Rezolute, Inc. (Nasdaq: RZLT), a
clinical-stage biopharmaceutical company committed to
developing novel, transformative therapies for serious
metabolic and rare diseases, today announced the initiation of
sunRIZE, a pivotal Phase 3 clinical study of RZ358 in patients with
congenital hyperinsulinism (cHI). Following country-level
regulatory and ethics committee approvals, the first clinical site
has now been activated outside the US, allowing for patient
screening and enrollment to commence. Additional sites are being
activated on a regular basis over the coming weeks and into the
beginning of 2024.
The company's progression into Phase 3 was
preceded and enabled by a successful Phase 2 trial (the RIZE
study), which demonstrated promising results in the treatment of
cHI, a serious condition with a tremendous burden on patients and
families and a substantial unmet medical need. The current
announcement of the Phase 3 start follows the receipt of a priority
medicines (PRIME) designation of RZ358 for the treatment of cHI
from the European Medicines Agency in October 2023.
“The start of the Phase 3 study marks a
significant milestone in the development of RZ358 and exemplifies
the tremendous progress of our Company as a whole. The unmet
medical need for patients and their families living with cHI is
unequivocal, particularly given that the only approved therapy,
diazoxide, is unable to work in more than half of patients, or
alternatively is associated with significant side effects in those
that respond,” remarked Nevan Charles Elam, Chief Executive Officer
and Founder of Rezolute. “Better therapies are desperately needed,
and we look forward to working closely with sites throughout the
world to advance patient screening and enrollment in this pivotal
study, so as to move one step closer to realizing that goal.”
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 RZ358 in patients
with cHI who are experiencing poorly controlled hypoglycemia.
Participants between the ages of 3 months to 45 years old are
eligible to participate. The study will enroll up to 56
participants and be conducted at approximately 20 expert centers in
more than a dozen countries around the world, many of which also
participated in the Phase 2 RIZE study.
In the main comparator-controlled portion of the
study, 48 participants in the age group of 1 year and older will be
randomized (2:1) to receive RZ358 at doses of either 5mg/kg (n=16)
or 10mg/kg (n=16), or matched placebo (n=16), in double-blind
fashion and as add-on to standard of care. In parallel, an
additional open-label arm will be conducted in approximately 8
additional participants in the age group of 3 months to 1 year old
at a starting dose of 5mg/kg dose, which may be increased to 10
mg/kg as needed. All participants will receive study drug bi-weekly
during a 6-week loading period, followed by every 4 weeks during
the remainder of a 24-week total pivotal treatment period.
The primary efficacy endpoint over the 24-week
pivotal period will be the change in average weekly hypoglycemia
events (<70 mg/dL), as measured by point-of-care blood glucose.
The key secondary endpoint is the change in average daily percent
time in hypoglycemia (<70 mg/dL), as measured by continuous
glucose monitor (CGM). Additional secondary endpoints to be
evaluated include serious/symptomatic hypoglycemia events and time,
hypoglycemia-related hospitalizations, and quality of life outcomes
related to hypoglycemia. Following the end of the pivotal treatment
period, participants may have the option to enter the open-label
extension portion of the study.
About Congenital HI 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. In those with diffuse congenital HI where
the whole pancreas is affected, a near-total pancreatectomy can be
undertaken, although about half of these children will continue to
have hypoglycemia and require medical treatment for congenital
HI.
About RZ358RZ358 is a fully
human monoclonal antibody that works downstream from the pancreas
and instead binds to a unique allosteric site on insulin receptors
in the liver, fat, and muscle. The antibody counteracts the effects
of excess insulin binding and activity, thereby correcting
hypoglycemia. Rezolute believes that RZ358 is ideally suited as a
potential therapy for congenital HI and other conditions
characterized by excessive insulin activity (hyperinsulinism).
Because RZ358 acts downstream from the pancreas, it has the
potential to be universally effective at treating congenital HI,
regardless of the causative genetic defect, as well as acquired
forms of HI such as those mediated by insulinomas and other tumor
types. RZ358 received Orphan Drug Designation in the United States
and European Union for the treatment of congenital HI, as well as
Pediatric Rare Disease Designation in the US. In the Phase 2 RIZE
study, participants with cHI ages 2 and older nearly universally
achieved significant improvements in hypoglycemia across multiple
endpoints, including the primary and key secondary endpoints
planned for the sunRIZE study. At doses and exposures that are
planned for the Phase 3 study, RZ358 was generally safe and
well-tolerated, and resulted in median improvements in hypoglycemia
exceeding 80%. Based on the RIZE clinical trial outcomes and the
evidence of benefit in this serious condition with substantial
unmet medical need, RZ358 was subsequently granted a priority
medicines (PRIME) designation for the treatment of cHI by the
European Medicines Agency
About Rezolute, Inc.Rezolute
strives to disrupt current treatment paradigms by developing
transformative therapies for devastating rare and chronic metabolic
diseases. Its novel therapies hold the potential to both
significantly improve outcomes and reduce the treatment burden for
patients, treating physicians, and the healthcare system. Rezolute
is steadfast in its mission to create profound, positive, and
lasting impacts on patients’ lives. Patient, clinician, and
advocate voices are integrated in the Company’s drug development
process. Rezolute places an emphasis on understanding the patient’s
lived experiences, enabling the Company to boldly address a range
of severe conditions. For more information,
visit www.rezolutebio.com.
Forward-Looking StatementsThis
release, like many written and oral communications presented by
Rezolute and our authorized officers, may contain certain
forward-looking statements regarding our prospective performance
and strategies within the meaning of Section 27A of the Securities
Act 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 and statements regarding PRIME designation and the
designation’s meaning on the ability of RZ358 to become an
effective treatment to congenital HI, the effectiveness or future
effectiveness of RZ358 for the treatment of congenital HI, and
statements regarding clinical trial timelines for RZ358. 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 the Rezolute’s Annual
Report on Form 10-K and Quarterly Reports on Form 10-Q, which are
available at the SEC’s website at www.sec.gov. You are urged to
consider these factors carefully in 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.
Investors:Stephanie CarringtonICR
WestwickeRezoluteIR@westwicke.com(646)277-1282
Media:media-relations@rezolutebio.com
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