Santhera Prepares for Pivotal Study with Omigapil in Congenital Muscular Dystrophy

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


Contact:
Santhera Pharmaceuticals Klaus Schollmeier Chief Executive Officer T.: 061 906 89 52 E.: klaus.schollmeier@santhera.com