Santhera Pharmaceuticals Holding AG : Santhera Secures Exclusive Rights from NIH for a Novel Approach for the Treatment of

 Santhera Pharmaceuticals Holding AG : Santhera Secures Exclusive Rights from
     NIH for a Novel Approach for the Treatment of Primary Progressive MS

Santhera Pharmaceuticals Holding AG / Santhera Secures Exclusive Rights from
NIH for a Novel Approach for the Treatment of Primary Progressive MS .
Processed and transmitted by Thomson Reuters ONE. The issuer is solely
responsible for the content of this announcement.

Liestal, Switzerland, June6, 2013 -Santhera Pharmaceuticals (SIX:SANN)
announced today that it has obtained an exclusive license from the National
Institutes of Health (NIH) to its rights on a patent granted in the USA for
the use of idebenone for the treatment of primary progressive Multiple
Sclerosis (ppMS), a currently untreatable disease affecting about 40,000
patients in the Unites States.The NIH is investigating the efficacy of
Catena^® (idebenone) in ppMS in a placebo-controlled PhaseII clinical trial.

Lead by the Neuroimmunology Branch of the National Institute of Neurological
Disorders and Stroke (NINDS), the NIH is investigating the efficacy of
Catena^® (idebenone) in patients with ppMS in a double-blind,
placebo-controlled PhaseII clinical trial (IPPoMS trial). Santhera is
providing study medication under a clinical trial agreement which gives
Santhera the rights to the results. Santhera has now obtained the exclusive
rights to the use patent for idebenone in ppMS granted in the USA. Patients
who complete the IPPoMS trial can enter into a 12-months open-label extension
trial for which Santhera and the NIH recently signed a Materials Cooperative
Research and Development Agreement (M-CRADA).

"Patients with ppMS do not respond to immunomodulatory therapies with proven
efficacy in relapsing remitting MS," said Bibiana Bielekova, M.D, the
principal investigator of the IPPoMS trial and Chief of the Neuroimmunology
Disease Unit at the NINDS. "Accumulating data indicate that mitochondrial
dysfunction and related oxidative stress may play a major role in the
pathogenesis of progressive MS. Idebenone enhances mitochondrial function and
acts as an anti-oxidant against membrane damage in laboratory models and is a
rational treatment choice in ppMS based on these pharmacological properties.
Recruitment is proceeding well in the IPPoMS trial and we are excited about
the opportunity to study the potential of idebenone in this currently
untreatable disease."

"We are very enthusiastic about this collaboration and about the progress
currently being made. ppMS is a disease with high medical need. In contrast to
other forms of MS, there is currently no treatment option available for the
10-15 % of MS patients affected by this primary progressive subtype,"
commented Nick Coppard, Santhera's Head of Development.

About primary progressive MS
Multiple Sclerosis (MS) is a chronic neurological disease of the central
nervous system affecting around 2.5 million people worldwide. Notable
differences in MRI findings can distinguish clinical subtypes of MS. The
relapsing-remitting form of MS (rrMS) is the most common form of the disease.
Patients affected by rrMS experience attacks of clinical worsening followed by
periods of clinical stability with partial or even complete recovery. So far,
effective treatments for rrMS are limited to immune-modulatory therapies which
have not been proven to be effective in ppMS patients. This subtype develops
much slower than rrMS but presents a steady functional decline without any
distinct episodes of regeneration or acute relapses. According to the MS
Association of America (MSAA) there are about 40,000 patients with ppMS living
in the United States.

About idebenone and rationale for use in ppMS
Mitochondrial dysfunction together with aberrant formation of reactive oxygen
species are believed to underlie at least partially the development of
progressive CNS tissue destruction in ppMS. Idebenone, a synthetic short-chain
benzoquinone and a cofactor for the enzyme NAD(P)H:quinoneoxidoreductase
(NQO1) is capable of transferring electrons directly onto complex III of the
mitochondrial electron transport chain, thereby restoring cellular energy
levels. In addition, idebenone is a powerful anti-oxidant. In view of the
hypothesized involvement of mitochondrial dysfunction, and particularly the
reduced activity of the mitochondrial electron transport chain in patients
affected by ppMS, idebenone is a rational treatment choice for this disease
based on its pharmacological properties including its ability to cross the
blood-brain barrier.

About the IPPoMS trial
Adult ppMS patients up to 65 years of age with disability ranging from none to
moderately severe are eligible to enroll in the trial. This is a PhaseI/II
safety/efficacy trial with an adaptive trial design: one year of pretreatment
baseline period serves the dual purpose of collecting patient-specific
biomarkers of disease progression and collecting longitudinal neuroimaging and
clinical data for selection of primary outcome measures. This baseline period
is then followed by a double-blind, idebenone (2250mg/day) versus placebo
treatment phase for a total of 2 years. Additional information can be obtained
under (NCT00950248).

                                    * * *

About the NINDS
The National Institute of Neurological Disorders and Stroke
( is the nation's leading funder of research on the brain
and nervous system. The NINDS mission is to reduce the burden of neurological
disease - a burden borne by every age group, by every segment of society, by
people all over the world.

The National Institutes of Health, the nation's medical research agency,
includes 27 Institutes and Centers and is a component of the U.S. Department
of Health and Human Services. NIH is the primary federal agency conducting and
supporting basic, clinical, and translational medical research, and is
investigating the causes, treatments, and cures for both common and rare
diseases. For more information about NIH and its programs, visit

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 neuromuscular and
mitochondrial diseases, areas of high unmet medical need with no current
therapies. For further information, please visit

Catena^® is a trademark of Santhera Pharmaceuticals.

For further information, contact
Thomas Meier, Chief Executive Officer
Phone: +41 61 906 89 64

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 ppMS


This announcement is distributed by Thomson Reuters on behalf of Thomson
Reuters clients.

The owner of this announcement warrants that:
(i) the releases contained herein are protected by copyright and other
applicable laws; and
(ii) they are solely responsible for the content, accuracy and originality of
information contained therein.

Source: Santhera Pharmaceuticals Holding AG via Thomson Reuters ONE

--- End of Message ---

Santhera Pharmaceuticals Holding AG
Hammerstrasse 49 Liestal Switzerland

ISIN: CH0027148649;
Press spacebar to pause and continue. Press esc to stop.