Pre-clinical development of a novel oral therapy for hyperinsulinemic hypoglycaemia

Newborns with a rare disease called Congenital Hyperinsulinism cannot stop producing insulin, causing their blood sugar to crash to dangerously low levels within hours of birth. This matters because these repeated, severe hypoglycaemic episodes can permanently damage a baby’s developing brain, leading to lifelong learning disabilities and other neurological problems. Current treatments are limited: some require major surgery to remove most of the pancreas, while others are difficult to administer or have serious side effects. There is no safe, easy-to-use oral therapy available. This project brings together a drug discovery company, a university, and a specialist children’s hospital to develop a pill that could be taken by mouth to safely control insulin levels. If successful, the research will produce a molecule ready for clinical trials—not just for Congenital Hyperinsulinism, but also for other forms of intractable hypoglycaemia, including in newborns, patients who have had gastric bypass surgery, and people with insulin-secreting tumours. A clinically ready oral therapy would transform care for these patients, replacing invasive procedures with a simple daily tablet that could prevent brain damage from the first days of life.