- Best Corrected and Low‑Light Visual Acuity Statistically
Significantly Improved
- As Assessed by Electroretinography, Time to Retinal Response
Statistically Significantly Improved
- As Assessed by Macular and Dark-Adapted Perimetry, Retinal
Sensitivity Statistically Significantly Improved
- ADX‑2191 Was Well Tolerated and No Safety Concerns Were
Identified
- Planned Phase 2/3 Clinical Trial to be Discussed with
Regulatory Authorities
- Company to Discuss Results in Conference Call and Webcast at
8:00 a.m. ET Today
Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a
biotechnology company devoted to discovering and developing
innovative therapies designed to treat immune-mediated diseases,
today announced positive top-line results from the Phase 2 clinical
trial of intravitreal ADX-2191 (methotrexate injection, USP), an
investigational drug candidate, in patients with retinitis
pigmentosa. Relative to baseline, the clinical trial demonstrated
statistically significant improvement in retinal function across a
number of different physiological and psychophysical
assessments.
“The improvement in retinal function relative to baseline
observed in this retinitis pigmentosa clinical trial of ADX-2191
may offer hope to patients that today have no therapeutic options,”
stated Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra.
“Based on compelling proof-of-concept clinical activity that is
consistent with a well-defined mechanism of action supported by
preclinical evidence, we are excited to meet with regulatory
authorities to discuss initiation of a potentially pivotal Phase
2/3 clinical trial, as we enthusiastically advance ADX-2191 to the
next stage of development.”
Based on preclinical evidence suggesting that methotrexate may
facilitate the clearance of mutated rhodopsin1, a protein critical
for visual cycle function, an open-label, single-center Phase 2
clinical trial of ADX‑2191 was performed in eight retinitis
pigmentosa patients with rhodopsin misfolding mutations. Over three
months of treatment with ADX-2191, four patients received monthly
injections and four patients received twice-monthly injections. The
primary endpoint of the clinical trial was safety. Secondary
endpoints included change from baseline in visual acuity; retinal
function, as assessed by macular and dark-adapted chromatic
perimetry and electroretinography; and retinal morphology, as
assessed by optical coherence tomography. Visual acuity, perimetry,
and morphology assessments were performed monthly for four months
from initiation of therapy. Electroretinography was performed at
baseline and at 90 days from initiation of therapy.
All enrolled patients completed the trial per protocol. Relative
to baseline, across all patients, statistical significance was
achieved for improvement in best corrected visual acuity
(P<0.0001), low-light visual acuity (P=0.0001), time to
electroretinographic response to light (P=0.02), macular
sensitivity to light (P<0.0001), and dark-adapted peripheral
sensitivity to light (P<0.0001). ADX‑2191 was well tolerated,
and no safety concerns were identified. No treatment-related
adverse events associated with retinal morphology were observed. No
serious adverse events were reported, and no patients discontinued
due to adverse events.
“Retinitis pigmentosa is a relentlessly progressive disease that
inevitably leads to loss of vision,” stated Ramiro S. Maldonado,
MD., the Principal Investigator of the clinical trial and Assistant
Professor of Ophthalmology at Duke University Medical Center. “The
promising results presented today are supportive of a potential
novel approach for the treatment of retinitis pigmentosa patients
with rhodopsin mutations.”
ADX-2191 is a novel intravitreal formulation of methotrexate in
clinical development for proliferative vitreoretinopathy and
retinitis pigmentosa, both of which are rare, sight-threatening
retinal diseases with no approved therapies. The prevalence of
retinitis pigmentosa is more than one million people worldwide, and
genetic mutations leading to rhodopsin misfolding account for
approximately one-third of cases. ADX-2191 has been granted orphan
drug designation by the U.S. Food and Drug Administration for the
treatment of proliferative vitreoretinopathy and retinitis
pigmentosa.
Conference Call & Webcast Information
Aldeyra will host a conference call at 8:00 a.m. ET today to
discuss top-line results of the Phase 2 clinical trial of ADX‑2191
in retinitis pigmentosa. The dial-in numbers are (888) 415-4305 for
domestic callers and (646) 960-0336 for international callers. The
access code is 5858366. A live webcast of the conference call will
be available on the Investor Relations page of the company’s
website at https://ir.aldeyra.com. After the live webcast, the
event will remain archived on the Aldeyra Therapeutics website for
90 days.
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 Aldeyra’s
future expectations, plans, and prospects, including without
limitation statements regarding: the goals, opportunity, including
market size, and potential for ADX-2191 and anticipated clinical
developments or regulatory milestones for 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,” “on schedule,” “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 may 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, 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.
1FASEB J. 34(8): 10146-10167, 2020.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230629018435/en/
Investor & Media: David Burke Tel: (917) 618-2651
investorrelations@aldeyra.com
Aldeyra Therapeutics (NASDAQ:ALDX)
Historical Stock Chart
From Apr 2024 to May 2024
Aldeyra Therapeutics (NASDAQ:ALDX)
Historical Stock Chart
From May 2023 to May 2024