Santhera Enters Agreement with Columbia University to Investigate
Additional Potential of Catena® in MELAS
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Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty
pharmaceutical company focused on orphan neuromuscular diseases,
announced today a collaboration with Columbia University in New York
to investigate Catena® in a Phase II study as treatment of MELAS
(Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like
episodes). The MELAS syndrome is a rare but devastating multisystem
disorder that affects the brain, nervous system, muscles and
cognitive abilities. Currently, there are no approved therapies
available or in development to help people with MELAS. Today's
announcement follows shortly after a similar collaboration between
Santhera and the US National Institutes of Health to investigate
Catena® in Primary Progressive Multiple Sclerosis.
The MELTIMI (MELAS Trial of Idebenone using MRS) trial is a Phase II
double-blind, placebo-controlled study investigating the safety and
efficacy of two doses of Catena® (INN: idebenone) versus placebo. The
primary endpoint of the proof-of-concept study is the change in
cerebral lactate concentration from baseline to week four as assessed
by Magnetic Resonance Spectroscopy (MRS). The MELTIMI study will also
investigate additional endpoints such as venous lactate levels,
fatigue and quality of life. The lactate level is considered a viable
biomarker associated with the neurological impairment in MELAS.
Overall, the MELTIMI study will enroll 21 patients aged between 8 and
65 years. Further details of the study protocol including contact
details of the study center are registered under
www.clinicaltrials.gov (identifier: NCT00887562).
The MELTIMI study is being performed at Columbia University. Under
the agreement, the Neurological Institute of Columbia University is
conducting the clinical trial while Santhera is supplying the study
medication and contributes data management and other support. The
Company has exclusive rights to the data for regulatory purposes.
Thomas Meier, Chief Scientific Officer of Santhera, commented: "MELAS
is a devastating multi-system syndrome for which no effective
treatment is available or in clinical development. There is a strong
scientific rationale that suggests that Catena® may protect patients
with MELAS from neuronal damage, based on the drug's ability to
enhance mitochondrial function. We are very pleased to announce this
collaboration with Columbia University, a leading academic research
university. Our goal is to validate cerebral lactate level as
biomarker and to conduct a proof of concept study of Catena® in
MELAS."
About the Mitochondrial Encephalopathy, Lactic Acidosis, and
Stroke-like Episodes
The Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like
Episodes (MELAS) syndrome is a progressive and devastating condition
that affects many of the body's systems, particularly the brain and
nervous system (encephalo-) and muscles (myopathy). The most
characteristic symptoms of MELAS are recurring, stroke-like episodes
in which severe, migraine-like headaches are followed by vomiting and
seizures. Repeated stroke-like episodes can progressively damage the
brain, leading to vision loss, problems with movement, and dementia.
Other symptoms include short stature, diabetes, exercise intolerance
and hearing loss. Most people suffering from MELAS have an
accumulation of lactic acid (lactic acidosis) in the blood which can
lead to vomiting, abdominal pain, extreme tiredness, muscle weakness,
and difficulty breathing. Elevated levels of lactic acid in the brain
are considered to be part of the underlying causes that lead to
stroke-like episodes and brain tissue damage. The maternally
inherited multisystem disorder runs in families and manifests in both
sexes. First signs and symptoms usually appear already in childhood.
MELAS is associated with a shortened life-expectancy.
The MELAS syndrome is caused by a mutation in the mitochondrial
genome leading to impaired function of the mitochondrial respiratory
chain. Catena® has been shown to enhance the electron flow in the
electron transport chain and increase cellular energy production. The
drug is also active in scavenging reactive oxygen species thereby
protecting the cell from oxidative stress. Catena® is crossing the
brain-blood barrier and is readily taken up by cells.
Current treatment options are limited to symptomatic management of
seizures, diabetes and other medical complications. There are no
approved therapies available or in clinical development to help
people with MELAS. In the absence of reliable epidemiological
studies, the prevalence is assumed to be several thousands patients
each in Europe and in the United States. Existing prevalence numbers
indicate regional hot spots and a high degree of under-diagnosis.
Given the orphan nature of the disease and the associated high unmet
medical needs, the program with Catena® qualifies for application of
orphan drug designations.
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About Santhera
Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty
pharmaceutical company focused on the discovery, development and
commercialization of small-molecule pharmaceutical products for the
treatment of severe neuromuscular diseases, an area of high unmet
medical need which includes many orphan indications with no current
therapy. Santhera's first product, Catena® to treat Friedreich's
Ataxia, is marketed in Canada. Data of the second pivotal Phase III
trial in Europe are expected for the first half of 2010. The drug has
also shown efficacy in a clinical trial as a potential treatment for
Duchenne Muscular Dystrophy. For further information, please visit
the Company's Web site www.santhera.com.
Catena® is a trademark of Santhera Pharmaceuticals.
For further information, contact
Klaus Schollmeier, Chief Executive Officer
Phone: +41 (0)61 906 89 52
klaus.schollmeier@santhera.com
Thomas Meier, Chief Scientific Officer
Phone: +41 (0)61 906 89 87
thomas.meier@santhera.com
Barbara Heller, Chief Financial Officer
Phone: +41 (0)61 906 89 54
barbara.heller@santhera.com
Thomas Staffelbach, Head Public & Investor Relations
Phone: +41 (0)61 906 89 47
thomas.staffelbach@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.
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Santhera Pharmaceuticals Holding AG
Hammerstrasse 47 Liestal
Switzerland
WKN: A0LCUK; ISIN: CH0027148649; Index: SPI, SPIEX, SSCI;
Listed:
Main Market in SIX Swiss Exchange;