Santhera Prepares for Pivotal Study with Omigapil in Congenital Muscular Dystrophy
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Liestal, Switzerland, September 29, 2010 - Santhera Pharmaceuticals (SIX: SANN)
informed today about recent achievements and next steps in the clinical
development program for omigapil as potential first treatment of Congenital
Muscular Dystrophy (CMD). This severe, genetically determined neuromuscular
disease frequently affects infants or young children with life-threatening
progressive muscle weakness. The preparation of the further clinical development
includes an expert meeting held this week in Bethesda, Maryland, hosted by the
US National Institute of Neurological Disorders and Stroke (NINDS) and Cure CMD,
a dedicated patient advocacy group.
Santhera is currently completing the remaining nonclinical development of
omigapil required for the use of the compound in pediatric patients with
financial support from the Association Française contre les Myopathies (AFM).
Santhera plans to seek protocol assistance and scientific advice from the US
Food and Drug Administration and the European Medicines Agency by early 2011.
Both agencies have already granted orphan drug designation to the program.
"We would like to thank AFM for their financial support of the nonclinical
development for omigapil and Cure CMD and NINDS for organizing this expert
meeting in Bethesda. This will be the first pivotal development program ever in
this devastating disease. The support of patient advocacy organizations and
clinical experts in advancing translational research into the clinic is
tremendously important", said Thomas Meier, Chief Scientific Officer of
Santhera. "We anticipate the potential initiation of a pivotal study in late
2011 or early 2012."
About Congenital Muscular Dystrophy (CMD)
CMD refers to a wide variety of inherited neuromuscular conditions characterized
by different forms of progressive loss of muscle tissue. Severe forms can affect
newborns or young children with life-threatening progressive muscle weakness
("floppy infant syndrome"). Complications associated with the disorder cause
immobility at young age and early mortality. Patients suffer from loss of body
weight, skeletal deformations and respiratory distress resulting in immobility
at young age and early mortality. A recent epidemiological estimate approximates
a prevalence of 0.89 per 100,000. No pharmacological therapy is currently
available or in advanced clinical development. Treatment options are confined to
ventilatory support and orthopedic surgery for scoliosis as well as
supplementary nutrition to avoid malnutrition.
Santhera focuses on distinct subtypes of CMD caused by collagen-VI (Ullrich,
Bethlem Myopathy) or laminin-alpha-2 (MDC1A) deficiency. Both subtypes are
associated with mitochondrial dysfunction and muscle cell apoptosis. In vivo
studies show that omigapil, an anti-apoptic compound, inhibits cell death and
reduces body weight loss and skeletal deformation while increasing locomotive
activity and protecting from early mortality [1].
Reference
[1] Erb M. et.al. (2009). Omigapil ameliorates the pathology of muscle dystrophy
caused by laminin-alpha 2 deficiency. Journal of Pharmacology and Experimental
Therapeutics 331: 787-795
* * *
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 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. For further information, please visit 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(at)santhera.com
Barbara Heller, Chief Financial Officer
Phone: +41 (0)61 906 89 54
barbara.heller(at)santhera.com
Thomas Staffelbach, Head Public & Investor Relations
Phone: +41 (0)61 906 89 47
thomas.staffelbach(at)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.
[HUG#1447446]
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Santhera Pharmaceuticals Holding AG
Hammerstrasse 49 Liestal Switzerland
ISIN: CH0027148649;
News Release Omigapil:
http://hugin.info/137261/R/1447446/390063.pdf
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Datum: 29.09.2010 - 07:00 Uhr
Sprache: Deutsch
News-ID 43173
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Town:
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Kategorie:
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