Full Press Release Details
FDA Clearance of IND Application for Phase 3 Registrational Study of RZ358 for Treatment of Hypoglycemia Due to Tumor Hyperinsulinism
Second rare disease program with RZ358 in Phase
Follows successful treatment of multiple patients
with tumor hyperinsulinism
under the Company's Expanded Access Program
REDWOOD CITY, Calif., August 5, 2024 -
Rezolute, Inc. (Nasdaq: RZLT) ("Rezolute" or the "Company"), a late-stage biopharmaceutical company
committed to developing novel, transformative therapies for serious rare diseases, today announced that it received U.S. Food and Drug
Administration (FDA) clearance for its Investigational New Drug (IND) application for RZ358 (ersodetug) to treat hypoglycemia in patients
with tumor hyperinsulinism (HI). The Company is initiating start-up activities for the study which will be primarily conducted in the
U.S. and patient enrollment is planned to commence in the first half of 2025. Ersodetug is also being studied in an ongoing global, pivotal,
Phase 3 clinical trial in patients with congenital HI. Topline data from that study is expected in mid-2025.
"Hypoglycemia associated with tumor HI requires
treatment to prevent serious adverse outcomes and to improve patients' daily function and quality of life, including enabling them
to receive tumor directed therapies," said Brian Roberts, M.D., Chief Medical Officer at Rezolute. "We are encouraged by the
substantial real-world benefit we've witnessed in tumor HI patients who have previously received ersodetug in our Expanded Access
Program, coupled with the safety and efficacy demonstrated in clinical studies in patients with congenital HI, a similar condition. We
believe that the clearance of our IND for this Phase 3 study reflects FDA's recognition of the potential for ersodetug to address
this serious unmet need and we are excited to be moving one step closer to a potential universal treatment for hypoglycemia caused by
The Phase 3 registrational study is a double-blind,
randomized, placebo-controlled trial of 24 participants who have inadequately controlled hypoglycemia because of tumor HI. Eligible participants
will be randomized in 1:1 fashion (12 per treatment arm) to receive ersodetug 9 mg/kg per week or matched placebo, as an add-on to standard
of care. Up to 24 additional participants may be enrolled into an open-label arm, in participants whose hypoglycemia is being managed
by IV glucose in a hospital setting. Following a 6-week pivotal treatment period, all participants may receive ersodetug in open-label
extension. The primary endpoint is the change in Level 2 (moderate) and Level 3 (severe) hypoglycemia events by self-monitored blood glucose.
Additional endpoints include overall hypoglycemia events, time in hypoglycemia by continuous glucose monitor, patient reported quality
of life, hospitalizations, and change in glucose requirements (for open-label hospitalized participants).
Ersodetug is a fully human monoclonal antibody
that binds to an allosteric site on the insulin receptor at target tissues such as liver, fat and muscle. Ersodetug counteracts excess
insulin receptor activation caused by insulin and related hormones thereby correcting hypoglycemia. Ersodetug has the potential to be
universally effective at treating hypoglycemia caused by any form of HI, including congenital or acquired forms.
About Tumor Hyperinsulinism (HI)
Tumor HI is a rare disease that 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 as a result
of excessive activation of the insulin receptor. Insulinomas are the most common type of ICT and may cause hypoglycemia by stimulating
the over production of insulin. A variety of different NICTs, particularly hepatocellular carcinoma, can cause hypoglycemia by producing
and secreting insulin-like paraneoplastic substances such as IGF-2 that bind to and activate the insulin receptor. With high morbidity
and mortality rates within tumor HI, there remains a significant unmet need for new therapies directed at hypoglycemia treatment. Ersodetug
has shown real-world benefit in patients with insulinoma and preclinical studies have shown that ersodetug can similarly blunt IGF-2 and
insulin-mediated insulin-receptor signaling.
About Rezolute, Inc.
Rezolute is a late-stage rare disease company
focused on significantly improving outcomes for individuals with hypoglycemia caused by 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.
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 the RZ358 Expanded Access Program, the Investigational
New Drug (IND) application for RZ358, the ability of the U.S. Ersodetug to become an effective treatment for congenital hyperinsulinism,
the effectiveness or future effectiveness of the U.S. Ersodetug to become an effective treatment for congenital hyperinsulinism, and statements
regarding clinical trial timelines for RZ358. 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, the potential completion of
the public offering, satisfaction of customary closing conditions related to the public offering and other factors discussed in the "Risk
Factors" section contained in the preliminary prospectus supplement and 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.
LHA Investor Relations