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Press Release: Santhera to Present Long-Term Data -2-

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*DJ Santhera to Present Long-Term Data with Puldysa(R) (Idebenone) in Duchenne Muscular Dystrophy at Upcoming Medical Congresses

(MORE TO FOLLOW) Dow Jones Newswires

September 17, 2019 01:00 ET ( 05:00 GMT)

Press Release: Santhera to Present Long-Term Data with Puldysa(R) (Idebenone) in Duchenne Muscular Dystrophy at Upcoming Medical Congresses

Pratteln, Switzerland, September 17, 2019 Santhera Pharmaceuticals
(SIX: SANN) will present data on the therapeutic effects of long-term
treatment with Puldysa(R) (idebenone) in Duchenne muscular dystrophy
(DMD) at the forthcoming medical conferences of the European Paediatric
Neurology Society (EPNS), European Respiratory Society (ERS) and World
Muscle Society (WMS). Clinical experts will present multiple posters on
the evolution of respiratory function and therapeutic effect of
idebenone in patients with DMD.

"It is very rewarding to have the opportunity to share the comprehensive
data package including the new long-term data at such reputable medical
congresses," said Kristina Sjöblom Nygren, MD, Chief Medical
Officer and Head of Development of Santhera. "The data we assembled to
date indicate a disease modifying potential of Puldysa which is highly
relevant for DMD patients in respiratory decline with currently no
alternative treatment options."

Leading clinical experts will present aggregate data and new analyses
from Santhera's Phase II (DELPHI) study, the long-term DELPHI-Extension
study, the pivotal Phase III (DELOS) study [1-5] and the SYROS study in
which patients were treated with idebenone for up to 6 years. In summary,
these data indicate that:

Long-term treatment with idebenone can result in a consistent and
sustained reduction in the rate of respiratory function decline.

Slowing the deterioration of respiratory function is of predictive value
for important patient-relevant clinical outcomes such as delaying the
time to needing assisted ventilation.

The risk of bronchopulmonary adverse events can be reduced during
long-term treatment with idebenone, with lower occurrence and severity of
clinically relevant pulmonary morbidity.

Treatment outcomes were consistent between two randomized studies and
their respective long term data collections, supporting the robustness of
the observed treatment effect and the potential of idebenone to preserve
respiratory function in DMD.

Conference posters will be made available the day after each respective
conference on Santhera's website at

The full list of posters and symposia can be found below:


EPNS 13th European Paediatric Neurology Society Congress

September 17-21, 2019 | Megaron Athens International Conference Centre |
Athens, Greece


"Targeting respiratory dysfunction in DMD"

September 20, 2019 | 13:00 - 14:30 | Mitropoulos Hall

Welcome and introduction Argirios Dinopoulos, MD, PhD (Athens, Greece)

Respiratory function decline as a predictor of clinical outcomes Oscar
H Mayer, MD (Philadelphia, USA)

Altering the course of respiratory dysfunction in DMD Laurent Servais,
MD, PhD (Oxford, UK and Liège, Belgium)

Summary, Q & A and Close Argirios Dinopoulos, MD, PhD


Poster Session 11: Neuromuscular

September 20 | 12:15 - 13:00 | Poster area

Poster #140: Servais et al.: Consistent long-term effect of idebenone on
the rate of respiratory function decline in advanced patients with
Duchenne muscular dystrophy (DMD)

Poster #138: Servais et al.: Long term analysis of the rate of
respiratory function decline in patients with Duchenne muscular dystrophy
(DMD) in a real-world setting: The SYROS study


ERS European Respiratory Society International Congress

September 28 to October 2, 2019 | IFEMA Exhibition Centre | Madrid,

Oral abstract presentation

Rare and ultra-rare diseases and the lungs: updates and new perspectives

October 2, 2019 | 11:45 - 12:15 | Session 551, 9B

OA5330: Mayer et al.: Evaluating the effect of long-term idebenone
treatment on respiratory morbidity in patients with Duchenne muscular
dystrophy (DMD)

OA5331: Mayer et al.: Long term efficacy: Idebenone reduces the rate of
both inspiratory and expiratory functional loss in Duchenne muscular
dystrophy (DMD)

ePosters of the oral presentations will be available throughout the
congress in the poster hall.


WMS 24th International Annual Congress of the World Muscle Society

October 1-5, 2019 | Tivoli | Copenhagen, Denmark


"Beyond the wheelchair: Targeting respiratory dysfunction in DMD"

October 2, 2019 | 18:30 - 20:00 | Room Axelborg

Welcome and introduction Eugenio Mercuri, MD, PhD (Rome, Italy)

Respiratory function decline as a predictor of clinical outcomes:
Insights from natural history Craig M. McDonald, MD (Sacramento, CA,

Beyond the wheelchair: Measuring clinical significance for patients
Laurent Servais, MD, PhD (Oxford, UK and Liège, Belgium)

What should we expect for the treatment of respiratory dysfunction in
DMD: rate change as the therapeutic goal? Eugenio Mercuri, MD, PhD

Q&A and Close Eugenio Mercuri, MD, PhD


Poster Session DMD clinical (P.258-268)

October 4, 2019 | 15:00 - 16:15 | Poster area

Poster #P261: Buyse et al.: Consistent long-term effect of idebenone in
reducing respiratory function decline in advanced patients with Duchenne
muscular dystrophy (DMD)

Poster #P264: Servais et al.: SYROS study long-term reduction in rate
of respiratory function decline in patients with Duchenne muscular
dystrophy (DMD) treated with idebenone



[1] Buyse et al. (2015), The Lancet 38 5:17 48-1757

[2] McDonald et al. (2016), Neuromuscular Disorders 26:47 3-480

[3] Buyse et al. (2017), Pediatric Pulmonology 5 2:50 8-515

[4] Mayer et al. (2017), Journal of Neuromuscular Diseases 4:18 9-198

[5] Buyse et al. (2018), Journal of Neuromuscular Diseases 5: 419-430

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 slow the loss of respiratory function. Supportive data for
idebenone were shown in the Phase II double-blind, placebo-controlled
DELPHI study and its 2-year open-label extension study (DELPHI-E).

SYROS was a prospectively planned, retrospective collection of long-term
respiratory function data from 18 patients who completed the DELOS study
and subsequently received idebenone (900 mg/day) under Expanded Access
Programs (EAPs). The SYROS study showed that the previously observed
beneficial effect of idebenone in reducing the rate of respiratory
function decline was maintained for up to six years during treatment.

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

(MORE TO FOLLOW) Dow Jones Newswires

September 17, 2019 01:00 ET ( 05:00 GMT)

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 09 17_DMDconferences_e_finalx

(END) Dow Jones Newswires

September 17, 2019 01:00 ET ( 05:00 GMT)

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