Full Press Release Details
Rezolute Receives Breakthrough Therapy Designation
from FDA for Ersodetug in the Treatment of Hypoglycemia Due to Congenital Hyperinsulinism
Breakthrough Therapy Designation granted based
on key positive data from the Phase 2b (RIZE) study and current unmet medical need in congenital hyperinsulinism (HI)
Ersodetug continues to advance in clinical development
as a potential treatment for hypoglycemia caused by all forms of hyperinsulinism; topline sunRIZE data expected second half of this year
REDWOOD CITY, Calif., January 07, 2025 - Rezolute, Inc.
(Nasdaq: RZLT) ("Rezolute" or the "Company"), a late-stage biopharmaceutical company dedicated to developing
transformative therapies for rare diseases with serious unmet needs, today announced that the U.S. Food and Drug Administration (FDA)
has granted Breakthrough Therapy Designation to ersodetug (RZ358) for the treatment of hypoglycemia due to congenital HI.
Breakthrough Therapy Designation for ersodetug
is designed to expedite the development and regulatory review of investigational treatments for serious or life-threatening conditions
that, based on clinical evidence, have the potential to substantially improve clinical outcomes compared with available therapies. The
Breakthrough Therapy Designation is based primarily on results from the Phase 2b (RIZE) study of ersodetug in participants with congenital
HI, which safely demonstrated significant improvements in hypoglycemia of 75% or better, with no clinically significant hyperglycemia.
"2024 was a transformative year for Rezolute, marked by important
clinical milestones and solid progress in advancing our mission to address hyperinsulinism in all forms," said Nevan Charles Elam,
Chief Executive Officer and Founder of Rezolute. "This Breakthrough Therapy Designation underscores the potential of ersodetug to
make a meaningful difference for patients with hyperinsulinism. This year we are focused on finishing recruitment for sunRIZE and announcing
topline results, as well as commencing our Phase 3 study for tumor HI."
Key highlights from 2024 include:
Clinical progress across two Phase 3 programs in two indications:
Strengthened financial position to advance pipeline and corporate
About Congenital Hyperinsulinism
Congenital hyperinsulinism
(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.
Ersodetug is a fully human monoclonal antibody
that binds allosterically to the insulin receptor to counteract the effects of insulin receptor over-activation by insulin and related
substances (such as IGF-2), thereby shifting over-signaling back into a more normalized range and improving hypoglycemia in the setting
of hyperinsulinism (HI). 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 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 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," "potential," 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 FDA's grant of the Breakthrough Therapy Designation, the ersodetug Expanded Access Program, the ability of ersodetug
to become an effective treatment for congenital hyperinsulinism, the effectiveness or future effectiveness of ersodetug for the treatment
of congenital hyperinsulinism, statements regarding clinical trial timelines for ersodetug, the timing of the Phase 3 sunRIZE study,
and the FDA's Orphan Drug Designation as it relates to ersodetug. 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
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.