Full Press Release Details
Rezolute Reports Positive Topline Results from
Phase 2 Proof of Concept Study of RZ402 in Patients with Diabetic Macular Edema (DME)
primary study endpoints: good safety profile and reduction in central subfield thickness (CST)
oral therapy to demonstrate reduction in macular edema; supports potential for early disease intervention
investor event to be held today at 5:30pm ET
CITY, Calif., May 21, 2024 - Rezolute, Inc. (Nasdaq: RZLT) ("Rezolute" or the "Company"),
a clinical-stage biopharmaceutical company committed to developing novel, transformative therapies for serious metabolic and
rare diseases, today announced positive topline results from the Phase 2 clinical study of RZ402 in patients with DME who are na ve
to or have received limited anti-vascular growth factor (anti-VEGF) injections.
are monumental for the DME community," said Quan Dong Nguyen, MD, MSc, FAAO, FARVO, FASRS, Professor of Ophthalmology at the Byers
Eye Institute, and Professor of Medicine and Professor of Pediatrics at the Stanford University School of Medicine, and a member of Rezolute's
Scientific Advisory Board. "I am impressed by the significant reduction in CST in this study across all three dosages as retinal
thickness is the key biomarker to determine whether a therapy may offer a potential benefit to patients. These data are very encouraging
and are supportive of the potential for a new first-line, non-invasive treatment for DME."
94 participants were enrolled in the U.S. multi-center,
randomized, double-masked, placebo-controlled, parallel-arm study to evaluate the safety, efficacy, and pharmacokinetics of RZ402 administered
as a monotherapy over a 12-week (three month) treatment period.
"I am encouraged to see the initial results of this proof-of-concept
study and am enthusiastic about the possibility of an oral therapy to treat DME," said Arshad Khanani, MD, MA, FASRS, Clinical
Professor at the Reno School of Medicine, University of Nevada, and Primary Investigator of the study. "Importantly, an oral therapy
would afford us the opportunity to address both eyes and intervene much earlier, potentially altering the long-term prognosis for individuals
"We are very excited about these results and, importantly, would
like to thank the patients, the leading retina specialists across the country, and their motivated staff who all made this study possible,"
said Raj Agrawal, MD, Vice President and Head of Ophthalmological Clinical Development at Rezolute.
Study design and eligibility criteria
| Patients must have received no more than three anti-VEGF injections previously (none within eight weeks of randomization) | ||
| CST of 320 microns in males and 305 microns in females | ||
| Best Corrected Visual Acuity (BCVA) of 78 letters on Early Treatment Diabetic Retinopathy Study (ETDRS) assessment | ||
| Eligible participants were randomized equally, to one of three RZ402 active treatment arms at doses of 50, 200, and 400 mg, or a placebo control arm, to receive study drug once daily for 12 weeks, before completing a four-week follow-up |
RZ402 met both primary endpoints of change
in macular edema (CST) and a good safety profile
| o | Continued downward trajectory in CST over course of study and at end of treatment | |
| o | No significant difference between RZ402 dose levels, though response was largest at the 200 mg dose | |
| o | Sub-analysis by DME severity (CST 400 microns) indicates an improvement of approximately 75 microns at the 200 mg dose | |
| o | CST declined in most patients who received the 200 mg dose of RZ402, including clinically significant improvements from baseline in more than 20% of participants, compared to none in placebo, with high rates of worsening |
| o | Adverse events (AEs) were generally mild and rates were comparable to placebo | |
| o | Three participants experienced serious AEs which were all judged by the Investigator as unrelated to study drug | |
| o | No ocular adverse effects that are typically seen with intravitreal injections | |
| o | Electrocardiograms (ECGs), vitals, and safety labs were unremarkable |
Secondary and additional endpoints
The Company plans to present these data and further
findings at an upcoming medical conference.
Virtual Investor Event
The Company will host a virtual investor event
to review these topline results today at 5:30pm ET. The event will be webcast live and a replay of the webcast will be archived
in the Events section of the Company's investor relations website following the event.
DME is a severe, systemic, vision-threatening
complication of diabetic retinopathy (DR) characterized by swelling of the retina and thickening of the macula, the part of the eye that
is responsible for high-resolution vision. Anti-VEGF injections into the eye are the current standard of care for DME, requiring continued
administration over long periods of time to preserve vision. Due to their invasive route of administration and occasional serious side
effects, there is a tendency to delay treatment until later in the disease course, and long-term compliance with eye injection regimens
can be difficult for patients. Coupled with inadequate responsiveness in some patients, this leads to overall undertreatment and suboptimal
vision outcomes in DME patients. DME is a result of a systemic microvascular complication of diabetes, manifesting in the retinal blood
vessels behind the eyes, and therefore commonly affects both eyes. With currently available intravitreal anti-VEGF therapies, two separate
eye injections are required, or treatment of one eye or both eyes may be deferred, when both eyes are affected.
RZ402 is an oral, small molecule, selective and potent, plasma kallikrein inhibitor (PKI), for the chronic treatment of DME.
By inhibiting the activation of kallikrein, RZ402 is designed to block bradykinin production and its resulting effects on vascular leakage
and inflammation. Topline results from the Phase 2 study of RZ402 in patients with DME demonstrated a significant reduction in central
subfield thickness (CST) in the Study Eye at all RZ402 dose levels compared to placebo (up to approximately 50 micron improvement) and
was safe and well-tolerated.
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 significantly improve outcomes and to reduce the treatment burden for patients, treating physicians and the healthcare system. Rezolute
is steadfast in its mission to create a profound, positive and lasting impact on the lives of patients. 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 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," "prove," "potential," "seek," "strive,"
"try," or future or conditional verbs such as "predict," "could," "may," "likely,"
"should," "will," "would," or similar expressions. These forward-looking statements include, but are not
limited to statements regarding the RZ402 study, the ability of RZ402 to become an effective treatment for diabetic macular edema, the
effectiveness or future effectiveness of RZ402 to become an effective treatment for diabetic macular edema, and statements regarding
clinical trial timelines for RZ402. 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.
LHA Investor Relations