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FDA Lifts Partial Clinical Holds on RZ358 for the Treatment of Congenital Hyperinsulinism and Authorizes U.S. Inclusion in Ongoing Phase 3 Study Commencing study startup activities in the U.S.; participant enrollment ant

Key Takeaway: Rezolute, Inc. announced that the FDA has lifted partial clinical holds on its treatment RZ358 for congenital hyperinsulinism, enabling the company to proceed with study activities in the U.S. The global Phase 3 study, called sunRIZE, will include U.S. participants starting in early 2025, with topline data expected in the latter half of that year. The lift follows the FDA's conclusion that previously observed liver toxicity in animal studies is strain-specific and not relevant to human subjects. This development is expected to advance Rezolute's dual Phase 3 programs for this rare disease.

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POSITIVE FACTORS

  • FDA lifted the clinical holds on RZ358, allowing progress in trials.
  • Study start-up activities in the U.S. are set to commence, expanding participation.
  • Phase 3 study aims to improve treatment for congenital hyperinsulinism.

CONCERNS & RISKS

  • Potential liver toxicity observed in animal studies, although likely not relevant to humans.
  • Future developments depend on regulatory approvals and clinical trial outcomes.

Full Press Release Details

Clinical Holds on RZ358 for the Treatment of Congenital Hyperinsulinism and Authorizes U.S. Inclusion in Ongoing Phase 3 Study
startup activities in the U.S.; participant enrollment anticipated in early 2025
CITY, Calif., September 9, 2024 - 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 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.
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
For more information
on ersodetug and our programs in congenital HI and tumor HI, please visit https://www.rezolutebio.com/pipeline/.
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
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.
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 the 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.

Frequently Asked Questions

What is RZ358 used to treat?

RZ358, also known as ersodetug, is designed to treat hypoglycemia caused by congenital hyperinsulinism.

When will U.S. enrollment for the study begin?

Participant enrollment in the U.S. for the study is anticipated to start in early 2025.

What does the FDA's removal of clinical holds mean?

The FDA has lifted the partial clinical holds, allowing progression of RZ358 trials at all doses.

What age group is eligible for the trial?

Participants aged from 3 months to 45 years can enroll in the Phase 3 study.

How many participants will be involved in the study?

The study aims to enroll up to 56 participants across more than a dozen countries.

Last updated: Sep 9, 2024