| Program | Phase 1 | Phase 2 | Phase 3 | Next Milestone | Milestone Expected |
|---|---|---|---|---|---|
| RZ358 (ersodetug)Congenital Hyperinsulinism (cHI) | Topline data | Mid-2025 | |||
| RZ358 (ersodetug)Tumor Hyperinsulinism (tHI) | Patient enrollment in Phase 3 study | 1H 2025 | |||
| RZ402Diabetic Macular Edema (DME) | POC complete; Available for partnering | - | |||
| RZ358 (ersodetug)Congenital Hyperinsulinism (cHI) | ||
|---|---|---|
| Phase 1 | Phase 2 | Phase 3 |
| Next Milestone: Topline data | ||
| Milestone Expected: Mid-2025 | ||
| RZ358 (ersodetug)Tumor Hyperinsulinism (tHI) | ||
| Phase 1 | Phase 2 | Phase 3 |
| Next Milestone: Patient enrollment in Phase 3 study | ||
| Milestone Expected: 1H 2025 | ||
| RZ402Diabetic Macular Edema (DME) | ||
| Phase 1 | Phase 2 | Phase 3 |
| Next Milestone: POC complete; Available for partnering | ||
| Milestone Expected: - | ||
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 mid-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.
Diabetic Macular Edema
Diabetic Macular Edema (DME) is a severe, systemic, vision-threatening complication of diabetic retinopathy (DR) characterized by swelling of the retina and thickening of the macula, the part of the eye that is responsible for high-resolution vision. Anti-VEGF injections into the eye are the current standard of care for DME, requiring continued administration over long periods of time to preserve vision. Due to their invasive route of administration and occasional serious side effects, there is a tendency to delay treatment until later in the disease course, and long-term compliance with eye injection regimens can be difficult for patients. Coupled with inadequate responsiveness in some patients, this leads to overall undertreatment and suboptimal vision outcomes in DME patients. DME is a result of a systemic microvascular complication of diabetes, manifesting in the retinal blood vessels behind the eyes, and therefore commonly affects both eyes. With currently available intravitreal anti-VEGF therapies, two separate eye injections are required, or treatment of one eye or both eyes may be deferred, when both eyes are affected.
RZ402 is an oral, small molecule, selective and potent, plasma kallikrein inhibitor (PKI), for the chronic treatment of DME. By inhibiting the activation of kallikrein, RZ402 is designed to block bradykinin production and its resulting effects on vascular leakage and inflammation. Topline results from the Phase 2 study of RZ402 in patients with DME demonstrated a significant reduction in central subfield thickness (CST) in the Study Eye at all RZ402 dose levels compared to placebo (up to approximately 50 micron improvement) and was safe and well-tolerated.