Hanmi Pharmaceutical's "Efegerglucagon" Shows Excellent Safety and Efficacy in Interim Phase 2 Analysis

A New Hope for Patients with Ultra-Rare Congenital Hyperinsulinism

Hanmi Pharmaceutical announced on May 26 that the Phase 2 clinical trial for "Efegerglucagon," an innovative new drug for congenital hyperinsulinism being developed as the world's first once-weekly formulation, is progressing smoothly.

Hanmi Pharmaceutical participated in the joint scientific meeting of the European Society for Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE), held in Copenhagen, Denmark from May 10 to 13. At the conference, the company presented interim results from the Phase 2 clinical trial of "Efegerglucagon," which is being developed as a treatment for congenital hyperinsulinism, through both oral and poster presentations.

Congenital hyperinsulinism is a rare disease characterized by excessive insulin secretion, leading to hypoglycemia. It occurs in approximately 1 out of every 25,000 to 50,000 people, with about 300 new cases diagnosed each year in the United States and Europe. Although one approved treatment currently exists, its efficacy is limited to certain genotypes and it is associated with numerous side effects. As a result, patients often resort to off-label medications or undergo pancreatectomy surgery despite the risks of adverse effects.

Hanmi Pharmaceutical is developing "Efegerglucagon" as the world's first once-weekly formulation of an innovative new drug for congenital hyperinsulinism, aiming to overcome the limitations of existing treatments. The global Phase 2 clinical trial is currently underway in five countries. In Korea, Professor Choi Jin-ho of the Department of Pediatrics and Adolescent Medicine at Asan Medical Center in Seoul is participating as a clinical investigator.

At the conference, Hanmi Pharmaceutical presented interim results from the Phase 2 clinical trial of Efegerglucagon, revealing data on safety, tolerability, pharmacokinetics, and efficacy after treating eight subjects enrolled in Cohort 1 for eight weeks.

Efegerglucagon demonstrated excellent safety and tolerability, with no notable findings in vital signs, physical examinations, safety laboratory tests, or electrocardiograms. In addition, no adverse events requiring discontinuation of treatment or other significant safety concerns were reported.

Throughout the entire treatment period, both the frequency and duration of weekly hypoglycemic episodes (blood glucose below 70 mg/dL) and severe hypoglycemia (blood glucose below 54 mg/dL) decreased significantly. Notably, after eight weeks, hypoglycemia below 70 mg/dL was reduced by 72.3%, and severe hypoglycemia below 54 mg/dL was reduced by 87.5%.

Furthermore, the average half-life of the drug at week 8 was found to be 89 hours, supporting the feasibility of a once-weekly dosing interval.

Dr. Antonia Dastamani, a researcher at Great Ormond Street Hospital in the United Kingdom who is conducting Hanmi Pharmaceutical's clinical research, stated, "Efegerglucagon is a promising new drug that significantly reduces the risk of hypoglycemia with just once-weekly administration. I look forward to its rapid development so that it can become a new treatment option for patients."

Lee Moonhee, General Manager of Clinical Development at Hanmi Pharmaceutical, said, "Efegerglucagon has shown excellent efficacy and safety in patients with congenital hyperinsulinism, which is very encouraging," adding, "We will devote ourselves to clinical development with the goal of improving the quality of life for suffering patients."

This content was produced with the assistance of AI translation services.