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SNT-MC17
(idebenone) enters Phase IIa study in Duchenne
muscular dystrophy
Liestal,
Switzerland, October 19, 2005 -- Santhera
Pharmaceuticals AG (“Santhera”) of
Switzerland announced today that it has started a
Phase IIa clinical study with its lead product
SNT-MC17 (idebenone) to evaluate its benefits in
the treatment of Duchenne muscular dystrophy
(DMD). DMD is the most common and devastating type
of muscular dystrophy, causing weakness and muscle
wasting in young boys for which there is no cure
or effective treatment. This is a new potential
indication for Santhera’s SNT-MC17 (idebenone),
which is expected to enter Phase III clinical
trials in Europe later this year for Friedreich’s
Ataxia, another life-threatening neuromuscular
disease.
The Phase IIa DMD study is a
double-blind, randomised, placebo-controlled trial
which aims to assess the efficacy of SNT-MC17 in
10 to 16 year old males with cardiac dysfunction
associated with DMD. The primary endpoint is to
evaluate cardiac function improvement in DMD
patients after one year of treatment. The effect
of SNT-MC17 on muscle strength in the limbs and
respiratory muscles will also be assessed as
secondary endpoints. This study will take place at
the University of Leuven in Belgium, and will
enroll a total of 21 patients. The principal
investigator of this study is Prof. Gunnar Buyse,
a distinguished physician in the field of
neuromuscular disease.
Thomas Meier, Ph.D.,
Chief Scientific Officer of Santhera commented:
“Duchenne muscular dystrophy is a devastating
neuromuscular disease, which affects all of the
voluntary muscles, as well as the muscles of the
heart and the respiratory system. In recent years,
progress has been made by using ventilation
support systems to prevent respiratory failure,
leaving heart failure as a major cause of
mortality in this patient group. By protecting the
muscle cells from oxidative stress, a major
pathological factor in the disease, with SNT-MC17,
we believe that we can improve cardiac function.
We will also study whether the general muscle
weakening in DMD patients can be slowed
down.”
Klaus Schollmeier, Ph.D., Chief
Executive Officer of Santhera declared:
“Santhera’s strategy is to investigate and
capitalize on the full potential of our lead
product in as wide a range of indications as
possible. Our strong expertise in the field of
neuromuscular disease together with the experts at
University of Leuven will enable us to evaluate
the potential benefits of our compound in patients
affected with Duchenne muscular dystrophy.”
About Duchenne muscular dystrophy
(DMD)
Duchenne muscular dystrophy
is the most common and devastating type of
muscular dystrophy. It is a genetic, degenerative
disease that is inherited in an X-linked recessive
mode. DMD affects approximately 30,000 to 50,000
patients worldwide and its incidence is
approximately 1 in 3,500 live born males. Women
can be carriers of DMD but usually exhibit no
symptoms. DMD is characterized by a complete loss
of the dystrophin protein, leading to impaired
calcium homeostasis and elevated oxidative stress
in muscle cells. This results in progressive
muscle weakness and wasting and the loss of
ambulation in teenage patients. Dilated
cardiomyopathy is commonly associated with this
disease leading to early morbidity and mortality
in DMD patients, usually in their twenties.
About Santhera
Santhera Pharmaceuticals AG is a Swiss
biopharmaceutical company focused on the
discovery, development and marketing of small
molecule pharmaceutical products for the treatment
of neuromuscular diseases. The Company’s lead
product, SNT-MC17 (idebenone) is about to enter
Phase III for the treatment of Friedreich’s
Ataxia, a rare but devastating disease which is
ultimately fatal. Santhera has orphan drug
designation for this indication in both the US and
EU. The Company intends to market the product in
the US, and has exclusively licensed to Takeda
rights to market the product in Europe. Santhera
has developed a pipeline of preclinical drug
candidates which it will progress in neuromuscular
diseases and out license in areas outside its core
therapeutic focus. Santhera’s program on novel DPP
IV inhibitors for the treatment of metabolic
diseases, including Type II diabetes, is licensed
to Biovitrum (Sweden).
Santhera was formed
in 2004 through the merger of MyoContract
AG and Graffinity Pharmaceuticals
AG providing it with a fully integrated
platform for the discovery and development of drug
candidates. The Company has operations in Basel,
Switzerland and Heidelberg, Germany. Santhera has
attracted investment from leading global industry
investors including Merlin Biosciences Limited,
Oxford Bioscience Partners, NGN Capital, 3i Group
plc, Carnegie Asset Management, The Novartis
Venture Fund, Varuma AG, GIMV, Heidelberg
Innovation, Clariden Bank, The Dow Chemical
Company, TechnoStart, tbg, Altana
Innovationsfonds, the Swiss Foundation for
Research on Muscle Diseases, and private
investors. Since its formation in 2004 Santhera
has raised €28 million.
For further
information, please visit www.santhera.com.
For further information, contact:
Santhera Pharmaceuticals
Klaus Schollmeier, CEO
Tel: +41 (0) 61 906
89 52
Thomas Meier, Chief Scientific Officer
Tel: +41 (0) 61 906 89 87
thomas.meier@santhera.com
Media contact:
Citigate
Chris Gardner
Tel : +44
(0) 207 638 9571
David Dible
Tel : +44 (0)
207 638 9571
david.dible@citigatedr.co.uk
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