Rezolute’s lead candidate, RZ358, is an antibody in Phase 2b development that is designed to prevent severe, persistent low blood sugar in patients with congenital HI.
Congenital hyperinsulinism (HI) is an ultra-rare pediatric genetic disorder characterized by excessive production of insulin by the pancreas. If untreated, it can lead to dangerously low blood sugar levels. There is no satisfactory treatment or cure for congenital HI.
Children usually begin to show symptoms, such as poor feeding, seizures, unresponsiveness, irritability and sleepiness, within the first few days of life or during infancy. Repeat episodes of dangerously low blood sugars increase the risk of neurological and developmental complications, including persistent feeding problems, learning disabilities, recurrent seizures, brain damage or even death.
Not all patients respond to existing medical options, which are often associated with side effects that discourage compliance and lead to suboptimal treatment outcomes. Surgical removal of the pancreas is sometimes an option, but this approach is invasive, may require repeat surgeries and may lead to the development of lifelong insulin-dependent diabetes.
There is a significant unmet medical need to develop safer, well-tolerated and effective therapies aimed at preventing chronic recurrent low blood sugar levels for patients with congenital HI and RZ358 may fill this need.
RZ358 is an intravenously administered human monoclonal antibody that binds strongly and specifically to a unique site on the insulin receptor found across effector cells throughout the body in the liver, fat and muscle. This action allows RZ358 to safely counteract the effects of elevated insulin in the body.
RZ358 has received orphan drug designation in the United States and European Union, highlighting its potential as an effective therapeutic in a disease area with great unmet need.