- TRANQUILITY Primary Endpoint of Ocular Redness Not Met, but
Statistical Significance Achieved for Secondary Endpoint of
Schirmer Test (p=0.0001), an Approvable Sign of Dry Eye
Disease
- Primary Endpoint of TRANQUILITY-2 Trial Modified to be Met if
Either Ocular Redness or Schirmer Test Achieved; Target Enrollment
Increased from 300 to up to 400 Patients; Top-Line Results Expected
Mid-2022
- Dry Eye Disease NDA Submission Expected Mid-2022, Pending
Safety Trial Enrollment and Outcome of TRANQUILITY-2
- Company to Host Conference Call at 5:00 p.m. ET Today
Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra or the
Company), a biotechnology company developing novel
immune-modulating therapies to treat ocular and systemic diseases,
today announced top-line results from the Phase 3 TRANQUILITY Trial
of 0.25% reproxalap ophthalmic solution (reproxalap), an
investigational product candidate for the treatment of dry eye
disease.
Although the primary endpoint of ocular redness was not met in
TRANQUILITY, statistical significance (p=0.0001) was achieved for
the dry eye disease sign of Schirmer test, a secondary endpoint.
Statistical significance (p<0.0001) was also achieved for the
post-hoc assessment of Schirmer test responders of ≥10 mm. The
Schirmer test has been accepted by the U.S. Food and Drug
Administration (FDA) as part of the basis of approval of other dry
eye disease products. The primary endpoint of the upcoming Phase 3
TRANQUILITY-2 Trial has been modified such that the endpoint will
have been met if either Schirmer test or ocular redness
demonstrates stastisical significance. In addition, target
enrollment in TRANQUILITY-2 has been increased from 300 to up to
400 patients. Top-line results from TRANQUILITY-2 are expected
mid-2022.
“Following the achievement of statistical significance in ocular
redness in our recent Phase 2 clinical trial, the achievement of
statistical significance of Schirmer test in TRANQUILITY may
provide an additional option to satisfy the remaining objective
sign requirement for dry eye disease NDA submission,” stated Todd
C. Brady, M.D., Ph.D., President and Chief Executive Officer of
Aldeyra. “Subject to agreement with the FDA, we believe that the
TRANQUILITY results allow for the possibility that, pending the
outcome of TRANQUILITY-2, the NDA submission for reproxalap could
represent the first time a dry eye disease drug will have qualified
for the demonstration of activity for two objective signs.”
Per draft FDA guidance, to be considered for regulatory approval
in the United States, a product candidate for the treatment of dry
eye disease must demonstrate efficacy in an objective sign in at
least two clinical trials and efficacy in a subjective symptom in
at least two clinical trials. To satisfy the symptom efficacy
requirements, Aldeyra intends to submit two previously completed
12-week adequate and well-controlled symptom trials that
pre-specified patient-reported ocular dryness score as a primary
endpoint, the Phase 3 RENEW-Part 1 Trial and the Formulation Phase
2 clinical trial. Aldeyra’s recently completed Phase 2 clinical
trial achieved the primary endpoint of ocular redness, an
approvable sign of dry eye disease. Pending discussion with the FDA
and the results of TRANQUILITY-2, Aldeyra may submit two pivotal
trials for either ocular redness (Phase 2 and TRANQUILITY-2) or
Schirmer test (TRANQUILITY and TRANQUILITY-2), or two trials for
both signs (Phase 2, TRANQUILITY, and TRANQUILITY-2) if ocular
redness and Schirmer test are achieved in TRANQUILITY-2. Phase 2
and Phase 3 clinical trials can be submitted as pivotal, provided
that the trials are adequate and well-controlled.
Pending enrollment of the ongoing 12-month safety trial in dry
eye disease patients and the outcome of TRANQUILITY-2, the dry eye
disease NDA (New Drug Application) submission is expected to occur
mid-2022. NDA submission for allergic conjunctivitis, another
proposed indication, is expected to occur after the dry eye disease
submission, and following completion of an additional allergen
chamber trial requested by the FDA. Primary and key secondary
endpoints in allergic conjunctivitis were previously achieved in
the Phase 3 ALLEVIATE conjunctival allergen challenge trial and the
Phase 3 INVIGORATE allergen chamber trial.
“Notwithstanding the inherent variability of dry eye disease
clinical trials, our investigational product candidate 0.25%
reproxalap has now demonstrated activity in four late-stage
clinical trials with the intended commercial dosing regimen,” Dr.
Brady stated. “We continue to advance reproxalap toward NDA
submission as we focus on the completion of TRANQUILITY-2 and
enrollment in the 12-month safety trial.”
No safety signals were observed in TRANQUILITY, and reproxalap
was well tolerated; there were no treatment-related
discontinuations or moderate or serious adverse events. Reproxalap
has now been tested in over 1,500 patients. The most common
reported adverse event is mild and transient instillation site
discomfort.
Conference Call Information
Aldeyra will host a conference call to discuss this announcement
at 5:00 p.m. ET today, December 20, 2021. The dial-in numbers are
(866) 211-4098 for domestic callers and (647) 689-6613 for
international callers. The Conference ID is 8641858. A live webcast
of the conference call will also be available on the “Investors
& Media section” of the Aldeyra Therapeutics website at
https://ir.aldeyra.com. Presentation slides will be available on
the investor relations page prior to the start of the conference
call and webcast.
About Reproxalap
Reproxalap, an investigational new drug, is a novel
small-molecule immune-modulating covalent inhibitor of RASP
(reactive aldehyde species), which are elevated in ocular and
systemic inflammatory disease. Reproxalap’s mechanism of action has
been validated with the demonstration of statistically significant
and clinically relevant activity in multiple physiologically
distinct late-phase clinical indications. Reproxalap is currently
in Phase 3 clinical development as a 0.25% ophthalmic solution for
the treatment of dry eye disease and allergic conjunctivitis, two
ocular inflammatory diseases that often occur together.
About Dry Eye Disease
Dry eye disease is a common inflammatory disease estimated to
affect 34 million or more adults in the United States.1 The disease
is characterized by insufficient moisture and lubrication in the
anterior surface of the eye, leading to dryness, inflammation,
pain, discomfort, irritation, diminished quality of life, and in
severe cases, permanent vision impairment. Among many physicians
and patients, existing therapy for dry eye disease is generally
regarded as inadequate and often requires weeks or months to
demonstrate activity. In patients with dry eye disease,
pro-inflammatory RASP may contribute to ocular inflammation and
changes in tear lipid composition.2 By diminishing RASP levels,
Aldeyra’s lead RASP inhibitor reproxalap represents a novel and
differentiated approach for the treatment of the symptoms and signs
of dry eye disease.
About Aldeyra Therapeutics, Inc.
Aldeyra Therapeutics, Inc. is a biotechnology company developing
novel immune-modulating therapies to treat ocular and systemic
diseases. Two of the Company’s lead product candidates, reproxalap
and ADX-629, target RASP, which are pre-cytokine, systems-based
mediators of inflammation. Reproxalap is being evaluated in Phase 3
clinical trials in patients with dry eye disease and allergic
conjunctivitis. The Company’s clinical pipeline also includes
ADX-2191 (methotrexate for intravitreal injection), a drug
candidate in Phase 3 testing for the prevention of proliferative
vitreoretinopathy. 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
development plans and expectations for its product candidates,
including plans and expectations relating to current or future
clinical development and regulatory progress of reproxalap in dry
eye disease and allergic conjuctivitis. 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 or completion of clinical trials. In
addition, FDA draft guidance and discussions with the Company are
not binding on the agency. 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, including the timing of the 12-month safety trial
of reproxalap in dry eye disease; 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 review and quality control analysis of
clinical data, Aldeyra's ability to design clinical trials with
protocols 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 FDA may not accept
Aldeyra’s NDA submission for review; 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) 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 related public health measures, 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, 2020 and Quarterly Report on Form 10-Q
for the quarter ended September 30, 2021, 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 Paulsen AJ, Cruickshanks KJ, Fischer ME, Huang GH, Klein BE,
Klein R, Dalton DS. Dry eye in the beaver dam offspring study:
prevalence, risk factors, and health-related quality of life. Am J
Ophthalmol. 2014 Apr;157(4):799-806. doi:
10.1016/j.ajo.2013.12.023. Epub 2014 Jan 2. PMID: 24388838; PMCID:
PMC3995164.
2 Choi W, Lian C, Ying L, Kim GE, You IC, Park SH, Yoon KC.
Expression of Lipid Peroxidation Markers in the Tear Film and
Ocular Surface of Patients with Non-Sjogren Syndrome: Potential
Biomarkers for Dry Eye Disease. Curr Eye Res. 2016
Sep;41(9):1143-9. doi: 10.3109/02713683.2015.1098707. Epub 2016 Jan
5. PMID: 26731289.
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version on businesswire.com: https://www.businesswire.com/news/home/20211220005789/en/
Corporate Contact: Joshua Reed Aldeyra Therapeutics, Inc.
Tel: 781-761-4904 ext. 218 jreed@aldeyra.com
Investor & Media Contact: Scott Solomon Sharon
Merrill Associates, Inc. Tel: 617-542-5300
ALDX@investorrelations.com
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