- Based on U.S. Food & Drug Administration (FDA)
Determination of Lack of Adequate and Well Controlled
Investigations in the Scientific Literature, Complete Response
Letter Received for New Drug Application (NDA) of ADX-2191
(methotrexate injection, USP) for the Treatment of Primary
Vitreoretinal Lymphoma (PVRL)
- Due to Shortage of Methotrexate, Lack of Approved Therapy
for PVRL, and Inbound Requests for ADX-2191, Expanded Access
Program Planned to be Discussed with FDA
(Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to
discovering and developing innovative therapies designed to treat
immune-mediated diseases, today announced receipt of a Complete
Response Letter from the U.S. Food and Drug Administration (FDA)
for the 505(b)(2) New Drug Application (NDA) of ADX-2191
(methotrexate for injection, USP), an investigational drug
candidate, for the treatment of primary vitreoretinal lymphoma
(PVRL). Although no safety or manufacturing issues with ADX-2191
were identified, the FDA stated that there was a “lack of
substantial evidence of effectiveness” due to “a lack of adequate
and well-controlled investigations” in the literature-based NDA
submission. Based on prior discussions with the FDA, Aldeyra did
not conduct any clinical trials of ADX-2191 in PVRL.
“While we appreciate the FDA’s position with respect to
providing evidence from adequate and controlled trials, we do not
currently believe that randomized clinical trials of ADX-2191 in
PVRL, a rare and fatal cancer with no approved therapy, are
feasible,” stated Todd C. Brady, M.D., Ph.D., President and Chief
Executive Officer of Aldeyra Therapeutics. “Given the current
shortage of methotrexate, the lack of approved therapy for PVRL,
and the desire to avoid potential safety risks associated with
ocular injection of compounded formulations, we look forward to
discussing with the FDA the potential for making ADX-2191 available
to PVRL patients under an Expanded Access Program.”
PVRL is a rare, high-grade, aggressive cancer, with a median
survival of less than five years.1 Methotrexate, the compounded
intravitreal injection of which is the standard of care for the
treatment of PVRL,2 is currently in shortage, per the FDA Drug
Shortages database. An Expanded Access Program allows for access to
treatment options for serious diseases when other therapeutic
options are not available. Aldeyra plans to discuss ADX-2191 for
the treatment of PVRL with the FDA, including the potential to make
ADX-2191 accessible to PVRL patients under an Expanded Access
Program protocol.
ADX-2191 is also under development for the treatment of
proliferative vitreoretinopathy and retinitis pigmentosa, both of
which are rare, sight-threatening retinal diseases. Top-line
results from a Phase 2 clinical trial of ADX-2191 in retinitis
pigmentosa are expected to be announced in June of 2023.
Additionally, Aldeyra plans to conduct a Type C meeting with the
FDA in the second half of 2023 to discuss the completion of
clinical development of ADX-2191 for the prevention of
proliferative vitreoretinopathy.
About Aldeyra
Aldeyra Therapeutics is a biotechnology company devoted to
discovering innovative therapies designed to treat immune-mediated
diseases. Our approach is to develop 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 product candidates
include RASP (reactive aldehyde species) modulators ADX‑629,
ADX‑246, ADX‑248, and chemically related molecules for the
potential treatment of systemic and retinal immune-mediated
diseases. Our pre-commercial product candidates are reproxalap, a
RASP modulator for the potential treatment of dry eye disease
(under U.S. Food and Drug Administration New Drug Application
Review) and allergic conjunctivitis, and ADX-2191, a novel
formulation of intravitreal methotrexate for the potential
treatment of proliferative vitreoretinopathy and retinitis
pigmentosa.
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 the potential
for an ADX-2191 Expanded Access Program, Aldeyra’s plans and
expectations for ADX-2191 and future discussions with the FDA
regarding ADX-2191. 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,” “contemplates,” “likely,”
“potential,” “continue,” “ongoing,” “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, funding,
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;
delay in or failure to obtain regulatory approval of Aldeyra's
product candidates, including as a result of the FDA not accepting
Aldeyra’s regulatory filings, issuing a complete response letter,
or requiring additional clinical trials or data prior to review or
approval of such filings; 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 different indications; the scope, progress, expansion, and costs
of developing and commercializing Aldeyra's product candidates; the
current and potential future impact of the COVID-19 pandemic on
Aldeyra’s business, results of operations, and financial position;
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 future revenue, the timing of
future revenue, the sufficiency or use of Aldeyra's cash resources
and needs for additional financing; 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 commercialization, marketing and manufacturing
capabilities and strategy; 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; 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; 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, 2022, and Aldeyra’s Quarterly Report on
Form 10-Q for the quarter ended March 31, 2023, 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 described in those sections of Aldeyra's Quarterly Report on
Form 10-Q for the quarter ended June 30, 2023, expected to be filed
with the SEC in the third quarter of 2023.
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.
_____________________
1Annals of Oncology, 18(11): 1851–1855,
2007.
2British Journal of Haematology, 194:
92–100, 2021.
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Investor & Media Contact:
David Burke Tel: (917) 618-2651
investorrelations@aldeyra.com
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