Zealand Pharma Reports Positive Results from Phase 3 Trial of Therapy for Congenital Hyperinsulinism

Zealand Pharma reported positive top-line results from the second phase 3 clinical trial of its experimental therapy dasiglucagon in pediatric patients with the ultra-rare condition congenital hyperinsulinism.

Photo: Adam Steensberg, president and CEO of Zealand Pharma

Congenital hyperinsulism (CHI) affects mainly newborns, infants, and toddlers. Due to a genetic defect in the insulin producing cells, these children have increased insulin levels, resulting in persistent and recurrent hypoglycemia throughout childhood. Current treatment options are limited, complex, and may be insufficient to adequately control hypoglycemia.

Dasiglucagon is a glucagon analog that is stable in aqueous solution and is thus suitable for chronic pump use. In 2017, both the U.S. Food and Drug Administration (FDA) and the European Commission granted orphan drug designation for dasiglucagon for the treatment of CHI. In 2021 Zealand initiated a collaboration with DEKA on utilizing their continuous infusion pump for the potential treatment of CHI with dasiglucagon. Under the terms of this agreement Zealand is responsible for distribution and commercialization of the drug-device combination.

Dasiglucagon met the primary endpoint of the global trial involving 12 children with CHI (ages 7 days to 12 months) by demonstrating a statistically significant difference between treatment arms. Baseline intravenous glucose infusion rate (IV GIR) was 15.7 mg/kg/min. Dasiglucagon treatment resulted in a significant reduction in the mean IV GIR versus placebo as measured during the last 12 hours of each treatment period (4.3 mg/kg/min for dasiglucagon and 9.4 mg/kg/min for placebo with a treatment difference of 5.2 mg/kg/min; p=0.0037). Dasiglucagon was assessed as well tolerated in the trial with an overall safety profile consistent with that reported in the previous phase 3 trial of older children with CHI (ages 3 months to 12 years). In total, 42 of 44 participants enrolled across the dasiglucagon CHI phase 3 clinical programs continued into the ongoing safety extension trial.

Based on the trial’s results, Zealand Pharma said it will engage discussions with the FDA for the submission of an application for marketing approval. Dasiglucagon has the potential to become the first medicine developed specifically for this ultra-rare disease in more than three decades.

“CHI is a serious ultra-rare condition with a significant unmet medical need which places a tremendous burden on both patients and families,” said Adam Steensberg, president and CEO of Zealand Pharma. “We believe the outcome of this trial supports the potential of dasiglucagon as a novel treatment for those living with CHI.”

Author: Rare Daily Staff