Rezolute, Inc. is celebrating a significant milestone in its pursuit of a treatment for a rare and debilitating condition. On Monday, the FDA removed partial clinical holds on the company’s RZ358 (ersodetug), a potential treatment for hypoglycemia (low blood sugar) caused by congenital hyperinsulinism (HI). This green light from the FDA allows Rezolute to resume US clinical trials for this promising therapy.
Congenital HI is a genetic disorder that affects the pancreas, leading to the overproduction of insulin. This constant excess of insulin can cause dangerous drops in blood sugar levels, leading to seizures, coma, and even death. Currently, there are limited treatment options available for patients with this condition.
Ersodetug is being studied in the sunRIZE study, a global Phase 3 trial. The FDA’s decision to remove the clinical holds means Rezolute can now proceed with study start-up activities in the US, with the goal of including American participants in the ongoing global trial. The company expects US enrollment to begin in early 2025, leading to the potential announcement of topline data from the study in the second half of 2025.
“We are delighted that FDA has completely removed the partial clinical holds and are allowing us to proceed in the U.S. at all doses and in participants as young as three months of age as part of our ongoing global study,” said Nevan Charles Elam, CEO and Founder of Rezolute.
“Of note, FDA specifically concluded that the liver toxicity observed in Sprague Dawley rats is likely strain-specific and not otherwise relevant to humans,” Charles Elam continued.
This news has been well-received by investors. RZLT stock saw a 14.5% jump in price following the FDA’s announcement. The company’s commitment to advancing ersodetug in both congenital and tumor-associated HI makes it a promising player in the rare disease space.
This positive development is a significant step forward in the fight against congenital hyperinsulinism and could bring much-needed relief to patients struggling with this serious condition. With the US now included in the sunRIZE study, Rezolute is closer than ever to bringing this innovative treatment to market.
