Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today
announced the submission of a New Drug Application (NDA) to the
U.S. Food and Drug Administration (FDA) for ADX‑2191 (methotrexate
injection, USP), an investigational drug candidate, for the
treatment of primary vitreoretinal lymphoma, a rare but potentially
fatal cancer with no FDA-approved therapy.
The NDA submission is supported by a combination of published
literature on the safety and efficacy of methotrexate for the
treatment of primary vitreoretinal lymphoma and safety data from
the recently completed Phase 3 GUARD Trial of ADX‑2191 for the
prevention of proliferative vitreoretinopathy. During the Phase 3
GUARD Trial, no safety signals were observed, 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 frequently observed
side effect of intravitreal methotrexate, that was most commonly
mild in severity. In the Phase 3 GUARD Trial, the incidence of
punctate keratitis with ADX-2191 administration was observed to be
less than that previously reported with intravitreal injection of
compounded methotrexate.1
“Compounding methotrexate for intravitreal injection, the
current standard of care for primary vitreoretinal lymphoma, poses
several challenges for physicians and patients, including risk of
infection and increased injection volume, potentially leading to
ocular hypertension and corneal inflammation,” stated Todd C.
Brady, M.D., Ph.D., Aldeyra’s President and Chief Executive
Officer. “ADX-2191 is a novel formulation of methotrexate that is
designed to be vitreous-compatible and has the potential to be the
first marketed drug for patients suffering from primary
vitreoretinal lymphoma.”
ADX-2191 has received FDA Orphan Drug Designation for the
treatment of primary vitreoretinal lymphoma. In addition, as part
of the NDA submission, Aldeyra requested Priority Review
Designation, which reduces the review period during which the FDA
aims to take action on an NDA to within 6 months, compared to 10
months under standard review.
A Type C meeting with the FDA to discuss the completion of
clinical development of ADX-2191 for the prevention of
proliferative vitreoretinopathy is planned for the first half of
2023, and results from the Phase 2 clinical trial of ADX-2191 in
retinitis pigmentosa are expected in the first half of 2023.
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, is designed to be
vitreous-compatible, and is 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 for the
treatment of primary vitreoretinal lymphoma and retinitis
pigmentosa.
About Primary Vitreoretinal Lymphoma
Primary vitreoretinal lymphoma is a rare, aggressive, and
potentially fatal retinal cancer that is diagnosed in approximately
300 to 600 patients in the United States per year. The median
survival for newly diagnosed patients is 4.83 years. The most
common ocular complaints reported by patients include blurred
vision, painless loss of vision, floaters, red eye, and
photophobia. No approved treatments are currently available, though
intravitreal injection of compounded methotrexate represents the
current standard of care.2
About Aldeyra
Aldeyra Therapeutics 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. Our pre-commercial
product candidates are reproxalap, a potential treatment for dry
eye disease and allergic conjunctivitis, and ADX-2191, a potential
treatment for primary vitreoretinal lymphoma, proliferative
vitreoretinopathy, and other rare sight-threatening retinal
diseases. In addition, we are developing other product candidates,
including ADX-629 and chemically related molecules, for the
potential treatment of systemic and retinal immune-mediated
diseases. 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 Aldeyra’s belief in the adequacy
of the data it submitted in the NDA for ADX-2191; the potential for
FDA acceptance of an NDA for ADX-2191; the potential for regulatory
approval and commencement of commercialization of ADX-2191 and
Aldeyra’s goals as to timing; the potential profile and benefit of
ADX-2191 in primary vitreoretinal lymphoma; the planned timing of
the Type C meeting with the FDA to discuss the completion of
clinical development of ADX-2191 for the prevention of
proliferative vitreoretinopathy; and the expected timing of results
from the Phase 2 clinical trial of ADX-2191 in retinitis
pigmentosa. 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,” “on schedule,” “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 September 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/.
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 Annals of Hematology, 95(4),
593–601, 2016. 2 Aldeyra internal estimates. Data on file; Primary
Vitreoretinal Lymphoma by D. J. Wilson on AAO EyeWiki; M. Sagoo,
Survey of Ophthalmology (2014); Grimm et. al., Annals of Oncology
(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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