On May 15, 2024 Rezolute, Inc., a clinical-stage biopharmaceutical company committed to developing novel, transformative therapies for serious metabolic and rare diseases, reported financial results and provided a business update for the three months ended March 31, 2024 (Press release, Rezolute, MAY 15, 2024, View Source [SID1234643338]).

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"In the past few months, we have been focused on global site activation and patient enrollment for sunRIZE, a pivotal Phase 3 clinical study of RZ358 in patients with congenital hyperinsulinism. We have also completed dosing of patients with diabetic macular edema for our Phase 2 multi-center clinical study of RZ402 and we expect to announce topline results from that study this month," said Nevan Elam, Chief Executive Officer and Founder of Rezolute. "Additionally, in the U.S. we continue to have productive interactions with FDA as we work towards achieving liberalization of the partial clinical holds on sunRIZE."

Recent Pipeline Progress and Anticipated Milestones

Congenital Hyperinsulinism (cHI)

Patient enrollment underway in sunRIZE, a global, pivotal Phase 3 clinical study of RZ358 in patients with cHI in Europe and other geographies outside of the U.S.
Enrollment is expected to complete by the end of 2024.
Topline results expected in mid-2025.

As part of the effort to resolve the partial clinical holds in the U.S., the Company conducted and recently completed an in-vivo toxicology study in brown Norway rats using Sprague Dawley (SD) rats as a positive control.
Early results show that at the highest tested dose of 40 mg/kg, there were no observed liver abnormalities in the brown Norway rat, which is approximately four times higher than the dose that results in microvascular liver abnormalities in SD rats. Additionally, at the 40 mg/kg dose in this study, SD rats had liver abnormalities consistent with previous in-vivo studies.
The Company believes that the Norway rat study adds to the body of evidence that the rat findings are specific to the SD rat and are not otherwise relevant, based on the absence of findings in other rat strains, other rodent species (CD-1 mice), primates, and humans in studies to date.
Final data tabulations and a report for this study will be completed in the coming weeks. The company plans to submit this study as well as additional in-vitro information to the FDA this summer as part of a complete response to the partial clinical holds.
Tumor Hyperinsulinism (HI)

Alignment with FDA to conduct a potential late-stage, registrational, clinical study in both non-islet cell tumor hypoglycemia (NICTH) and insulinoma patients, which would be the second development program and rare disease indication for RZ358.
To date, five metastatic insulinoma patients have been treated with RZ358 in the Expanded Access Program (EAP).
Potential initiation of a development program for this indication is currently under evaluation by the Company. Updates will be provided later this year.

Diabetic Macular Edema (DME)

Completed patient dosing for Phase 2 U.S., multi-center clinical study in 94 participants with DME who are naïve to or have received limited anti-VEGF injections.
Primary endpoints include (i) stabilization of disease and/or change in study eye macular central subfield thickness, as measured by Spectral Domain Ocular Coherence Tomography, (ii) change in study eye visual acuity as measured by the early treatment diabetic retinopathy scale, (iii) the repeat dose pharmacokinetics of RZ402 in patients with DME, and (iv) the safety and tolerability of RZ402.
Topline results expected in May 2024.

Fiscal Third Quarter Financial Results

Cash, cash equivalents and investments in marketable securities were $81.6 million as of March 31, 2024, compared with $118.4 million as of June 30, 2023.

Research and development expenses were $12.4 million for the third quarter of fiscal 2024, compared with $14.2 million for the same period a year ago, with the decrease primarily attributable to a reduction in milestone expense of $3.0 million due to Phase 2 dosing milestone triggered in RZ402, with no comparative expense incurred in the current year, offset partially by an increase of R&D personnel-related expenses due to increased headcount.

General and administrative expenses were $3.8 million for the third quarter of fiscal 2024, compared with $2.9 million for the same period a year ago, with the increase primarily attributable to personnel-related expenses due to increased headcount.

Net loss was $17.1 million for the third quarter of fiscal 2024 compared with a net loss of $15.7 million for the same period a year ago.