FDA Lifts Partial Clinical Holds on RZ358 for the Treatment of Congenital Hyperinsulinism and Authorizes U.S. Inclusion in Ongoing Phase 3 Study
Rhea-AI Summary
Rezolute, Inc. (Nasdaq: RZLT) announced that the FDA has lifted partial clinical holds on RZ358 (ersodetug), their potential treatment for hypoglycemia caused by congenital hyperinsulinism (HI). This allows the company to include U.S. participants in their ongoing global Phase 3 sunRIZE study. Rezolute plans to begin U.S. enrollment in early 2025, aiming to announce topline data in the second half of 2025. The FDA concluded that liver toxicity observed in rats is likely strain-specific and not relevant to humans. This development follows FDA clearance for a separate Phase 3 study in tumor-associated HI, positioning Rezolute to advance two Phase 3 rare disease programs globally.
Positive
- FDA lifted partial clinical holds on RZ358 (ersodetug) for congenital hyperinsulinism treatment
- Approval to include U.S. participants in ongoing global Phase 3 sunRIZE study
- FDA concluded liver toxicity in rats likely not relevant to humans
- Advancing two Phase 3 rare disease programs globally
Negative
- U.S. enrollment not expected to begin until early 2025
- Topline data from the study not anticipated until second half of 2025
Insights
The FDA's decision to lift the partial clinical holds on RZ358 (ersodetug) for congenital hyperinsulinism (HI) treatment is a significant milestone for Rezolute. This allows the inclusion of U.S. participants in the ongoing global Phase 3 sunRIZE study, potentially accelerating the drug development process. The FDA's conclusion that the liver toxicity observed in rats is likely strain-specific and not relevant to humans addresses a major safety concern. This development could expand the patient pool for the study, including participants as young as three months old, which is important for a rare pediatric disease like congenital HI. The anticipated U.S. enrollment in early 2025 and potential topline data release in the second half of 2025 provide a clear timeline for investors to monitor progress.
This regulatory breakthrough for Rezolute (NASDAQ: RZLT) could have positive implications for the company's financial outlook. The removal of partial clinical holds on RZ358 potentially reduces development risks and costs associated with separate U.S. trials. Inclusion of U.S. sites in the global Phase 3 study may accelerate patient recruitment, potentially leading to earlier completion and data readout. This could shorten the time to market if the drug proves successful. Moreover, with two Phase 3 programs now advancing in the U.S. and globally, Rezolute's pipeline value may increase. Investors should note that while this news is positive, the company still faces the challenges of completing clinical trials and obtaining regulatory approval, which are critical for future revenue generation.
The FDA's decision to lift the partial clinical holds on RZ358 could strengthen Rezolute's position in the rare disease market. Congenital hyperinsulinism, while rare, represents an unmet medical need with treatment options. The inclusion of U.S. patients in the global Phase 3 study may enhance the drug's market potential if approved, as it would likely facilitate eventual FDA approval for the U.S. market. The company's unique position with two Phase 3 programs for different forms of hyperinsulinism (congenital and tumor-associated) could allow it to address a broader patient population. This diversification might reduce market risks and potentially increase the overall market opportunity. However, investors should remain cautious as success in clinical trials and regulatory approvals are still important hurdles to overcome before any market penetration can be achieved.
Commencing study startup activities in the U.S.; participant enrollment anticipated in early 2025
REDWOOD CITY, Calif., Sept. 09, 2024 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), a late-stage rare disease company developing a novel therapy to treat hyperinsulinism (HI), today announced that the U.S. Food and Drug Administration (FDA) has removed the partial clinical holds on RZ358 (ersodetug), a potential treatment for hypoglycemia caused by congenital HI.
Ersodetug is currently being studied in sunRIZE, a global Phase 3, multi-center, double-blind, randomized, placebo-controlled, safety and efficacy registrational study in participants with congenital HI. The Company will now commence study start-up activities in the U.S. with the goal of including U.S. participants in the global sunRIZE study. The Company anticipates potential U.S. enrollment to begin in early 2025, which will enable 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. Of note, FDA specifically concluded that the liver toxicity observed in Sprague Dawley rats is likely strain-specific and not otherwise relevant to humans,” said Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute. “Coming on the heels of our recent announcement of FDA clearance of a separate Phase 3 study in tumor-associated HI, we are in the unique and fortunate position to be advancing ersodetug in two Phase 3 rare disease programs in the U.S. and globally.”
For more information on ersodetug and our programs in congenital HI and tumor HI, please visit https://www.rezolutebio.com/pipeline/.
About Ersodetug
Ersodetug is a fully human monoclonal antibody that binds to a unique allosteric site on insulin receptors to counteract the effects of insulin receptor over-activation by insulin and related substances (such as IGF-2), thereby improving hypoglycemia in the setting of hyperinsulinism. Because ersodetug acts downstream from the pancreas, it has the potential to be universally effective at treating hypoglycemia due to any form of HI.
About sunRIZE
The Phase 3 sunRIZE study is a multi-center, randomized, double-blind, placebo-controlled, parallel arm study designed to evaluate the efficacy and safety of ersodetug in patients with congenital HI who are experiencing poorly controlled hypoglycemia. Participants between the ages of 3 months to 45 years old are eligible to participate. The study is enrolling up to 56 participants in more than a dozen countries around the world.
Forward-Looking Statements
Any statements in this press release about the Company’s future expectations, plans and prospects, including statements regarding the public offering, constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Forward-looking statements include any statements about the Company’s strategy, future operations and future expectations and plans and prospects for the Company, and any other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend”, “goal,” “may”, “might,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions. These forward-looking statements include statements about RZ358 as a sunRIZE Phase 3 study, the Phase 2 RIZE study, the complete removal of the partial clinical holds on RZ358 (ersodetug) for the treatment of hypoglycemia, the ability of RZ358 to become an effective treatment for congenital hyperinsulinism, the effectiveness or future effectiveness of RZ358 to become an effective treatment for congenital hyperinsulinism, and statements regarding the timeliness of the ongoing global Phase 3 sunRIZE study. These forward-looking statements are based on information currently available to the Company and its current plans or expectations, and are subject to a number of uncertainties and risks that could significantly affect current plans. Such forward-looking statements involve substantial risks and uncertainties that could cause the Company’s development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, those related to market and other financial conditions, and other factors discussed in the “Risk Factors” section contained in the reports that the Company files with the SEC. Any forward-looking statements represent the Company’s views only as of the date of this press release. The Company anticipates that subsequent events and developments will cause its views to change. While the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so except as required by law.
Contacts:
Rezolute, Inc.
Christen Baglaneas
cbaglaneas@rezolutebio.com
508-272-6717
LHA Investor Relations
Tirth T. Patel
tpatel@lhai.com
212-201-6614
FAQ
What is the latest FDA decision regarding Rezolute's RZ358 (ersodetug)?
When does Rezolute (RZLT) expect to begin U.S. enrollment for the RZ358 Phase 3 study?
What did the FDA conclude about the liver toxicity observed in rats during RZ358 trials?
How many Phase 3 rare disease programs is Rezolute (RZLT) currently advancing?
