Santhera Obtains European Patent for Use of Omigapil for the Treatment of Congenital Muscular Dystrophy

The patent granted covers the treatment or prevention of muscular dystrophy especially congenital muscular dystrophies resulting from laminin-alpha-2 deficiency as in the MDC1A form of Congenital Muscular Dystrophy. The patent protection in Europe will last until 2026. In the United States, a similar patent is pending. In 2008, both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) granted orphan drug designation providing market exclusivity for 10 and 7 years, respectively, following marketing approval.

"The European patent for omigapil in Congenital Muscular Dystrophy validates our in- house research and strengthens our strategic position in the field of rare neuromuscular diseases", commented Thomas Meier, Chief Scientific Officer of Santhera.

With a grant from the patient organization Association Française contre les Myopathies (AFM), Santhera completed the nonclinical development required for omigapil in support of a clinical study. Santhera is collaborating with international experts for the development of a clinical trial protocol and will seek protocol assistance and scientific advice from the FDA and the EMA in early 2011. The start of a Phase II/III program is anticipated for late 2011.

About Congenital Muscular Dystrophy
Congenital Muscular Dystrophy 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 Congenital Muscular Dystrophy 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-apoptotic compound, inhibits cell death and reduces body weight loss and skeletal deformation while increasing locomotive activity and protecting from early mortality [1].

Contact
Santhera Pharmaceuticals Thomas Meier Chief Scientific Officer T.: 061 906 89 64 E.: thomas.meier@santhera.com

Thomas Staffelbach Head Public & Investor Relations T.: 061 906 89 47 E.: thomas.staffelbach@santhera.com