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2014-05-22

Santhera Pharmaceuticals Holding AG: Santhera Reports Positive Outcome for Catena®/Raxone® in Phase III DMD Trial Supported by Additional Respiratory Functio

Santhera Pharmaceuticals Holding AG / Santhera Reports Positive Outcome for
Catena®/Raxone® in Phase III DMD TrialSupported by Additional Respiratory
Function Data . Processed and transmitted by NASDAQ OMX Corporate
Solutions.The issuer is solely responsible for the content of this
announcement.
Liestal, Switzerland, May 22, 2014
- Santhera Pharmaceuticals (SIX: SANN) today reported that the
results of
secondary
respiratory function endpoints from the on-going analysis of the DELOS trial
in Duchenne Muscular Dystrophy (DMD) corroborate the positive outcome for the
primary endpoint. These data provide further supportive evidence of a
treatment benefit for Catena®/Raxone®
in DMD.

As previously announced, the DELOS trial met the primary endpoint, the
difference between Catena®/Raxone®and placebo in the change from baseline to
week 52 in Peak Expiratory Flow (PEF as percent predicted, PEF%p).
Hospital-based spirometry assessments demonstrated that
Catena®/Raxone®significantly reduced the annual decline in PEF%p by 66%
compared to patients taking placebo. The average annual decline in PEF%p was
9.0% for placebo (Baseline: 54.3%; Week 52: 45.3% (n=27), p<0.001) versus
3.1% for Catena®/Raxone®(Baseline PEF%P: 53.1%; Week 52: 50.1% (n=30);
p=0.13) for a treatment group difference in change from Baseline to Week 52
of 5.96% (p=0.04).

Santhera today announced that this finding has been corroborated by the
results of secondary endpoints assessing respiratory function in all
randomized and treated subjects. When measured weekly by the patient at home
using the hand-held ASMA-1 device (secondary endpoint),
Catena®/Raxone®significantly reduced the annual decline in PEF%p by 80%
compared to patients taking placebo. The ASMA-1 device showed a significant
9.0% decline in PEF%p occurred between Baseline and Week 52 in the placebo
group (n=31; p<0.001), compared to a non-significant decline of 1.8% in the
Catena®/Raxone®group (n=31; p=0.44), for a treatment group difference in
change from Baseline to Week 52 of 7.2% (p=0.03).

Furthermore, for Forced Expiratory Volume in 1 second (as percent predicted,
FEV1%p), an additional endpoint for respiratory muscle strength,
Catena®/Raxone®significantly reduced the annual decline by 78% compared to
patients taking placebo. The annual decline in FEV1%p in the placebo group
was 10.7% compared to 2.4% in the Catena®/Raxone®group (p=0.03).

Importantly, the outcome for Forced Vital Capacity (as percent predicted,
FVC%p), a measure of restrictive lung disease predictive of morbidity and
mortality in DMD, also supported a treatment benefit of Catena®/Raxone®. The
annual decline in FVC%p was reduced by 37% in Catena®/Raxone®-treated
patients (9.0% decline in FVC%p in the placebo group versus a 5.7% decline in
the Catena/Raxone group; p=0.08).

No differences were observed between treatment groups in Maximal Inspiratory
or Expiratory Pressures or in Peak Cough Flow.

In summary these outcomes provide clear evidence of a clinical benefit for
Catena®/Raxone®in delaying the loss of respiratory function in patients with
DMD compared to placebo.

As also reported previously, treatment with Catena®/Raxone®was safe and well
tolerated. A total of 93.8% of Catena®/Raxone®-treated and 94.1% of
placebo-treated patients experienced at least one Adverse Event (AE). Serious
AEs were reported in 6.3% of Catena®/Raxone®treated and in 14.7% of
placebo-treated patients. Nasopharyngitis (25.8%) and headache (19.7%) were
the most common adverse events but there were no differences in the incidence
of these events between the treatment groups. Diarrhoea was slightly more
commonly observed in Catena®/Raxone®-treated patients (25.0% versus 11.8%),
whilst upper respiratory tract infections were more frequently observed in
placebo-treated patients (17.6% versus 6.3%). Overall adverse events were
mild to moderate in intensity.

"We are very excited to observe this treatment benefit for Catena®/Raxone®in
delaying respiratory deterioration in DMD, as evidenced by the significant
outcome for the primary PEF endpoint in DELOS. The positive results of
secondary outcomes, in addition to the primary endpoint, are clearly
supportive and establish the clinical meaningfulness of
Catena®/Raxone®treatment. This is of critical importance for the
demonstration of substantial evidence of effectiveness in the regulatory
process", explained Nick Coppard, SVP Development at Santhera. "Having
demonstrated a significant and clinically relevant benefit without safety
concerns, we believe we are well positioned to discuss regulatory approval
with the authorities".

These data are presented today at the Bio€quity Europe 2014 Conference in
Amsterdam and will be available for download on the Company's
websitewww.santhera.comunderInvestors/Presentations.

About DELOS
DELOS was a Phase III, double-blind, placebo-controlled trial which randomized
and treated 64 European and US patients with DMD who were not receiving
concomitant corticosteroids to either Catena®/Raxone®tablets (900mg: 300mg 3
times daily, N=31) or to matching placebo (N=33). Their average age was 14.3
years and 5 were ambulatory and 59 non-ambulatory at Baseline. There were no
eligibility criteria for mutational status. The study was designed to assess
the efficacy of Catena®/Raxone®in improving or delaying the loss of
respiratory function in patients with DMD not using corticosteroids compared
to placebo. The preservation of respiratory function is acknowledged by
clinicians and by regulatory authorities as of major clinical importance for
patients with DMD.

About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (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 approximately 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 due to
respiratory failure. 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 and supplementing cellular
energy levels.

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 Catena®/Raxone®as treatment for
patients with Leber's Hereditary Optic Neuropathy (LHON), Duchenne Muscular
Dystrophy (DMD) and primary progressive Multiple Sclerosis (ppMS), all areas
of high unmet medical need with no current therapies. Santhera previously
received temporary approval (cATU) for Raxone®in the treatment of LHON in
France and has recently submitted a Marketing Authorization Application (MAA)
to the European Medicines Agency for the treatment of LHON in the European
Union. Santhera recently estimated that the combined annual peak sales
potential for Catena®/Raxone®in the LHON and DMD indications could reach CHF
600 million.

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

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 two
http://hugin.info/137261/R/1787840/614031.pdf

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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

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