- ADX-2191 Statistically Superior to Historical Control for
Primary Endpoint of Prevention of Retinal Detachment (P=0.024)
- Numerical Superiority of ADX-2191 over Routine Surgical Care
Achieved for Majority of Secondary, Exploratory, and Safety
Endpoints
- ADX-2191 Observed to be Well Tolerated with No Safety Concerns
Noted
- Company Plans to Discuss Completion of Clinical Development in
Proliferative Vitreoretinopathy with the U.S. Food and Drug
Administration in the First Half of 2023
- Company to Host Conference Call at 8:00 a.m. ET Today
Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra or the
Company) today announced the achievement of the primary endpoint in
Part 1 of the Phase 3 GUARD Trial of ADX-2191 (methotrexate
injection, USP1) for intravitreal administration, an
investigational drug candidate, for the prevention of proliferative
vitreoretinopathy (PVR), a rare sight-threatening retinal disease
with no approved therapy. ADX-2191 was statistically superior to
historical control2 for the prevention of retinal detachment due to
PVR over six months (P=0.024).
“Proliferative vitreoretinopathy represents a major unmet
medical need and is particularly difficult to treat, highlighting
the need for an effective therapy,” stated Marco Zarbin, M.D.,
Ph.D., Professor and Chair of the Institute of Ophthalmology and
Visual Science, Rutgers New Jersey Medical School. “The recent
reports describing the activity of methotrexate in preventing PVR,
in conjunction with the results of the GUARD Trial, offer hope to
many patients and physicians that today have few options for
treatment.”
Part 1 of the GUARD Trial was designed to assess the preliminary
activity of ADX-2191, a novel vitreous-compatible formulation of
methotrexate, versus historical control and routine surgical care
without therapy in patients with PVR. Sixty-eight patients received
ADX-2191, and 38 patients received routine surgical care. Relative
to historical control, statistically significant reduction
(P=0.024) in retinal detachment over six months was observed
following serial intravitreal injection of ADX-2191. Although not
statistically powered for secondary or exploratory endpoints, the
results of the GUARD Trial demonstrated numerical superiority of
ADX-2191 over routine surgical care in reducing the dichotomous
endpoints of retinal detachment rate over six months, hypotony (low
intraocular pressure), complete retinal attachment by six months,
macular attachment by six months, and epiretinal membrane formation
(overall P=0.047). Visual acuity was similar between ADX-2191
treatment and routine surgical care groups. Central macular
thickness was numerically lower in ADX-2191-treated patients.
No safety signals were observed in the trial, and ADX-2191 was
well tolerated; there were no observed treatment-emergent serious
adverse events. The most common adverse event associated with
ADX-2191 treatment was punctate keratitis, a well-known side effect
of intravitreal methotrexate, that was most commonly mild in
severity. Across all other treatment-emergent adverse events
occurring in at least 10% of patients in either treatment arm,
relative to patients treated with routine surgical care,
ADX-2191-treated patients had numerically fewer side effects,
including pain, cystoid macular edema, corneal edema, macular
fibrosis, corneal epithelial defects, anterior uveitis, ocular
hypertension, and post-operative inflammation (overall P=0.0002).
In the ADX-2191 group, there was one discontinuation, which was due
to scheduling difficulties.
Aldeyra intends to discuss completion of clinical development of
ADX-2191 for the prevention of PVR in a Type C meeting with the
U.S. Food and Drug Administration (FDA) in the first half of 2023.
ADX-2191 has received FDA Orphan Drug Designation and FDA Fast
Track Designation for the prevention of PVR, and EU Orphan
Medicinal Product Designation for the treatment of retinal
detachment. ADX-2191 has also received FDA Orphan Drug Designation
for the treatment of primary vitreoretinal lymphoma and retinitis
pigmentosa.
“The ADX-2191 platform for the prevention and treatment of rare
retinal disease continues to advance towards commercialization, and
is a critical late-stage pipeline program with the potential to
address a number of diseases with no FDA-approved therapies,”
stated Todd C. Brady, M.D., Ph.D., President and Chief Executive
Officer of Aldeyra.
Conference Call Information
Aldeyra will host a conference call to discuss this announcement
at 8:00 a.m. ET today, October 6, 2022. The dial-in numbers are
(844) 200-6205 for domestic callers and (646) 904-5544 for
international callers. The access code is 005042. A live webcast of
the conference call will also be available on the “Investors &
Media” section of the Aldeyra website at https://ir.aldeyra.com.
Presentation slides, which contain material information and should
be reviewed in conjunction with this press release, will be
available on the investor relations page prior to the start of the
conference call and webcast.
After the live webcast, the event will remain archived on the
Aldeyra website for 90 days.
About ADX-2191
ADX-2191 (methotrexate injection, USP) is a sterile,
non-compounded intravitreal formulation of methotrexate for the
potential prevention or treatment of specific rare retinal
diseases, including primary vitreoretinal lymphoma, proliferative
vitreoretinopathy, and retinitis pigmentosa. The ADX-2191
intravitreal formulation is preservative-free, designed to be
vitreous-compatible, and optimized for excipient composition,
viscosity, density, tonicity, pH, concentration, and volume of
administration. ADX-2191 has received FDA Orphan Drug Designation
for the prevention of proliferative vitreoretinopathy, and the
treatment of primary vitreoretinal lymphoma and retinitis
pigmentosa.
About Proliferative Vitreoretinopathy
Proliferative vitreoretinopathy (PVR) is a rare inflammatory
fibroproliferative disorder that leads to severe retinal scarring
and blindness, and is the leading cause of failure of retinal
reattachment surgery. Left untreated, retinal detachment due to PVR
can progress to permanent blindness. PVR affects approximately
4,000 patients per year in the U.S. There is currently no approved
therapy for the treatment of PVR.
About Aldeyra
Aldeyra is a clinical-stage biotechnology company developing
innovative therapies designed to treat immune-mediated diseases.
Our approach is to discover pharmaceuticals that modulate
immunological systems, instead of directly inhibiting or activating
single protein targets, with the goal of optimizing multiple
pathways at once while minimizing toxicity. Two of our lead product
candidates, reproxalap and ADX-629, target pre-cytokine,
systems-based mediators of inflammation known as RASP (reactive
aldehyde species). Reproxalap is in late-stage clinical trials in
patients with dry eye disease and allergic conjunctivitis. ADX-629,
an orally administered RASP modulator, is in Phase 2 clinical
testing for the treatment of systemic immune-mediated diseases. Our
pipeline also includes ADX-2191 (methotrexate injection) for
intravitreal administration, in development for the prevention of
proliferative vitreoretinopathy and the treatment of retinitis
pigmentosa and primary vitreoretinal lymphoma. For more
information, visit https://www.aldeyra.com and follow us on
LinkedIn, Facebook, and Twitter.
Safe Harbor Statement
This release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995,
including, but not limited to, statements regarding Aldeyra’s
future expectations, plans, and prospects, including without
limitation statements regarding: the potential regulatory approval
of ADX-2191 and Aldeyra’s goals as to timing; the potential profile
and benefit of ADX-2191; and other statements regarding the goals,
opportunity and potential for ADX-2191, anticipated clinical or
regulatory milestones for ADX-2191, including expectations
regarding timing and results of meetings with the FDA, including
scheduled Type C and pre-NDA meetings, and submissions to the FDA.
Aldeyra intends such forward-looking statements to be covered by
the safe harbor provisions for forward-looking statements contained
in Section 21E of the Securities Exchange Act of 1934 and the
Private Securities Litigation Reform Act of 1995. In some cases,
you can identify forward-looking statements by terms such as, but
not limited to, "may," "might," "will," "objective," "intend,"
"should," "could," "can," "would," "expect," "believe,"
"anticipate," "project," "on track," "scheduled," "target,"
"design," "estimate," "predict," "potential," "aim," "plan" or the
negative of these terms, and similar expressions intended to
identify forward-looking statements. Such forward-looking
statements are based upon current expectations that involve risks,
changes in circumstances, assumptions, and uncertainties. Aldeyra
is at an early stage of development and may not ever have any
products that generate significant revenue. All of Aldeyra's
development timelines may be subject to adjustment depending on
recruitment rate, regulatory review, preclinical and clinical
results, and other factors that could delay the initiation,
enrollment or completion of clinical trials. Important factors that
could cause actual results to differ materially from those
reflected in Aldeyra's forward-looking statements include, among
others, the timing of enrollment, commencement and completion of
Aldeyra's clinical trials, the timing and success of preclinical
studies and clinical trials conducted by Aldeyra and its
development partners; updated or refined data based on Aldeyra's
continuing or post-hoc review and quality control analysis of
clinical data, Aldeyra's ability to design clinical trials with
protocols, data analysis methodologies, and endpoints acceptable to
applicable regulatory authorities; delay in or failure to obtain
regulatory approval of Aldeyra's product candidates; the ability to
maintain regulatory approval of Aldeyra's product candidates, and
the labeling for any approved products; the risk that prior
results, such as signals of safety, activity, or durability of
effect, observed from preclinical or clinical trials, will not be
replicated or will not continue in ongoing or future studies or
clinical trials involving Aldeyra's product candidates in clinical
trials focused on the same or on different indications; the risk
that the results from earlier clinical trials, portions of clinical
trials, or pooled clinical data may not accurately predict results
of subsequent trials or the remainder of a clinical trial; the
scope, progress, expansion, and costs of developing and
commercializing Aldeyra's product candidates; uncertainty as to
Aldeyra’s ability to commercialize (alone or with others) and
obtain reimbursement for Aldeyra's product candidates following
regulatory approval, if any; the size and growth of the potential
markets and pricing for Aldeyra's product candidates and the
ability to serve those markets; Aldeyra's expectations regarding
Aldeyra's expenses and revenue, the sufficiency or use of Aldeyra's
cash resources and needs for additional financing; political,
economic, legal, social, and health risks, including the COVID-19
pandemic and subsequent public health measures, and war or other
military actions, that may affect Aldeyra’s business or the global
economy; the rate and degree of market acceptance of any of
Aldeyra's product candidates; Aldeyra's expectations regarding
competition; Aldeyra's anticipated growth strategies; Aldeyra's
ability to attract or retain key personnel; Aldeyra’s limited sales
and marketing infrastructure; Aldeyra's ability to establish and
maintain development partnerships; Aldeyra’s ability to
successfully integrate acquisitions into its business; Aldeyra's
expectations regarding federal, state, and foreign regulatory
requirements; regulatory developments in the United States and
foreign countries; Aldeyra's ability to obtain and maintain
intellectual property protection for its product candidates; the
anticipated trends and challenges in Aldeyra's business and the
market in which it operates; and other factors that are described
in the “Risk Factors” and “Management's Discussion and Analysis of
Financial Condition and Results of Operations” sections of
Aldeyra's Annual Report on Form 10-K for the year ended December
31, 2021, and Aldeyra’s Quarterly Report on Form 10-Q for the
quarter ended June 30, 2022, which are on file with the Securities
and Exchange Commission (SEC) and available on the SEC's website at
https://www.sec.gov/. Additional factors may be set forth in those
sections of Aldeyra's Quarterly Report on Form 10-Q for the quarter
ended September 30, 2022, expected to be filed with the SEC in the
fourth quarter of 2022.
In addition to the risks described above and in Aldeyra's other
filings with the SEC, other unknown or unpredictable factors also
could affect Aldeyra's results. No forward-looking statements can
be guaranteed and actual results may differ materially from such
statements. The information in this release is provided only as of
the date of this release, and Aldeyra undertakes no obligation to
update any forward-looking statements contained in this release on
account of new information, future events, or otherwise, except as
required by law.
1 United States Pharmacopeia 2 Ophthalmology 124(6):757-767,
2017; Archives of Ophthalmology 25(9):1161-7, 2007.
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Investor & Media Contact: Scott Solomon Sharon
Merrill Associates, Inc. Tel: (857) 383-2409
ALDX@investorrelations.com
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