Accueil / Communiqués / Newron Completes Patient Enrollment in Pivotal STARS Study

Newron Completes Patient Enrollment in Pivotal STARS Study

Friday, February 1st 2019 at 12:00pm UTC
  • Top line results expected in Q4 2019
  • Almost all patients continued treatment with sarizotan in a
    long-term extension study
  • Positive study results may lead to the approval of the first
    drug to benefit a key symptom of Rett syndrome, a severe
    neuro-developmental orphan disease

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, today announced
the successful completion of patient enrollment in its STARS (Sarizotan
for the Treatment of Apneas in Rett Syndrome) clinical study. Newron
expects to report top line results from the STARS study in Q4 2019.

Rett syndrome, a severe neuro-developmental orphan disease with no
approved treatment options, overwhelmingly affects girls starting at a
very young age. Currently, more than 130 patients of four years or
older, have qualified for inclusion into Newron’s study, which is being
conducted at 14 centers throughout the US, Europe, Asia and Australia.
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
percent of patients, which contribute significantly to other
co-morbidities, as well as to a reduced quality of life.

Only patients who experienced clinically significant apnea, i.e. at
least 10 episodes of more than 10 seconds each per hour while awake,
qualified for inclusion in the study. The apneas were monitored and
recorded using a state-of-the-art medical device providing an objective
measure of breathing dysfunction. The recordings were performed over a
5- to 6-hour period per day, for 3 days per week, with the opportunity
for patients to repeat the procedure in the subsequent 3 weeks in case
they did not qualify in the first week of screening. During the 6-month
study, patients who met the eligibility criteria were randomized for
treatment with daily doses of 10 or 20 mg of sarizotan, or placebo.
Recordings of respiration take place at home, at four separate
time-points during the 24-week double-blind period of the study, which
is still ongoing for the last patients enrolled. The primary endpoint of
the STARS study is the percentage reduction in these episodes of apnea
during waking time compared with placebo.

Treatment with sarizotan has been very well tolerated to date, 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 study. 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 modification.

During its R&D day on October 31, 2018, Newron presented baseline data
from more than 100 patients enrolled in the STARS study, for the first
time providing an objective measure of breathing dysfunction in the home
environment over a long period of wakefulness. Data suggests that
untreated, up to 70 percent of patients experience clinically
significant apnea and minimally 10 percent of their time is spent
without breathing. In these patients, oxygen saturation falls below 90
percent between 4.2 to 24 times per hour, and the cumulative duration of
this state may last as long as 48 minutes per hour.

Anticipated clinical results from the STARS study, if positive, could
position the Company to submit a filing for marketing authorization with
the US, Canadian and European regulatory agencies. The Company continues
its plans to commercialize sarizotan in key territories and has been
working with the Rett syndrome community to build disease awareness.

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 http://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 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: 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 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.

Contacts

Newron
Stefan Weber – CEO
+39 02 6103 46 26
pr@newron.com

UK/Europe
Julia Phillips / Natalie Garland-Collins, FTI
Consulting
+44 20 3727 1000
SCnewron@fticonsulting.com

Switzerland
Martin Meier-Pfister, IRF Communications
+41
43 244 81 40
martin.meier-pfister@irfcom.ch

Germany/Europe
Anne Hennecke, MC Services
+49 211
52925222
anne.hennecke@mc-services.eu

USA
Paul Sagan, LaVoieHealthScience
+1 617 374 8800,
Ext. 112
psagan@lavoiehealthscience.com

Source: Newron Pharmaceuticals S.p.A.


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