Santhera Prepares for Pivotal Study with Omigapil in CMD

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 con-tre 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 agen-cies 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 piv-otal development program ever in this devastating disease. The support of patient advocacy organi-zations and clinical experts in advancing translational research into the clinic is tremendously impor-tant”, 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 supple-mentary nutrition to avoid malnutrition. Santhera Prepares for Pivotal Study with Omigapil in Congenital Muscular Dystrophy September 29, 2010 / Page 2 of 3

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 dysfunc-tion and muscle cell apoptosis. In vivo studies show that omigapil, an anti-apoptic compound, inhib-its cell death and reduces body weight loss and skeletal deformation while increasing locomotive activity and protecting from early mortality [1].


[1] Erb M. (2009). Omigapil ameliorates the pathology of muscle dystrophy caused by laminin-alpha 2 deficiency. Journal of Pharmacology and Experimental Therapeutics 331: 787-795

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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 Catena

® is a trademark of Santhera Pharmaceuticals. For further information, contact

Klaus Schollmeier, Chief Executive Officer

Phone: +41 (0)61 906 89 52

Barbara Heller, Chief Financial Officer

Phone: +41 (0)61 906 89 54

Thomas Staffelbach, Head Public & Investor Relations

Phone: +41 (0)61 906 89 47

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  • Congenital Muscular Dystrophy

    A group of diseases causing muscle weakness at birth. Several defined genetic mutations cause muscles to break down faster than they can repair or grow. A child with CMD may have various neurological or physical impairments. Some children never gain the ability to walk, while others lose the ability as they grow older. Learn more...

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