Rezolute Announces Positive Recommendation After Independent Interim Analysis of Phase 3 sunRIZE Study of Ersodetug in Congenital Hyperinsulinism (“HI”)
Rhea-AI Summary
Rezolute (NASDAQ: RZLT) received a positive recommendation from the Independent Data Monitoring Committee (DMC) for its Phase 3 sunRIZE study of ersodetug in Congenital Hyperinsulinism (HI). The DMC advised continuing the trial as planned without increasing the sample size.
The interim analysis was conducted after approximately half of enrolled patients completed primary assessments, focusing on hypoglycemia events as the primary endpoint. The company remains on track to complete enrollment in May 2025, with topline data expected in December 2025.
U.S. clinical sites are now active and enrolling patients as part of the global study. The company remains blinded to the partial study data and statistical outputs from the DMC. The recommendation aligns with previous Phase 2 RIZE study outcomes, real-world observations from their Expanded Access Program, and pharmacokinetic data from the sunRIZE study's open-label arm.
Positive
- DMC's positive recommendation validates trial design and powering assumptions
- Study progressing on schedule with no need for sample size increase
- Expansion of trial to U.S. sites enhances enrollment capabilities
- Previous Phase 2 results and real-world data support potential efficacy
Negative
- Company remains blinded to interim analysis data
- Final results not expected until December 2025
Insights
DMC recommendation to continue Phase 3 trial without increasing sample size indicates the trial is on track and appropriately powered.
The independent Data Monitoring Committee's recommendation to continue Rezolute's Phase 3 sunRIZE trial without sample size adjustment represents a significant procedural milestone. In clinical development, interim analyses are critical checkpoints where DMCs evaluate whether a study remains adequately powered to detect the pre-specified treatment effect. When no sample size increase is recommended after reviewing unblinded interim data, it typically suggests that the observed effect and variability patterns align with the statistical assumptions used in the original trial design.
The primary endpoint measuring hypoglycemia events is objective and clinically relevant, which strengthens the study's methodological robustness. The maintained timeline with enrollment completion expected in May 2025 and topline results anticipated in December 2025 indicates efficient trial execution, particularly noteworthy for a rare disease indication where patient recruitment is often challenging.
It's methodologically important that Rezolute remains blinded to the interim analysis data, as highlighted in the article. This preserves trial integrity while allowing the DMC to make necessary procedural adjustments. The company's reference to consistency with previous Phase 2 RIZE study outcomes, Expanded Access Program observations, and pharmacokinetic data provides context for their confidence, though the actual efficacy outcomes remain to be determined from the completed study.
Positive DMC recommendation maintains momentum for potentially transformative therapy in congenital hyperinsulinism, a serious rare disease with treatment options.
Congenital hyperinsulinism (HI) represents one of the most challenging conditions in pediatric endocrinology, characterized by dysregulated insulin secretion causing severe, potentially brain-damaging hypoglycemia. Current management approaches are woefully inadequate, with severe cases often requiring partial or near-total pancreatectomy, which carries significant lifelong consequences including diabetes risk.
The DMC's recommendation to continue the sunRIZE trial without modifying sample size is particularly significant in this therapeutic landscape. For rare disease clinical programs, where statistical powering is inherently challenging due to small patient populations, maintaining the original trial design suggests the observed interim data supports the pre-specified statistical assumptions.
The global expansion to include U.S. clinical sites broadens access to this investigational therapy while potentially accelerating the pathway to regulatory submission if results prove positive. While specific mechanism details aren't provided, the company's characterized confidence that ersodetug could be "a best-in-class treatment option" suggests it may address fundamental aspects of the disease pathophysiology.
For patients with congenital HI and their families, who face extreme challenges managing dangerous hypoglycemic episodes, the continued progress of this Phase 3 program represents hope for a potential advance in care standards, though final efficacy determination awaits completion of the full trial in December 2025.
Independent Data Monitoring Committee (the “DMC”) recommends continuation of sunRIZE trial as planned with no need to increase sample size
Enrollment on track and expected to be completed in May 2025; topline data anticipated in December 2025
U.S. sites activated and enrolling patients
REDWOOD CITY, Calif., April 23, 2025 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (the “Company or “Rezolute”), a late-stage biopharmaceutical company dedicated to developing transformative therapies for rare diseases with serious unmet needs, today announced the DMC’s recommendation to continue the Phase 3 sunRIZE study as planned in patients with congenital HI, without an increase in the study sample size.
“We are thrilled with the DMC’s favorable recommendation, which appears to validate our initial assumptions for the design and powering of the Phase 3 sunRIZE study,” said Brian Roberts, M.D., Chief Medical Officer at Rezolute. “While we remain blinded to the partial study data and are not privy to any of the statistical outputs from the DMC, we are nevertheless encouraged by this recommendation considering past outcomes from the Phase 2 RIZE study with the same objectively measured glycemic primary endpoint, real-world observations from our Expanded Access Program, and previously announced pharmacokinetic data from the open-label arm of the sunRIZE study. With U.S. clinical sites now also enrolling patients as part of our global study, and the Company tracking to complete enrollment next month, we look forward to announcing topline results, anticipated in December of this year, and remain confident in the potential of ersodetug to be a best-in-class treatment option for people living with hyperinsulinism.”
The interim analysis of the Phase 3 sunRIZE study was performed by an unblinded DMC and was based on a pre-specified analysis of the primary study endpoint (hypoglycemia events), after approximately half of enrolled patients completed the primary assessments. The analysis was intended to evaluate for study futility or otherwise to inform a potential sample size increase, for purposes of optimizing the study power and statistical confidence in the final analysis outcomes. The Company has remained blinded throughout the interim analysis and has not seen the information made available to the DMC to inform its decision making, nor received any other information from the DMC after their meeting, other than its recommendation to continue the study as planned.
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.
About Ersodetug
Ersodetug is a fully human monoclonal antibody that binds allosterically to the insulin receptor to decrease receptor over-activation by insulin and related substances (such as IGF-2) in the setting of HI, thereby improving hypoglycemia. Because ersodetug acts downstream from the pancreas, it has the potential to be universally effective at treating hypoglycemia due to any congenital or acquired form of HI.
About Congenital Hyperinsulinism
Congenital HI is the most common cause of recurrent and persistent hypoglycemia in children. Patients with congenital HI typically present with signs or symptoms of hypoglycemia within the first month of life. These episodes can result in significant brain injury and death if not recognized and managed appropriately. Additionally, recurrent, or cumulative, hypoglycemia can lead to progressive and irreversible damage over time, including serious and devastating brain injury, seizures, neuro-developmental problems, feeding difficulties, and significant impact on patient and family quality of life. In cases of congenital HI that are unresponsive to medical management, surgical removal of the pancreas may be required. More than half of children with congenital HI require long-term medical treatment for hypoglycemia that is not addressed by available therapies.
About Rezolute, Inc.
Rezolute is a late-stage rare disease company focused on significantly improving outcomes for individuals with hypoglycemia caused by hyperinsulinism (“HI”). The Company’s antibody therapy, ersodetug, is designed to treat all forms of HI and has shown substantial benefit in clinical trials and real-world use for the treatment of congenital HI and tumor HI. For more information, visit www.rezolutebio.com.
Forward-Looking Statements
This release, like many written and oral communications presented by Rezolute and its authorized officers, may contain certain forward-looking statements regarding our prospective performance and strategies within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and are including this statement for purposes of said safe harbor provisions. Forward-looking statements, which are based on certain assumptions and describe future plans, strategies, and expectations of Rezolute, are generally identified by use of words such as "anticipate," "believe," "estimate," "expect," "intend," "plan," "project," "seek," "strive," "try," or future or conditional verbs such as "could," "may," "should," "will," "would," or similar expressions. These forward-looking statements include, but are not limited to, statements regarding the ability of ersodetug to become an effective treatment for congenital HI, statements regarding the potential regulatory approval and commercialization of ersodetug, the efficacy of the Phase 3 sunRIZE study, statements related to the Phase 3 sunRIZE study sample size, and statements regarding the timing of the completion of enrollment and release of topline data. Our ability to predict results or the actual effects of our plans or strategies is inherently uncertain. Accordingly, actual results may differ materially from anticipated results. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release. Except as required by applicable law or regulation, Rezolute undertakes no obligation to update these forward-looking statements to reflect events or circumstances that occur after the date on which such statements were made. Important factors that may cause such a difference include any other factors discussed in our filings with the SEC, including the Risk Factors contained in Rezolute’s Annual Report on Form 10-K and Quarterly Reports on Form 10-Q, which are available on the SEC’s website at www.sec.gov. You are urged to consider these factors carefully by evaluating the forward-looking statements in this release and are cautioned not to place undue reliance on such forward-looking statements, which are qualified in their entirety by this cautionary statement.
Contacts:
Investors
Christen Baglaneas
cbaglaneas@rezolutebio.com
508-272-6717
Media
Sarah Lima
Sarah@GalvinPR.com
(774) 766-0200
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