Santhera Pharmaceuticals has announced that the first patient in the CALLISTO Phase I study assessing the pharmacokinetics, safety and tolerability of oral omigapil in patients with Congenital Muscular Dystrophy (CMD) has been dosed and all participating patients have been recruited.
This study, which is being conducted at the US National Institutes of Health (NIH), will also evaluate the feasibility of conducting disease-relevant clinical assessments that could be used as endpoints in future efficacy trials in pediatric and adolescent CMD patients. Omigapil is a drug candidate in-licensed from Novartis and repositioned by Santhera for development in CMD.
The study is being conducted at the NIH's National Institute of Neurological Disorders and Stroke (NINDS) in Bethesda, Maryland, and will evaluate use of the compound in 20 ambulatory and non-ambulatory patients aged 5 to 16 years affected by either Ullrich or MDC1A subtypes of CMD.
All patients have been selected, pre-screened and randomly assigned to one of the three study cohorts that are starting sequentially with ascending doses of omigapil. Following a 4-week vehicle run-in phase, each patient will receive one of three doses of omigapil (0.02, 0.08 or 0.2 mg/kg/day) for 12 weeks, during which period the pharmacokinetics of omigapil will be assessed as well as the patients' respiratory function, muscle strength and motor function.
To ensure the collection of as much information as possible from both subtypes of CMD, Ullrich or MDC1A, all patients have been identified and randomized prior to the first dosing.
The first patient received the starting dose of 0.02 mg/kg/day, applied in a new liquid formulation developed by Santhera for this patient population. Only after an independent data and safety monitoring board (DSMB) has assessed the pharmacokinetic and safety data in each completed cohort, dosing in subsequent cohorts will follow at ascending doses. Due to the staggered dosing of patients, the study is expected to run until the end of 2016.
"We are very excited about the start of this trial with the first investigational new drug for this group of severe neuromuscular diseases where children are affected with ultimately devastating loss of functional muscle and no treatment currently available to slow down or stop progression of the disease", commented Carsten Bönnemann, MD, a senior investigator in the NINDS Neuromuscular and Neurogenetic Disorders of Childhood Section and Principal Investigator of this study. "To ensure as much information as possible can be collected from patients with both Ullrich or MDC1A subtypes of CMD in this trial, it was important to have all patients identified and randomized prior to the first patient commencing treatment."
"We are very proud of being able to collaborate with the NIH in developing omigapil for CMDs", emphasized Thomas Meier, PhD, Chief Executive Officer of Santhera. "We are thankful to the patients and families who are making tremendous efforts to enroll into CALLISTO, which is the first intervention trial with an investigational medicinal product in this group of neuromuscular diseases."