Santhera Announces Successful Completion of First Clinical Trial with Omigapil in Patients with Congenital Muscular Dystrophy

Santhera Pharmaceuticals Holding AG / Santhera Announces Successful Completion of First Clinical Trial with Omigapil in Patients with Congenital Muscular Dystrophy . Processed and transmitted by Nasdaq Corporate Solutions. The issuer is solely responsible for the content of this announcement.

Pratteln, Switzerland, April 5, 2018 - Santhera Pharmaceuticals (SIX: SANN) reports the successful completion of the first clinical trial with omigapil in patients with two forms of congenital muscular dystrophy (CMD) conducted in the US at the National Institutes of Health (NIH). The ascending multiple dose cohort study (CALLISTO) met its primary objective to establish a favorable pharmacokinetic profile of omigapil and demonstrated that the study drug was safe and well tolerated in children and adolescents with CMD. Following further data analysis, the Company will discuss these results with clinical experts and regulatory authorities to prepare for a pivotal trial in patients with CMD.

The single-center interventional trial to establish the pharmacokinetic profile and to evaluate the safety and tolerability of omigapil in pediatric and adolescent patients with CMD was conducted at the NIH's clinical center in Bethesda, Maryland (USA), and led by Carsten Bönnemann, MD, and A. Reghan Foley, MD, of the NIH's National Institute of Neurological Disorders and Stroke. Ambulant and non-ambulant patients aged 5-16 years with either of two of the most common forms of CMD resulting from collagen VI-deficiency (COL6-related dystrophies or COL6-RDs) or laminin alpha2-deficiency (LAMA2-related dystrophy or LAMA-RD) were eligible to participate in the trial. A total of 20 patients were enrolled in this ascending multiple dose cohort study. Participants were randomized to one of five groups and received omigapil at a once-daily dose ranging from 0.02 mg/kg to 0.08 mg/kg body weight as a liquid oral formulation for a period of 3 months.

The trial met its primary objective and established that the pharmacokinetic profile of omigapil is suitable for further development in pediatric patients and demonstrated that omigapil was safe and well tolerated in this fragile population of CMD patients.

"We are grateful to participating patients and their families for enrolling in this first interventional trial with a drug candidate for CMD and to the clinical researchers at the NIH for their dedication to this milestone trial for these forms of CMD," said Thomas Meier, PhD, CEO of Santhera. "This is an important step towards profiling the therapeutic potential of omigapil for the LAMA2 and COL6 related forms of CMD for which there is currently no treatment available. We will now collaborate with international experts and seek advice from regulators to further advance the clinical development of omigapil towards a pivotal trial."