Santhera Pharmaceuticals Holding AG: Santhera Presents New Efficacy Data from an Expanded Access Program with Raxone® for the

Santhera Pharmaceuticals Holding AG: Santhera Presents New Efficacy Data from
     an Expanded Access Program with Raxone® for the Treatment of Leber's
                      Hereditary Optic Neuropathy (LHON)

Santhera Pharmaceuticals Holding AG / Santhera Presents New Efficacy Data from
an Expanded Access Program with Raxone® for the Treatment of Leber's
Hereditary Optic Neuropathy (LHON) . Processed and transmitted by NASDAQ OMX
Corporate Solutions. The issuer is solely responsible for the content of this

Liestal, Switzerland, March4, 2014 - Santhera Pharmaceuticals (SIX:SANN)
announced today that it will present data from its ongoing Expanded Access
Program (EAP) with Raxone^® in the treatment of LHON at the 2014 Annual
Meeting of the North American Neuro-Ophthalmology Society (NANOS) in Rio
Grande, Puerto Rico. Current data from the EAP demonstrate that 50% of
patients have achieved a clinically relevant improvement in their vision and
63% of the patients were protected from further vision loss following Raxone^®
treatment. Raxone^® was safe and well tolerated following long-term use in the
EAP, which provides further evidence of safety and efficacy for Raxone^® in
the treatment of LHON in the routine clinical setting.

Safety and efficacy data were collected in an ongoing EAP for LHON patients
with recent onset of symptoms in which they were given access to Raxone^® in
response to an unsolicited request from their treating physician. As of
January31, 2014, 29 physicians in Europe, the USA, Australia and New Zealand
had enrolled 61 LHON patients. Participants received Raxone^® typically at
900mg/day and were assessed for safety and visual acuity (VA) during routine
clinical care for up to 21 months. Currently data from 48 patients carrying
the G11778A, G3460A or T14484C mutation have provided best corrected VA data
from at least one on-treatment visit. Patient demographics such as
distribution of mtDNA mutation carried, gender, age and extent of VA loss of
enrolled patients were consistent with the known natural history of LHON.
Efficacy analyses assessed (i) the proportion of patients with clinically
relevant and stable improvement in VA and (ii) the proportion of patients
without further deterioration in VA.

LHON patients treated with Raxone^® experienced rapid, clinically relevant
improvement in vision
In the EAP, 24 of 48 patients (50%) have so far experienced a clinically
relevant and stable improvement in VA defined as either (i) improvement from
VA nadir of at least 10 letters (logMAR 0.2) on the ETDRS chart or (ii)
improvement to reading at least 5 letters on-chart in patients with severe
vision loss unable to read any letters on the ETDRS chart at nadir. In both
cases, VA recovery had to be stable until the last available visit. Patients
have been treated for an average of 11 months (range 3 to 21 months). Analyzed
by disease-causing mtDNA mutation carried, 89% of patients with the T14484C
mutation, 70% with the G3460A and 31% with the G11778A mutation have had
clinically relevant recovery of vision. The percentage of Raxone^® treated
patients with VA recovery is markedly higher than that observed in a
comparable population from a recently completed natural history case record
survey of over 350 LHON cases collected from centers in Europe and the USA.

In the EAP, the average treatment effect size in patients with VA recovery was
29 letters from the VA nadir across all mtDNA mutations (49 letters for
patients with the T14484C, 26 letters for patients with the G3460A and 13
letters for patients with the G11778A mtDNA mutation). The efficacy of
Raxone^® in promoting recovery of VA occurs rapidly, as 75% of patients have
responded within the first 6 months and 83 % within 12 months of the
initiation of Raxone^® treatment.

Raxone^® treatment protects LHON patients from further vision loss
In the EAP, there were 38 patients able to read at least 5 letters on the
ETDRS chart at the time of enrolment (baseline). Of those 24 (63%) had not
worsened by the clinically relevant margin (defined as 10 letters of on-chart
VA or transition from on-chart VA to off-chart vision) in either eye at the
last available visit. Moreover, 9 of 13 patients (69%) who were not legally
blind at baseline were protected from legal blindness (defined as logMAR
>=1.0) during their Raxone^® treatment period.

Data will be presented at this weeks' 40th Annual Meeting of the North
American Neuro-Ophthalmology Society (NANOS) in Puerto Rico.

"The clinical experience with Raxone^® from the ongoing Expanded Access
Program clearly supports previously published evidence of the safety and
efficacy of Raxone^® in the treatment of LHON", commented Thomas Meier, CEO of
Santhera. "With this program, we have increased greatly the long-term data
available for Raxone^® in this disease. These new efficacy and natural history
data will be important supporting elements in the European Marketing
Authorization Application which we plan to submit in the coming weeks."

About Leber's Hereditary Optic Neuropathy and the use of Raxone^®
Leber's Hereditary Optic Neuropathy (LHON) is a heritable genetic disease
causing blindness. The disease typically presents in young adult men as rapid,
painless loss of central vision in one eye, followed by the fellow eye within
a few weeks or months of the onset of symptoms, usually leading to complete
and permanent blindness. Over 95% of patients harbour one of three pathogenic
mutations of their mitochondrial DNA which causes a defect in the complexI
subunit of the mitochondrial respiratory chain. This defect leads to decreased
cellular energy (ATP) production, increased oxidative stress and retinal
ganglion cell dysfunction which cause progressive loss of visual acuity and

Raxone^® (idebenone), a synthetic short-chain benzoquinone and a cofactor for
the enzyme NAD(P)H:quinone oxidoreductase (NQO1), is capable of transferring
electrons directly onto complex III of the mitochondrial electron transport
chain, thereby circumventing the complexI defect and restoring cellular
energy levels in retinal ganglion cells and promoting recovery of visual

About the Expanded Access Program

The EAP was established to provide access to Raxone^® treatment to individual
"named" LHON patients at the request and under the personal care of a
registered physician according to applicable regulations. All patients receive
Raxone^® (usually 900 mg/day) and there is no control group. Visual acuity and
safety information (adverse events) is collected at the patient's regular
(generally 3-monthly) clinic visits and reported to Santhera.

Together with the contributing physicians, Santhera intends to submit the
results of the EAP to a peer reviewed journal for publication.

The North American Neuro-Ophthalmology Society (NANOS) is a professional
organization of more than 500 ophthalmologists or neurologists. The Society
seeks to promote the field of neuro-ophthalmology by supporting all forms of
education, encouraging research and fostering clinical expertise. The 40th
Annual NANOS Meeting is held March 1-6, 2014 in Rio Grande, Puerto Rico.

                                    * * *

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, such as Leber's Hereditary Optic Neuropathy, Duchenne
Muscular Dystrophy and primary progressive Multiple Sclerosis, all of them
areas of high unmet medical need with no current therapies. For further
information, please visit

Raxone^® 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 NANOS


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Source: Santhera Pharmaceuticals Holding AG via Globenewswire

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