Santhera Presents Preclinical Data on Oral MC4 Receptor Antagonists for Treatment of Cancer Cachexia at AICR Research Conference

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Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty
pharmaceutical company focused on neuromuscular diseases, today
announced preclinical data on a novel generation of melanocortin-4
(MC4) receptor antagonists for potential treatment of cancer cachexia
(severe muscle wasting). The most advanced candidates significantly
increase food intake in healthy animals and prevent cancer-induced
body weight loss in disease-relevant models. In-vivo data will be
presented today at the 2008 Research Conference on Food, Nutrition,
Physical Activity & Cancer of the American Institute for Cancer
Research (AICR) in Washington DC [1].

Santhera's two novel, yet chemically unrelated MC4 receptor
antagonists were both found to significantly increase appetite in
healthy mice. In studies of mice carrying a C26 adenocarcinoma
repeated oral administration significantly inhibited the development
of cachexia, i.e. reduced tumor-induced weight loss, and showed
beneficial effects on body weight, lean mass and fat mass. Both
compounds are orally available, were shown to be highly selective and
have crossed the blood/brain barrier.

Thomas Meier, Chief Scientific Officer of Santhera, said: "Cancer
cachexia represents a life-threatening metabolic condition
contributing to the morbidity and mortality of cancer patients.
Current treatment options for cachexia are very limited and,
consequently, this syndrome is an area of high unmet medical need.
Our data confirm that MC4 receptor antagonists offer a new and
promising treatment strategy for this severe muscle wasting.
Santhera's advanced candidates significantly increase food intake and
prevent body weight loss in disease-relevant models."

Klaus Schollmeier, Chief Executive Officer of Santhera, said:
"Santhera's research team has discovered and developed two series of
novel MC4 receptor antagonists with promising preclinical results in
cancer cachexia. Preclinical work is expected to be completed by the
end of 2009 with an anticipated entry into the clinic in 2010. We are
now seeking to build a strategic partnership for further development
and commercialization of the program. The preferred partner would
bring a complimentary expertise in oncology or cancer support."

Cancer cachexia affects about one million patients in the North
America and Europe. Up to 80% of cancer patients suffer from the
syndrome which accounts for approximately 20% of cancer deaths.
Despite this high unmet medical need, no effective treatments are
available. Currently used pharmacological interventions have limited
utility or produce severe side-effects. The potential market size of
the cancer cachexia market is estimated to be above USD 1 billion.
About cachexia syndrome and MC4 receptor antagonists
Cachexia (Greek for "poor condition") syndrome is one of the most
debilitating and life-threatening aspects of cancer and other severe
chronic illnesses. The syndrome is associated with anorexia (lack of
appetite), fat and muscle tissue wasting, psychological distress and
a progressively decreasing quality of life. Cachexia is characterized
by major metabolic abnormalities and maladaptations such as reduced
food and energy intake, increased resting energy expenditure and
accelerated catabolism. Typically, the involuntary weight loss in
excess of 5% in 12 months cannot be compensated for by increased food
intake, which results in wasting of both adipose and skeletal muscle
tissue and poor physical performance.

The exact nature of the underlying processes remains largely unknown,
however, appetite regulating peptides, pro-inflammatory cytokines and
other regulatory peptides are considered to be major factors. One
pathway by which cytokines can induce anorexia is via an increase of
proopiomelanocortin (POMC) expression in hypothalamic feeding
circuits, leading to an enhanced production of alpha-melanocyte
stimulating hormone (alpha-MSH), a strong inhibitor of appetite. The
MC4 receptor in the hypothalamus is a crucial target in the pathway
through which alpha-MSH exerts its appetite inhibiting effects. AgRP
(agouti related protein) or small molecule MC4 receptor antagonists
enhance food intake, reduce energy expenditure and catabolic
activity. Accordingly, an interruption of this signaling pathway by
means of MC4 receptor antagonists is seen as a promising treatment
option for cachexia.

Reference
[1] Philipp Weyermann, Corinne Anklin, Isabelle Courdier-Fruh, Robert
Dallmann, Judith Dubach-Powell, Martina Hufschmid, Josef P. Magyar,
Bernd Löffler, Bettina Cardel, Florian Schärer, Gesa Santos, Reto
Bolliger, Marco Henneböhle, Holger Herzner, Stéphane Kervennic,
Audrey LeGall, Sonja Nordhoff, Hervé Siendt, Michael Soeberdt,
Miroslav Terinek, Achim Feurer, Cesare Mondadori & Thomas Meier: New
types of selective, orally active melanocortin-4 receptor antagonists
stimulate food intake and reduce cancer-induced cachexia in mice.
Poster presented at the AICR 2008 Research Conference on Food,
Nutrition, Physical Activity & Cancer in Washington DC from November
6 to 7, 2008.

* * *

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 currently investigates three compounds in five
clinical-stage development programs. The Company's first product,
SNT-MC17 (INN: idebenone), has received a marketing approval with
conditions from Health Canada to treat Friedreich's Ataxia and is
marketed under its brand name CATENA®. The product is also under
review by health authorities in the EU and in Switzerland, while in
the United States, a pivotal Phase III trial is recruiting patients.
The compound has also shown efficacy in a clinical trial as a
potential treatment for Duchenne Muscular Dystrophy and is currently
in further clinical development. For additional information, please
visit the Company's website www.santhera.com.
CATENA® is a trademark of Santhera Pharmaceuticals, registered in
Canada and the United States.

For further information, contact
Klaus Schollmeier, Chief Executive Officer
Phone: +41 (0)61 906 89 52
klaus.schollmeier@santhera.com

Barbara Heller, Chief Financial Officer
Phone: +41 (0)61 906 89 54
barbara.heller@santhera.com

Thomas Meier, Chief Scientific Officer
Phone: +41 (0)61 906 89 87
thomas.meier@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;
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