Market Overview

Newron Provides Update on STARS Clinical Study with Sarizotan for the Treatment of Rett Syndrome

  • Trial now expected to complete enrollment in H2 2018, with data
    readout expected in H1 2019
  • 90% of patients have continued into a long-term extension study
  • Rett syndrome is an orphan disease with no approved treatment

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 announces
that the STARS clinical study (Sarizotan Treatment of Apneas in Rett
Syndrome) in patients with Rett syndrome is expected to complete
enrollment in H2 2018. Newron expects to report results from the STARS
study in H1 2019.

Currently over 100 patients, aged six years and over, have qualified for
inclusion into the trial. Based on the current rate of screening and
qualification, Newron anticipates that randomization of the target
number of 129 patients will be completed in H2 2018. The STARS study is
being performed in patients who present with clinically significant
apneas during the course of the disease. Apneas are a cardinal feature
of Rett syndrome, present in approximately 70% of patients, and
contribute significantly to other co-morbidities, as well as leading to
a reduced quality of life. Rett syndrome, a severe neuro-developmental
orphan disease with no approved treatment options, overwhelmingly
affects girls starting at a very young age.

Only patients who experience at least 10 episodes per hour of clinically
significant apnea, (i.e. more than 10 seconds each in duration, while
awake during the day) qualify for inclusion in the study. These
recordings are performed over a 5-6-hour period per day, over 3 days per
week, with the opportunity for the patients to repeat the procedure in
the subsequent 3 weeks in case they do not qualify in the first week of
screening. Patients who meet eligibility criteria are randomized to
treatment or placebo and undergo the same recording of respiration at
home at four separate time-points during the 24-week double-blind period
of the study. The primary endpoint for the STARS study is reduction in
episodes of these apneas during waking time by 20 percent. During this
6-month study, patients will receive treatment with daily doses of 10
and 20 mg of sarizotan, or placebo.

Treatment with sarizotan has been very well tolerated, with a very low
rate of discontinuation due to adverse events or lack of efficacy.
Approximately 90 percent of the patients who have completed the 24-week
double-blind period have continued in the long-term open-label
extension. The safety of the patients in the trial is overseen by an
independent international safety monitoring board which has reviewed all
safety data and has recommended that the study be continued without any

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:

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

View Comments and Join the Discussion!