Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic
biopharmaceutical company focused on developing and commercializing
therapies for the treatment of several eye disorders, announced
today that it has screened the first patient in ZETA-1, a Phase 2
trial to evaluate APX3330 in non-proliferative diabetic retinopathy
(NPDR) and mild proliferative diabetic retinopathy (mild PDR).
Effects on diabetic macular edema will be explored as a secondary
outcome. A number of retinal centers across the US are active and
recruiting eligible diabetic retinopathy patients.
Diabetes is the leading cause of blindness among
adults aged 20 – 74. In the United States alone, over 7 million
patients suffer from diabetic retinopathy (DR), a complication of
diabetes in which chronically elevated blood sugar levels cause
damage to blood vessels in the retina. An additional 750,000
patients suffer from diabetic macular edema (DME), one of the most
common complications of diabetic retinopathy where the macula
swells from fluid leaked from damaged blood vessels. The disease
progression of both DR and DME involves abnormal vessel
proliferation and inflammation. Thus, current approved treatments
for DR and DME encompass an over $10 billion global market and
involve administering anti-VEGF injections (such as EYLEA® by
Regeneron, Lucentis® by Genentech, and Avastin® by Genentech) to
decrease vessel formation or steroids (such as OZURDEX® by
Allergan) to decrease inflammation into eyes with advanced retinal
disease. ZETA-1 is investigating the potential of APX3330 to offer
an innovative and conveniently administered oral treatment for
diabetic retinopathy that addresses both of these disease
pathways.
Dr. Peter K. Kaiser, Professor of Ophthalmology
at the Cole Eye Institute of the Cleveland Clinic Foundation
commented, “There remains a strong need to develop a non-injectable
alternative treatment option for patients with DR as these
injectables—although approved for this indication— are not widely
used. If successfully developed, APX3330 could lead to the first
oral option for DR as well as an adjunct therapy that may improve
dosing convenience and compliance by alleviating some of the burden
of chronic anti-VEGF injection treatments for DME and other retinal
diseases.”
APX3330 is a small molecule oral drug candidate
and a first-in-class inhibitor of the transcription factor
regulator Ref-1 (reduction-oxidation effector factor-1). With its
novel mechanism of action, APX3330 blocks the downstream pathways
regulated by Ref-1, including those involving angiogenesis (VEGF)
and inflammation (NF-kB), to decrease abnormal activation of both
angiogenesis and inflammatory pathways that are implicated across
several ocular diseases, including diabetic retinopathy (DR),
diabetic macular edema (DME), and age-related macular degeneration
(AMD).
Dr. Mark R. Kelley, Professor in the Department
of Pediatrics and Glick Eye Center at Indiana University School of
Medicine, co-founder of the APX3330 program, and member of
Ocuphire’s Medical Advisory Board stated, “APX3330, a potential
first oral therapy for DR, is not only novel in its oral route of
administration, but it builds on decades of studies targeting Ref-1
as an impactful way to block both angiogenesis and inflammation
using a single drug candidate. It is rewarding to see APX3330 begin
this Phase 2 trial in ophthalmology with the potential to offer a
new treatment option for patients with retinal diseases,
particularly diabetics.”
The ZETA-1 trial is a randomized,
placebo-controlled, double-masked study designed to evaluate the
efficacy of APX3330 to improve diabetic retinopathy over 24 weeks.
The study will be conducted in up to 20 U.S. sites and is expected
to enroll approximately 100 subjects with moderately-severe to
severe NPDR or mild PDR in the study eye. If patients who are
enrolled also have DME in their non-study eye, this eye will also
be followed during the trial for potential improvement. The primary
endpoint of the study will evaluate the percentage of subjects with
a ≥ 2 step improvement on the Diabetic Retinopathy Severity Scale
(DRSS) score. Secondary endpoints include evaluation of central
subfield thickness to assess effects on diabetic macular edema,
BCVA, safety and tolerability. For more information, refer to
www.ClinicalTrials.gov Identifier: NCT04692688.
Mina Sooch, MBA, President and CEO of Ocuphire
Pharma commented, “We are very excited to advance APX3330 in the
ZETA-1 Phase 2 clinical trial. Building off of 11 prior trials that
have demonstrated a favorable safety and tolerability profile in
over 300 oncology and hepatic patients, APX3330 has the potential
to become the first oral therapy used for diabetic retinopathy. Due
to its highly differentiated mechanism of action, we believe that
APX3330 could also emerge as an important add-on therapy with the
currently approved anti-VEGF treatments and extend the time between
injections. The team at Ocuphire has now initiated all 4 clinical
trials planned since its public listing last November, and we look
forward to continuing enrollment and data readouts over the next 12
months.”
About Diabetic Retinopathy
Diabetes, a worldwide epidemic, is the leading
cause of blindness among adults age 20 to 74. DR is the most common
diabetic complication that affects the eyes and is manifested when
chronically elevated blood sugar levels cause damage to blood
vessels in the retina. DR affects over 7 million patients in the
U.S. and 93 million patients worldwide. This problem is projected
to worsen as the number of individuals at risk of developing
diabetes increases by 55% by 2035 to a worldwide total of 592
million people.
There are two major types of DR: (1)
non-proliferative DR (NPDR) and (2) proliferative DR (PDR). NPDR is
an earlier, more typical stage of DR that can progress to more
severe forms of DR if untreated and if the underlying diabetes
remains uncontrolled. PDR is a more advanced stage of DR that is
characterized by retinal neovascularization that, if left
untreated, can lead to permanent damage and blindness. When DR is
in its early stages, blood vessels in the retina are damaged and
can leak fluid into the retina, a complication called diabetic
macular edema (DME). Fluid from DME and hemorrhage of the abnormal
blood vessels formed in PDR, can interfere with vision and can
cause irreversible visual impairment due to retinal scarring and
retinal detachment. Despite the approval of intravitreal injection
therapies for DR, patients with DR are not widely treated.
About Ocuphire Pharma
Ocuphire is a publicly traded (NASDAQ: OCUP), clinical-stage
ophthalmic biopharmaceutical company focused on developing and
commercializing therapies for the treatment of several eye
disorders. Ocuphire’s pipeline currently includes two
small-molecule product candidates targeting front and back of the
eye indications. The company’s lead product candidate,
Nyxol® (0.75% phentolamine ophthalmic solution) Eye Drops, is
a once-daily preservative-free eye drop formulation of phentolamine
mesylate, a non-selective alpha-1 and alpha-2 adrenergic antagonist
designed to reduce pupil size, and is being developed for several
indications, including dim light or night vision disturbances
(NVD), reversal of pharmacologically-induced mydriasis (RM), and
presbyopia, and has been studied in 8 clinical trials including the
recently completed Phase 3 trial in RM. Ocuphire reported positive
topline data on March 15, 2021 for MIRA-2 Phase 3 FDA registration
study for treatment of RM. Nyxol is also currently in Phase 3
clinical development for NVD and in Phase 2 for presbyopia.
Ocuphire’s second product candidate, APX3330, is an oral tablet
designed to inhibit angiogenesis and inflammation pathways relevant
to retinal and choroidal vascular diseases, such as diabetic
retinopathy (DR) and diabetic macular edema (DME) and has been
studied in 11 Phase 1 and 2 trials. APX3330 is entering Phase 2
clinical development for DR/DME. As part of its strategy, Ocuphire
will continue to explore opportunities to acquire additional
ophthalmic assets and to seek strategic partners for late-stage
development, regulatory preparation and commercialization of drugs
in key global markets. Please
visit www.clinicaltrials.gov to learn more about
Ocuphire’s completed Phase 2 trials, recently completed Phase 3
registration trial (NCT04620213), ongoing Phase 3 registration
trial (NCT04638660) and Phase 2 trial in presbyopia
(NCT04675151), and Phase 2 trial in DR/DME (NCT04692688). For more
information, please visit www.ocuphire.com.
Forward Looking Statements
Statements contained in this press release regarding matters
that are not historical facts are “forward-looking statements”
within the meaning of the Private Securities Litigation Reform Act
of 1995. Such statements include, but are not limited to,
statements concerning Ocuphire’s product candidates, results
of ongoing and future clinical trials, and commercialization and
market opportunities. These forward-looking statements are based
upon Ocuphire’s current expectations and involve assumptions that
may never materialize or may prove to be incorrect. Actual results
and the timing of events could differ materially from those
anticipated in such forward-looking statements as a result of
various risks and uncertainties, including, without limitation: (i)
the success and timing of regulatory submissions and pre-clinical
and clinical trials, including enrollment and data readouts; (ii)
regulatory requirements or developments; (iii) changes to clinical
trial designs and regulatory pathways; (iv) changes in capital
resource requirements; (v) risks related to the inability of
Ocuphire to obtain sufficient additional capital to continue to
advance its product candidates and its preclinical programs; (vi)
legislative, regulatory, political and economic
developments, (vii) changes in market
opportunities, (viii) the effects of COVID-19 on clinical
programs and business operations, and (ix) the success and timing
of commercialization of any of Ocuphire’s product candidates. The
foregoing review of important factors that could cause actual
events to differ from expectations should not be construed as
exhaustive and should be read in conjunction with statements that
are included herein and elsewhere, including the risk factors
detailed in documents that have been and may be filed by Ocuphire
from time to time with the SEC. All forward-looking statements
contained in this press release speak only as of the date on which
they were made. Ocuphire undertakes no obligation to update such
statements to reflect events that occur or circumstances that exist
after the date on which they were made.
Ocuphire Contacts
Mina Sooch, President & CEO Ocuphire Pharma,
Inc. ir@ocuphire.com www.ocuphire.com
Corey Davis, Ph.D.LifeSci
Advisorscdavis@lifescieadvisors.com
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