Rezolute, Inc., a US bio-venture with Handok and Genexine as the largest shareholders, announced major results of its Phase 2b RIZE study of RZ358 in patients with congenital hyperinsulinism (HI) at the Pediatric Endocrine Society 2022 on May 1 (US time).

The study involved repeatedly measuring blood glucose levels using a continuous glucose monitoring system (CGM). Results showed meaningful effects as the time to reach severe hypoglycemia (less than 50 mg/dl) was reduced by up to 75% in the RZ358 administered group, and the number of hypoglycemia events was also improved.

Dr. Paul Thornton, a pediatric endocrinologist at Cook Children’s Health Care System, said, “As confirmed in the RIZE clinical trial, patients with congenital HI continue to experience hypoglycemia.” He added, “The positive results of this study on improvement in hypoglycemia suggests that it could be an important treatment for HI.”

The RIZE study enrolled patients with congenital HI with an average age of 6.5 years, including 16 patients between the ages of 2 and 6 years old, and with substantial continued hypoglycemia despite being on currently available therapies. Three doses (3 mg/kg, 6 mg/kg, 9 mg/kg) were administered to the patient participants, and their blood glucose was monitored using a CGM. As a result, the time for blood sugar to fall to hypoglycemic level (less than 70 mg/dL) accounted for 23% of total monitoring time, while the average number of hypoglycemic events decreased from 15.5 at the start of treatment to 7.5 at the end of treatment.

In addition, hypoglycemia was improved in more than 50% of high-dose cohort patients, and there were no drug side effects or clinically significant glycosemia. Improvement in hypoglycemia exceeded expectations across multiple indicators, including the number of events and duration of hypoglycemia. Furthermore, high levels of safety and efficacy were confirmed across all doses with no cases of discontinuation of the study midway due to intolerable drug side effects for the patient. The results of this study confirmed that RZ358 has a wide range of therapeutic potential for all forms of hyperinsulinemia, as well as the possibility of it being used as monotherapy and the acquisition of important clinical results for phase 3 clinical trials.

Furthermore, Rezolute recently raised a total of USD 130 million in investment through direct offering and private placements, in which the company directly distributes its stock to institutions. Rezolute plans to use the funds for the phase 3 clinical trial of RZ358 for the treatment of congenital HI, the Phase 2 clinical trial for RZ402 for the treatment of diabetic macular edema, and additional pipeline expansion.

Rezolute is a US bio-venture developing targeted treatments for rare and metabolic diseases and was listed on the NASDAQ in 2020. Major R&D pipelines include RZ358, a treatment for the rare pediatric endocrine disease congenital hyperinsulinism (HI), and RZ402, an oral plasma kallikrein inhibitor for the treatment of diabetic macular edema. Handok holds commercialization rights for these two products in Korea.