Santhera and Parent Project Muscular Dystrophy (PPMD) Team Up on Benefit/Risk Study in Duchenne Muscular Dystrophy

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Liestal, Switzerland, November 25, 2014 - Santhera Pharmaceuticals (SIX: SANN) and Parent Project Muscular Dystrophy (PPMD), the leading US advocacy organization working to end Duchenne Muscular Dystrophy (DMD), will collaborate on a benefit/risk study in DMD. The study will focus specifically on patient and caregiver preferences regarding pulmonary therapies in the disease, and will be based on data from Santhera's successful phase III clinical trial with Catena^®/Raxone^® (idebenone).

Earlier this year, PPMD convened a broad coalition of over 80 stakeholders to draft and submit the first-ever patient advocacy-initiated guidance for a rare disease to the U.S. Food and Drug Administration (FDA) to help accelerate development and review of potential therapies for DMD. One of the recommendations in the guidance was to create partnerships between patient groups and industry to study the benefit/risk preferences of the disease community to better inform the company's new drug application.

Pursuant to this recommendation, Santhera and PPMD have now initiated such a partnership and collaborate to study patient and caregiver benefit/risk preferences for pulmonary therapy with Santhera's Catena/Raxone. The study will be based on data from Santhera's successful Phase III (DELOS) trial which provided clear evidence of a clinical benefit for Catena/Raxone in delaying the loss of respiratory function in DMD patients not using concomitant glucocorticoid steroids. The preservation of respiratory function is considered to be of major clinical importance.

"Following the successful outcome of our Phase III trial, we are excited about this collaboration with PPMD to determine patients' and caregivers' views of the benefit/risk balance for Catena/Raxone", emphasized Thomas Meier, CEO of Santhera. "PPMD has become a powerful and reliable partner of industry in facilitating the development of promising treatment options. With this study of the benefit/risk preferences of the disease community, which is the first of its kind, we will proactively meet regulators' expectations with regard to the content of our NDA filing."