Omigapil, an investigational therapy by Santhera Pharmaceuticals, proved to be safe and well-tolerated in children and adolescents with congenital muscular dystrophy (CMD), in a Phase 1 clinical trial.
Switzerland-based Santhera recently announced the successful completion of the CALLISTO trial (NCT01805024), which tested omigapil in pediatric patients with two of the most common types of CMD.
“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 [National Institutes of Health] for their dedication to this milestone trial for these forms of CMD,” Thomas Meier, PhD, CEO of Santhera, said in a press release.
Omigapil is a compound with anti-apoptotic properties, meaning it helps cells avoid death. Animal studies showed that omigapil prevents cell death, increases body weight, and reduces skeletal deformation, while improving movement ability and survival.
In this single-center, interventional, open-label Phase 1 trial, researchers evaluated omigapil’s properties, safety, and tolerability in 20 ambulatory and non-ambulatory patients between 5 and 16 years old with either the Ullrich or MDC1A subtypes of CMD.
Conducted at the NIH’s clinical center in Bethesda, Maryland, the trial was led by Carsten Bönnemann, MD, and A. Reghan Foley, MD, of the NIH’s National Institute of Neurological Disorders and Stroke.
Patients enrolled in the study were randomly divided into one of five groups and received omigapil once daily at a dose ranging from 0.02 mg/kg to 0.08 mg/kg body weight for three months.
Data collected from the trial showed that omigapil is safe and well-tolerated in pediatric patients, and its profile is suitable for further development in this CMD patient population.
“This collaboration with Santhera and the patient community allowed us to test for the first time an investigational therapy in children with these more common types of CMD for which no other treatment options are currently available,” Bönnemann said. “With the help of Ken Cheung, PhD at Columbia University, this clinical trial applied an innovative design by utilizing a novel adaptive dose-finding algorithm.”
He also said they plan to share data from the trial at upcoming conferences and with the patient community, and will work with stakeholders and regulators to prepare for a pivotal trial on omigapil.
“Cure CMD and the CMD community are thrilled that this first-ever interventional trial for congenital muscular dystrophy has been successfully completed, in partnership with the NIH and Santhera Pharmaceuticals. For the affect individuals and their families who enrolled in CALLISTO, trial participation represented a considerable burden, and we are forever grateful for their commitment to seeing this through to the end,” said Rachel Alvarez, director of operations for Cure CMD, a leading nonprofit organization focused on finding treatments and supporting the CMD community.
Santhera focuses on the development and commercialization of innovative medicines for orphan diseases or other diseases with high unmet medical needs. The company obtained an exclusive license for omigapil from Novartis for the treatment of CMD. It was designated an orphan drug for CMD in the U.S. and Europe, and was granted fast track designation in the U.S.