- Based on positive biomarker results observed in adult cohort
of Phase 2 clinical trial of ADX-629 in Sj�gren-Larsson Syndrome,
proposed expansion to include pediatric patients expected to be
submitted to the US Food and Drug Administration (FDA) in the first
half of 2024
- Phase 2 clinical trial of ADX-629 in moderate alcoholic
hepatitis initiated; top-line results expected in the second half
of 2024
- Investigational New Drug (IND) application of ADX-246
submitted to FDA for Phase 1 clinical trial in healthy volunteers
expected to be expanded to include patients with atopic
dermatitis
- IND application of ADX-248 expected to be submitted to FDA
for Phase 1/2 clinical trial in dry age-related macular
degeneration patients with dark adaptation deficit in 2024
- Special Protocol Assessment for clinical trial of reproxalap
in dry eye disease amended based on FDA feedback
Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra), a
biotechnology company devoted to discovering and developing
innovative therapies designed to treat immune-mediated diseases,
today announced advancement of its RASP modulator platform,
including the expected submission to the FDA of a proposed
expansion of the Phase 2 clinical trial of the investigational RASP
modulator ADX-629 in Sj�gren-Larsson Syndrome to include pediatric
patients, initiation of a Phase 2 clinical trial of ADX-629 in
moderate alcoholic hepatitis, submission of an IND application of
the investigational RASP modulator ADX-246 for a Phase 1 clinical
trial that is expected to be expanded to include atopic dermatitis
patients, anticipated submission of an IND application of the
investigational RASP modulator ADX-248 for a Phase 1/2 clinical
trial in patients with the dry form of age-related macular
degeneration (AMD) and dark adaptation deficit, and initiation of a
preclinical program of RASP modulators in metabolic disease.
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“With an unparalleled RASP modulator discovery and development
platform, Aldeyra remains a leader in the development of RASP
modulators for the treatment of immune-mediated disease,” stated
Todd C. Brady, M.D., Ph.D., President and Chief Executive Officer
of Aldeyra.
Sj�gren-Larsson Syndrome is a rare inborn error of metabolism
due to mutations in fatty aldehyde dehydrogenase, leading to
accumulation of fatty alcohols and fatty aldehydes. The disease is
characterized clinically by cognitive delay, spasticity, and dermal
compromise. There is no FDA-approved therapy for Sj�gren-Larsson
Syndrome. ADX-629, administered twice daily over 90 days, has
completed initial clinical testing in three adult patients in an
investigator-sponsored clinical trial. Broad-based normalization of
the majority of abnormal metabolomic signatures was observed along
with reduction in accumulation of fatty alcohols. A proposed
expansion of the clinical trial to include pediatric patients is
expected to be submitted to the FDA in the first half of 2024.
Moderate alcoholic hepatitis is a common disease that leads to
significant morbidity and mortality due to hepatic compromise and
related complications. There is no FDA-approved therapy for
alcoholic hepatitis. In preclinical models of ethanol toxicity,
ADX-629 decreased hepatic levels of RASP, triglycerides, and
inflammatory cytokines. In a sequence-randomized, double-masked,
placebo-controlled crossover Phase 2 clinical trial announced last
year, relative to placebo, ADX-629 reduced dermal flushing
(P=0.0007), increased Romberg test balance time (P=0.02), and
lowered levels of the ethanol RASP metabolite acetaldehyde (P=0.03)
following acute exposure to alcohol. The moderate alcoholic
hepatitis Phase 2 clinical trial is intended to assess the activity
of ADX-629, administered orally twice daily over 90 days, initially
in approximately 10 patients. The primary endpoint is safety and
tolerability; secondary endpoints include liver function
biomarkers, model for end-stage liver disease (MELD) score,
hospitalization, and mortality rate. Top-line results from the
first patient cohort are expected in the second half of 2024.
Atopic dermatitis is a common disease characterized by immune
dysregulation that leads to hyperreactivity to endogenous and
exogenous factors, potentially including RASP. There is no widely
accepted oral therapy for the treatment of patients with mild to
moderate atopic dermatitis. Recently announced results from a
90-day, open-label Phase 2 clinical trial of orally administered
ADX-629 in eight mild to moderate atopic dermatitis patients
demonstrated statistically significant and clinically relevant
improvement in investigator-assessed and patient-reported outcomes
across a number of different physiological and psychosocial
assessments, including complete resolution of affected body surface
area observed in one patient and elimination of itching reported by
two patients. Based on the results of the clinical trial, an IND
application of next-generation investigational RASP modulator
ADX-246 for a Phase 1 clinical trial in healthy volunteers has been
submitted to the FDA and is expected to be expanded to include
clinical testing in patients with atopic dermatitis. The clinical
trial is anticipated to be initiated in the first half of 2024, and
top-line results are expected in the second half of 2024.
AMD is a common disease, the so-called dry form of which is
characterized by accumulation of toxic metabolites in the retina
and visual deficit, typically beginning with difficulty seeing in
low-light settings. Metabolites of RASP are associated with
compromise in dark adaptation. Based on data demonstrating that
RASP modulation reduces accumulation of toxic metabolites in a
preclinical model of AMD, an IND application of next-generation
investigational RASP modulator ADX-248 is expected to be submitted
in 2024. The Phase 1/2 clinical trial is anticipated to enroll dry
AMD patients with dark adaptation deficit.
Highlighting the advancement of the RASP platform and the
selection of key clinical indications for future development,
Aldeyra released a focused pipeline. The previously announced
programs of ADX-629 in chronic cough and idiopathic nephrotic
syndrome have been de-prioritized due to regulatory and trial
feasibility challenges, respectively. In addition, a new
preclinical RASP modulator program in metabolic disease has been
announced, supported by a number of clinical trials with ADX-629
that demonstrated a reduction in triglycerides and cholesterol.
Based on Special Protocol Assessment feedback received from the
FDA last week on reproxalap in dry eye disease, Aldeyra has amended
the proposed clinical trial protocol and statistical analysis
plan.
“With the success achieved in our ADX-629 program, which
includes a number of positive, signal-finding clinical results, we
are excited to advance our RASP modulator pipeline to systemic and
retinal immune-mediated diseases, as well as metabolic diseases,
exacerbated by RASP,” stated Dr. Brady. “We anticipate 2024 to
provide a variety of clinical-stage catalysts, as Aldeyra continues
to progress its novel RASP platform.”
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 and
allergic conjunctivitis, and ADX-2191, a novel formulation of
intravitreal methotrexate for the potential treatment of
proliferative vitreoretinopathy and retinitis pigmentosa.
About RASP Modulators ADX-629, ADX-246, and ADX-248
ADX-629 is an orally administered RASP modulator currently in
development as a signal-finding molecule for the treatment of
mass-market immune-mediated diseases. ADX-629 has demonstrated
potential activity in clinical trials of patients with psoriasis,
asthma, COVID, ethanol toxicity, chronic cough, and atopic
dermatitis. In more than 100 healthy volunteers and patients, no
consistent adverse events associated with ADX-629 have been
identified.
An analog of ADX-629, ADX-246 is an orally administered
next-generation RASP modulator expected to initiate clinical
testing in the first half of 2024 in a Phase 1/2 clinical trial in
healthy volunteers and patients with atopic dermatitis. Top-line
results from the Phase 1/2 clinical trial are expected in the
second half of 2024.
ADX-248 is an intravitreally administered RASP modulator in
development for the treatment of immune-mediated diseases of the
retina, including the dry form of age-related macular degeneration,
geographic atrophy, and related diseases. ADX-248 is expected to
initiate clinical testing in 2024.
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 and
potential for reproxalap, ADX-629, ADX-246, and ADX-248, outcome
and expected timing of discussions with the FDA on the Special
Protocol Assessment, and anticipated clinical or regulatory
milestones for reproxalap, ADX-629, ADX-246, and ADX-248. 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, 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; 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,
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 September 30, 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 Annual
Report on Form 10-K for the year ended December 31, 2023, expected
to be filed with the SEC in the first quarter of 2024, and
Aldeyra’s other filings with the SEC.
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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240104883191/en/
Investor & Media: David Burke Tel: (917) 618-2651
investorrelations@aldeyra.com
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