Eiger BioPharmaceuticals has reported positive findings from a Phase II trial where its investigational therapy avexitide showed to substantially lower the chances of fasting and protein-induced hypoglycemia in children with congenital hyperinsulinism (HI).

A glucagon-like peptide-1 receptor (GLP-1r) antagonist, avexitide is being developed to treat congenital HI and post-bariatric hypoglycemia (PBH). 

Conducted at Children’s Hospital of Philadelphia, US, the investigator-sponsored, open-label, crossover trial analysed the effects of avexitide versus a saline control on fasting- and protein-induced hypoglycemia in children with HI. 

Findings showed that treatment with avexitide resulted in a 76% and 84% decline in the chances of fasting hypoglycemia in the mid-dose and high dose groups, respectively, versus control. 

Administering avexitide during protein tolerance testing led to an 82% decline in the chances of hypoglycemia. 

Furthermore, the mid-dose group showed a 20% rise in fasting glucose while the high dose led to a 28% rise in glucose following food intake and a 30% rise in glucose after protein challenge.

The Phase II trial offers further evidence of avexitide’s targeted therapeutic approach for the treatment of HI and supports progressing the therapy into the Phase III stage.

The company has developed a subcutaneous formulation of avexitide for the Phase III registration programme, anticipated to begin by the end of this year.

Eiger BioPharmaceuticals president and CEO David Cory said: “Their early investigation of avexitide in congenital hyperinsulinism generated important foundational proof of concept data in this patient population. 

“Armed with these data, we are moving forward into Phase III this year as we believe avexitide has the potential to transform the lives of children with this life-threatening condition.”

A rare genetic disease, congenital HI is the most prevalent cause of persistent hypoglycemia in infants and children. 

In December last year, the company obtained Data Safety Monitoring Board recommendation to progress the Phase III TOGETHER trial of Peginterferon Lambda without any changes.