Santhera Pharmaceuticals Holding AG : Independent DSMB Recommends Continuation of Santhera's Phase III Trial of Catena® in

Santhera Pharmaceuticals Holding AG : Independent DSMB Recommends Continuation
   of Santhera's Phase III Trial of Catena® in Duchenne Muscular Dystrophy

Santhera Pharmaceuticals Holding AG / Independent DSMB Recommends Continuation
of Santhera's Phase III Trial of Catena® in Duchenne Muscular Dystrophy .
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responsible for the content of this announcement.

Liestal, Switzerland, April16, 2013 - Santhera Pharmaceuticals (SIX: SANN)
announced today that the PhaseIII DELOS study of orally administered Catena^®
in patients with Duchenne Muscular Dystrophy (DMD) successfully passed a
planned futility and safety analysis. The independent Data Safety Monitoring
Board (DSMB) for DELOS informed Santhera that the study has a reasonable
chance of achieving its primary endpoint for improving or delaying the loss of
respiratory function in Duchenne patients not using corticosteroids and since
no safety issues were detected, recommended that the study should continue as

"I am very enthusiastic about this news, which is important for the DMD
community as it indicates that Catena^® eventually may evolve into a real
treatment for all patients with this devastating disease. Importantly it
suggests that the DELOS data are consistent with the outcome of the PhaseII
DELPHI study, which showed that treatment with Catena^® resulted in a
significant increase in peak expiratory flow and may improve early signs of
respiratory weakness and dysfunction" commented Gunnar Buyse M.D., Ph.D.,
Professor of Child Neurology at the University Hospitals Leuven (Belgium) and
Principal Investigator for the DELOS study. "Some promising therapies for DMD
have failed to demonstrate clinical benefit in late-stage development, so it
is encouraging to see that we now may be on track to clear this hurdle for
this patient group".

"This futility analysis is particularly relevant since it included primary
efficacy variable data after at least 6 months of treatment from 80%, and
final outcome data after 12 months of treatment from over 60% of the planned
total number of patients in the corticosteroid non-using cohort. This
increases the probability of a positive outcome in the final analysis of this
cohort", said Dr. Nick Coppard, Santhera's Senior VP Development. "Since DELOS
is not restricted to patients with a specific mutational or ambulatory status,
the potential benefit in respiratory function may extend to all Duchenne

Santhera has previously announced that it explores strategic and financing
options including product licensing and the possibility of a merger or
acquisition. The Company's ability to complete the DELOS program is contingent
upon the availability of the financial resources required. To this end,
Santhera has started the process to seek a development or licensing partner
for Catena^® in DMD outside Europe, where commercialization rights have been
licensed to Takeda.

The European Medicines Agency (EMA) and US Food and Drug Administration (FDA)
have both granted orphan drug status to Santhera's program with Catena^® in
DMD in the European Union and the United States of America, respectively.

About the DELOS study
DELOS (DuchEnne Muscular Dystrophy Long-term IdebenOne Study) is a PhaseIII
randomized, double-blind study in patients aged 10-18 years with DMD of any
mutational etiolology, ambulatory or non-ambulatory, recruited in Europe and
in North America. The study is designed to assess the efficacy of orally
administered Catena^® (900mg/day), compared to placebo, in improving or
delaying the loss of respiratory function in Duchenne patients. DELOS uses the
change from baseline to 12 months in per cent predicted Peak Expiratory Flow
(PEF) as the primary endpoint. The study design was agreed with both the EMA
and the FDA. The study plans to sequentially enroll two cohorts of patients,
60 corticosteroid non-using and 200 corticosteroid using patients, to allow
these sub-groups to be independently powered for efficacy and analyzed
separately in the final primary endpoint analysis. The study is powered to
detect a 10.3% difference in the change from baseline to 12 months in per cent
predicted PEF between the Catena^® and placebo groups. As was agreed with both
the US and European regulatory agencies, DELOS is intended to provide pivotal
efficacy data for regulatory filings for Catena^® in the treatment of patients
with DMD in these territories.

About Duchenne Muscular Dystrophy
DMD is the most common and a devastating type of muscular degeneration and
results in rapidly progressive muscle weakness. It is a genetic, degenerative
disease that is inherited in an X-linked mode. DMD is characterized by a loss
of the protein dystrophin, leading to progressive muscle weakness and wasting
through a complex cascade that involves impaired calcium homeostasis,
mitochondrial dysfunction and oxidative stress. The average age of onset is
between 3 and 5 years of age with a loss of ambulation in teenage patients.
Dilated cardiomyopathy and respiratory failure are commonly associated with
this chronic disease leading to early morbidity and mortality in Duchenne

                                    * * *

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 DELOS


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