| Program | Phase 1 | Phase 2 | Phase 3 | Next Milestone | Milestone Expected |
|---|---|---|---|---|---|
| RZ358 (ersodetug)Congenital Hyperinsulinism (cHI) | Topline data | 2H 2025 | |||
| RZ358 (ersodetug)Tumor Hyperinsulinism (tHI) | Patient enrollment in Phase 3 study | 1H 2025 | |||
| RZ358 (ersodetug)Congenital Hyperinsulinism (cHI) | ||
|---|---|---|
| Phase 1 | Phase 2 | Phase 3 |
| Next Milestone: Topline data | ||
| Milestone Expected: 2H 2025 | ||
| RZ358 (ersodetug)Tumor Hyperinsulinism (tHI) | ||
| Phase 1 | Phase 2 | Phase 3 |
| Next Milestone: Patient enrollment in Phase 3 study | ||
| Milestone Expected: 1H 2025 | ||
Congenital Hyperinsulinism
Congenital hyperinsulinism (cHI) is a rare pediatric genetic disorder characterized by excessive production of insulin by the pancreas. If left untreated, elevated insulin levels can cause extreme hypoglycemic (low blood sugar) events, increasing the risk of neurologic and developmental complications, including persistent feeding problems, learning disabilities, recurrent seizures, brain damage or even death.
In the Phase 2 RIZE study in patients with cHI ages two and older, nearly all participants achieved significant improvement in hypoglycemia across multiple endpoints, including the primary and key secondary endpoints planned for the sunRIZE study. At doses and exposures that are planned for sunRIZE, RZ358 (ersodetug) was generally safe and well-tolerated, and resulted in median improvements in hypoglycemia of up to ~90% at top doses.
sunRIZE, a Phase 3, multi-center, double-blind, randomized, controlled, safety and efficacy registrational study is ongoing. Topline results are expected in second half of 2025.
Tumor Hyperinsulinism
Tumor HI may be caused by two distinct types of tumors: islet cell tumors (ICTs) and non-islet cell tumors (NICTs), both of which lead to hypoglycemia due to excessive activation of the insulin receptor. Insulinomas are the most common type of functional ICT and cause hypoglycemia because of over production of insulin. NICTs can cause hypoglycemia by producing and secreting insulin-like paraneoplastic substances such as IGF-2 or related variants that bind to and activate the insulin receptor. This form of hypoglycemia can occur in multiple tumor types including hepatocellular carcinoma, fibrosarcomas, and mesotheliomas, among others.
Rezolute has received FDA clearance of its investigational new drug (IND) application for a Phase 3, multi-center, double-blind, randomized, controlled, safety and efficacy registrational study for RZ358 (ersodetug) in patients with hypoglycemia resulting from tumor HI.