the caregiver survey additionally evaluating caregiver burden. The surveys are being developed in
accordance with regulatory guidance, with the final versions being used for data collection in the United States, the United
Kingdom, Italy and Germany.
“This collaborative project is a great example of how advocacy and academia can work together with private and public sectors to
gain further insight into Rett syndrome, allowing us to share this insight with the communities affected by this disease,” said
Stefan Weber, CEO of Newron Pharmaceuticals.
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 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.
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 up-date 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 document does not contain or constitute an offer or invitation to purchase or subscribe
for any securities of Newron and no part of it shall form the basis of or be relied upon in connection with any contract or
commitment whatsoever.
Media
Newron
Stefan Weber, +39 02 6103 46 26
CEO
pr@newron.com
or
UK/Europe
FTI Consulting
Julia Phillips, +44 (0)20 3727 1000
or
Switzerland
IRF Communications
Martin Meier-Pfister, +41 43 244 81 40
or
Germany
MC Services AG
Anne Hennecke, +49 211 52925222
anne.hennecke@mc-services.eu
or
USA
LaVoieHealthScience
Alison Chen, +1 617-374-8800, Ext. 104
achen@lavoiehealthscience.com
or
Investors and Analysts
Newron
Stefan Weber, +39 02 6103 46 26
CEO
pr@newron.com
or
UK/Europe
FTI Consulting
Julia Phillips, +44 (0)20 3727 1000
or
Switzerland
IRF Communications
Martin Meier-Pfister, +41 43 244 81 40
or
Germany/Europe
MC Services AG
Anne Hennecke, +49 211 52925222
anne.hennecke@mc-services.eu
or
USA
LaVoieHealthScience
Beth Kurth, +1 617-374-8800, Ext. 109
bkurth@lavoiehealthscience.com
View source version on businesswire.com: http://www.businesswire.com/news/home/20160627005627/en/