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Press Release: Santhera Announces Publication of Long-term Idebenone Data from SYROS Study in Duchenne Muscular Dystrophy

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*DJ Santhera Announces Publication of Long-term Idebenone Data from SYROS Study in Duchenne Muscular Dystrophy

(MORE TO FOLLOW) Dow Jones Newswires

November 19, 2019 01:00 ET ( 06:00 GMT)

Pratteln, Switzerland, November 19, 2019 Santhera Pharmaceuticals
(SIX: SANN) announces publication of the previously reported SYROS study
data in prominent peer-reviewed medical journal
Neuromuscular Disorders [1], demonstrating long-term efficacy with
idebenone in slowing respiratory function loss in patients with Duchenne
muscular dystrophy (DMD) under routine clinical care.

The publication reports data collected as retrospective cohort study
(SYROS) from 18 DMD patients not using glucocorticoids who were treated
with idebenone (900 mg/day) under Expanded Access Programs (EAPs) after
having completed the Phase III DELOS study. The objective was to assess
the respiratory function evolution during periods of treatment with
idebenone ('On-Idebenone') compared to periods without idebenone
treatment ('Off-Idebenone') in the same patients. Respiratory function
was measured as forced vital capacity (FVC) or peak expiratory flow
(PEF), both expressed as percent of predicted (FVC%p, PEF%p).

This study collected data from DMD patients receiving long-term idebenone
treatment under routine clinical care for an average of 4.2 years (range
2.4 to 6.1 years) after completion of the DELOS study.

In the patients who were 'Off-Idebenone' before the EAPs, the annual rate
of decline in FVC%p (primary endpoint) was reduced by approximately 50%
from -7.4% (95% CI: -9.1, -5.8) 'Off-Idebenone' to -3.8% (95% CI: -4.8,
-2.8) 'On-Idebenone'. Similarly, the annual rate of decline in PEF%p was
reduced from -5.9% 'Off-Idebenone' to -1.9% 'On-Idebenone'.

The annual rate of decline in FVC%p and PEF%p remained stable year after
year for the entire follow-up time of up to six years and was
consistently lower than the decline in matched untreated patients from a
natural history study.

During 'On-Idebenone', the rate of bronchopulmonary adverse events was
reduced (0.10 events per person-year) compared with 'Off-Idebenone' (0.33
events per person-year). Similarly, the rate of hospitalizations due to
respiratory infections or related disorders was reduced under idebenone
treatment (0.06 hospitalization events per person-year 'On-Idebenone'
versus 0.15 'Off-Idebenone').

"Reducing the annual rate of decline in respiratory function during
idebenone treatment appears to be clinically relevant as it was
associated with a smaller number of bronchopulmonary complications and
hospitalization for respiratory events," said lead author Laurent
Servais, MD, PhD, Professor of Neuromuscular Paediatric Diseases at the
University of Oxford (MDUK Oxford Neuromuscular Center, department of
Paediatrics) and Head of the Neuromuscular Centre (Liège, Belgium).
"Furthermore, a sustained reduction in the rate of decline in FVC%p
indicates a potential therapeutic benefit during long-term treatment as
it could result in a delay in the need of assisted ventilation."

"Data from this long-term follow-up period in patients receiving
idebenone under routine clinical care provide a highly valuable data set
complementing the previously reported body of evidence, especially the
positive Phase III DELOS trial [2]. The totality of available data
demonstrate that idebenone preserves respiratory function and holds
disease-modifying therapeutic potential," said principal investigator
and corresponding author Gunnar M. Buyse, MD, PhD, Professor of
Pediatrics and Child Neurology, University Hospitals Leuven (Leuven,

About Duchenne Muscular Dystrophy

DMD is one of the most common and devastating types of progressive
muscle weakness and degeneration starting at an early age and leading to
early morbidity and mortality due to respiratory failure. It is a
genetic, degenerative disease that occurs almost exclusively in males
with an incidence of up to 1 in 3,500 live male births worldwide. DMD is
characterized by a loss of the protein dystrophin, leading to cell
damage, impaired calcium homeostasis, elevated oxidative stress and
reduced energy production in muscle cells. With age, progressive
respiratory muscle weakness affecting thoracic accessory muscles and the
diaphragm causes respiratory disease, impaired clearance of airway
secretions, recurrent pulmonary infections due to ineffective cough, and
eventually respiratory failure. There is currently no treatment approved
for slowing loss of respiratory function in patients with DMD.

About Idebenone in Duchenne Muscular Dystrophy

Idebenone is a synthetic short-chain benzoquinone and a cofactor for the
enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating
mitochondrial electron transport, reducing and scavenging reactive
oxygen species (ROS) and supplementing cellular energy levels.

DELOS was a Phase III, double-blind, placebo-controlled 52-week study
which randomized 64 patients, not taking concomitant steroids, to
receive either idebenone (900 mg/day) or matching placebo. The study met
its primary endpoint, the change from baseline in peak expiratory flow
(PEF) expressed as percent of predicted, which demonstrated that
idebenone can reduce the loss of respiratory function [2, 3]. Further
pre-specified and post-hoc analyses of the DELOS study outcome have been
published [4-7].


[1] Servais L, et al. Long-term data with idebenone on respiratory
function outcomes in patients with Duchenne muscular dystrophy.
Neuromuscular Disorders 2019; DOI:

[2] Buyse GM, et al. Lancet 2015; 38 5:17 48-57.

[3] Meier T, et al. Neuromuscular Disorders 2017; 27:30 714.

[4] McDonald CM, et al. Neuromuscular Disorders 2016; 26:47 3-80.

[5] Buyse GM, et al. Pediatric Pulmonology 2017; 5 2:50 8-15.

[6] Mayer OH, et al. J Neuromuscular Diseases 2017; 4:18 9-98.

[7] Buyse GM, et al. J Neuromuscular Diseases 2018; 5:41 930.

About Santhera

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical
company focused on the development and commercialization of innovative
medicines for rare neuromuscular and pulmonary diseases with high unmet
medical need. Santhera is building a Duchenne muscular dystrophy (DMD)
product portfolio to treat patients irrespective of causative mutations,
disease stage or age. A marketing authorization application for
Puldysa(R) (idebenone) is currently under review by the European
Medicines Agency. Santhera has an option to license vamorolone, a
first-in-class dissociative steroid currently investigated in a pivotal
study in patients with DMD to replace standard corticosteroids. The
clinical stage pipeline also includes POL6014 to treat cystic fibrosis
(CF) and other neutrophilic pulmonary diseases, as well as omigapil and
an exploratory gene therapy approach targeting congenital muscular
dystrophies. Santhera out-licensed ex-North American rights to its first
approved product, Raxone(R) (idebenone), for the treatment of Leber's
hereditary optic neuropathy (LHON) to Chiesi Group. For further
information, please visit

Raxone(R) and Puldysa(R) are trademarks of Santhera Pharmaceuticals.

For further information please contact:

public-relations@santhera.com or

Eva Kalias, Head External Communications

Phone: +41 79 875 27 80


Disclaimer / Forward-looking statements

This communication does not constitute an offer or invitation to
subscribe for or purchase any securities of Santhera Pharmaceuticals
Holding AG. This publication may contain certain forward-looking
statements concerning the Company and its business. Such statements
involve certain risks, uncertainties and other factors which could cause
the actual results, financial condition, performance or achievements of
the Company to be materially different from those expressed or implied
by such statements. Readers should therefore not place undue reliance on
these statements, particularly not in connection with any contract or
investment decision. The Company disclaims any obligation to update
these forward-looking statements.

# # #


2019 11 19_Syros Publication NMD_e_final

(END) Dow Jones Newswires

November 19, 2019 01:00 ET ( 06:00 GMT)

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