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2015-04-21

Santhera Pharmaceuticals Holding AG: Santhera's Positive Phase III Trial (DELOS) in Patients with Duchenne Muscular Dystrophy Published in The Lancet

Santhera Pharmaceuticals Holding AG / Santhera's Positive Phase III Trial
(DELOS) in Patients with Duchenne MuscularDystrophy Published in The Lancet .
Processed and transmitted by NASDAQ OMX Corporate Solutions.The issuer is
solely responsible for the content of this announcement.
First successful Phase III trial in DMD shows Raxone®/Catena®preserves
respiratory function

Liestal, Switzerland, April 21, 2015 - Santhera Pharmaceuticals (SIX: SANN)
announced that the full results of the double-blind placebo-controlled Phase
III trial (DELOS) demonstrating efficacy and safety of Raxone®/Catena®(INN:
idebenone) in patients with DMD have been published inThe Lancet
(Lancet 2015; 385: 1748-57. Online publication:
http://dx.doi.org/10.1016/S0140-6736(15)60025-3).

The results of the DELOS trial demonstrated that Raxone/Catena significantly
reduced the annual decline in Peak Expiratory Flow (PEF as percent predicted,
PEF%p) by 66% compared to patients taking placebo. Other respiratory function
endpoints such as Forced Vital Capacity (FVC) and Forced Expiratory Volume
(FEV1) corroborated these results and showed a consistent pattern with
treatment differences supporting efficacy of Raxone/Catena over placebo in
the preservation of respiratory function. Researchers concluded that
Raxone/Catena represents a new treatment option for DMD patients.

"Publication of the DELOS trial outcome inThe
Lancet,
one of the most prestigious medical journals worldwide, is an extraordinary
tribute to this first ever successful phase III trial in DMD", saidGunnar M.
Buyse
, MD, PhD, Professor of Child Neurology at the University Hospitals Leuven
(Belgium) and Principal Investigator for the DELOS trial and lead author of
the publication. "It's also a tribute to the hard work of so many scientists,
patients and families involved in the 10 years of innovative research in
which we have brought idebenone from the lab bench to the patient.
Statistically significant and clinically relevant outcomes of primary and
secondary endpoints coherently demonstrated that Raxone/Catena reduced the
loss of respiratory function and that it was safe and well tolerated. I am
very enthusiastic about the positive data from the trial which demonstrate
that this drug represents a suitable treatment option to ameliorate a
life-threatening complication of the disease."

"With morbidity and mortality in DMD being associated with progressive
restrictive lung disease and irreversible loss of lung function, these
findings represent an important treatment effect and are of major clinical
relevance for patients with DMD", addedCraig McDonald
, MD, Professor and Chair of the Department of Physical
Medicine&Rehabilitation at UC Davis (USA), investigator of the DELOS trial
and co-author of theLancet
publication.

"The degree of slowing of respiratory function loss demonstrated in DELOS is
of major clinical relevance for patients with DMD", commentedNicholas Coppard
, PhD, SVP Development at Santhera. "Based on this benefit and its
well-established safety profile, we are very excited about the prospects of
Raxone/Catena as a treatment option for DMD patients and we are currently
preparing the regulatory filing dossier for application of marketing
authorization both in the US and Europe."

About the DELOS trial

DELOS was a Phase III, double-blind, placebo-controlled trial which randomized
and treated 64 European and US DMD patients not receiving concomitant
corticosteroids. Patients 10-18 years of age received either Raxone/Catena
tablets (900 mg/day) or matching placebo for 52 weeks. The primary endpoint
was change in Peak Expiratory Flow % predicted (PEF%p) from baseline to week
52. PEF%p declined significantly (-9.01%p; 95% CI: -13.2, -4.8; p<0.001) from
baseline to week 52 in the placebo group compared to a non-significant
decline (-3.05%p; 95% CI: -7.1, 0.97; p=0.134) in the Raxone/Catena group,
resulting in a statistically significant difference between treatment groups
of 5.96%p (95% CI: 0.16, 11.8; p=0.044) at week 52 and representing a 66%
reduction in loss of PEF%p. A statistically significant treatment effect was
also seen at week 26 (p=0.007) and week 39 (p=0.034) and across all
assessment timepoints (p=0.018). Data for the primary endpoint were robust
across multiple sensitivity analyses and supported by positive outcomes of
additional respiratory endpoints.

About Duchenne Muscular Dystrophy (DMD)

DMD is one of the most common and devastating types of muscle degeneration and
results in rapidly progressive muscle weakness. It is a genetic, degenerative
disease that is inherited in an X-linked recessive mode with an incidence of
up to 1 in 3,500 live born males 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. This
results in progressive muscle weakness and wasting and early morbidity and
mortality due to cardio-respiratory failure. Currently, glucocorticoid
steroids are the only available medical treatment that can slow the decline
in muscle strength and function irrespective of the disease-causing mutation.
However, the effect is only partial and clinical use is limited by well-known
side effects caused by steroids. A recent study showed that ~42% of DMD
patients 10 years and older had either never used steroids or have
discontinued their use.

About Idebenone in Duchenne Muscular Dystrophy

Raxone/Catena (idebenone) is a synthetic short-chain benzoquinone and a
substrate for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of
stimulating mitochondrial electron transport and supplementing cellular
energy levels. A prior phase II randomized placebo-controlled trial (DELPHI)
demonstrated trends for beneficial effects of Raxone/Catena on early
functional cardiac and respiratory parameters. An important finding of the
DELPHI trial was that patients treated with idebenone stabilized in PEF%p, a
marker of expiratory muscle strength, compared to patients receiving placebo
who declined as expected from the natural course of the disease. Additional
analyses indicated that the Raxone/Catena treatment effect on respiratory
function outcomes was larger in patients not taking concomitant
glucocorticoid steroids.

Idebenone has been granted orphan drug designation for DMD in Europe and the
US and Fast Track designation by the US FDA. Use patent protection extends
until 2026 in Europe and 2027 in the US.

About Santhera

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical
company focused on the development and commercialization of innovative
pharmaceutical products for the treatment of orphan mitochondrial and
neuromuscular diseases. Santhera develops Raxone®/Catena®as treatment for
patients with Leber's Hereditary Optic Neuropathy (LHON), Duchenne Muscular
Dystrophy (DMD) and primary progressive Multiple Sclerosis (ppMS) and
omigapil for Congenital Muscular Dystrophies (CMD), all areas of high unmet
medical need for which no therapies are currently available. For further
information, please visit the Company's websitewww.santhera.com.

Raxone
®and Catena
®are trademarks of Santhera Pharmaceuticals.

For further information, contact:

Thomas Meier, PhD, Chief Executive Officer
Phone +41 61 906 89 64
thomas.meier@santhera.com

US investor contact:

US Public Relations contact:

Andrew McDonald, LifeSci Advisors, LLC Deanne Eagle,
Planet Communications
Phone +1 646 597 6979 Phone +1
917 837 5866
andrew@lifesciadvisors.com
deanne@planetcommunications.nyc

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.

# # #

News release Lancet
http://hugin.info/137261/R/1912574/682668.pdf

---------------------------------------

This announcement is distributed by NASDAQ OMX Corporate Solutions on behalf of NASDAQ OMX Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Santhera Pharmaceuticals Holding AG via Globenewswire

HUG#1912574

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