Ocular Therapeutix, Inc. (NASDAQ: OCUL, “Ocular”, the “Company”), a
biopharmaceutical company committed to enhancing people’s vision
and quality of life through the development and commercialization
of innovative therapies for wet age-related macular degeneration
(wet AMD), diabetic retinopathy, and other diseases and conditions
of the eye, today hosted an Investor Day where it highlighted
excellent clinical development progress with AXPAXLI for wet
age-related macular degeneration (wet AMD) and non-proliferative
diabetic retinopathy (NPDR) and updated its corporate strategy.
“2024 has been a time of significant accomplishment for Ocular.
To support our mission to become a leader in retinal care, we
successfully assembled a 'Retina Dream Team' of recognized leaders
in the field, fortified our balance sheet with additional capital
and streamlined the organization. We are already seeing the
positive impact of these efforts, based on exceptional enrollment
in the pivotal SOL-1 AXPAXLI study for wet AMD, coupled with plans
for a new SOL-R repeat dosing study and the report of positive
48-week topline data from the Phase 1 HELIOS NPDR study,”
said Pravin U. Dugel, MD, Executive Chairman, President and
Chief Executive Officer of Ocular Therapeutix. “Our
transformation into a retina-focused company is built on three
pillars: convincing data from three clinical studies with AXPAXLI,
de-risking the regulatory pathway in wet AMD, and a focus on
expansive, retinal vascular disease markets. We are more confident
today than ever before that AXPAXLI has demonstrated monotherapy
activity, with potential best-in-class durability and a favorable
safety profile that is well positioned to disrupt today’s treatment
paradigm which includes frequent, burdensome regimens.”
Investor Day Highlights
- Excellent enrollment in AXPAXLI SOL-1 study (Phase 3,
wet AMD) with 60 study sites active and 151 subjects
enrolled in various stages of loading and randomization as of June
7, 2024, reflecting strong investigator engagement. The pivotal
SOL-1 study is designed for regulatory approval and is being
conducted under a Special Protocol Assessment (SPA) with the US
FDA. The Company intends to randomize 300 treatment naïve patients
in the SOL-1 study, comparing a single AXPAXLI implant (450µg) to a
single aflibercept injection (2mg) after all patients have received
two loading doses of aflibercept.
- Company plans to initiate SOL-R AXPAXLI repeat dosing
study (Phase 3, wet AMD) to evaluate repeat dosing of
AXPAXLI (450µg) at six months (Q6M) compared to aflibercept (2mg)
dosed every eight weeks (Q8W) and a comparator arm dosed Q6M in 825
wet AMD patients. This trial is designed for commercial impact and
is being initiated at regulatory risk. Ocular has requested a Type
C meeting to seek FDA guidance regarding the SOL-R protocol.
Patients enrolled in SOL-R are initially expected to include
patients that constitute loading or randomization failures from the
SOL-1 study. The SOL-R protocol requires that all patients enrolled
in SOL-R be enriched through multiple aflibercept (2mg) loading
doses and monitored to ensure limited retinal fluid fluctuations
prior to randomization. After the completion of enrollment and
randomization of SOL-1, patients will be enrolled directly into the
SOL-R study.
- Positive HELIOS 48-week data (Phase 1, NPDR)
improves on previously reported 40-week data with all signals of
diabetic retinopathy severity scale (DRSS) improvement coming from
AXPAXLI-treated patients while any vision threatening complications
(VTCs) that developed were in sham-treated patients. The HELIOS
study compares a single AXPAXLI implant (600µg) to a single sham
injection with neither arm receiving a loading treatment. The
Company plans to discuss these results with the FDA to determine a
path forward for the development of AXPAXLI in NPDR.
- 23.1% of patients (N=3) demonstrated a ≥2-step DRSS improvement
in the AXPAXLI arm at week 48 compared to 0% in the sham arm; an
additional 23.1% of patients (N=3) demonstrated a 1-step DRSS
improvement in the AXPAXLI arm compared to 0% in the sham arm.
- 0% of patients in the AXPAXLI arm showed worsening in DRSS by
week 48 compared to 25% of patients (N=2) in the sham arm.
- 0% of patients in the AXPAXLI arm developed proliferative
diabetic retinopathy (PDR) or center-involved diabetic macular
edema (CI-DME) by week 48 compared to 37.5% of patients (N=3) in
the sham arm.
- On average, patients in the AXPAXLI arm showed improvement in
central subfield thickness compared to the sham arm, which showed
worsening over the 48-week follow up.
- AXPAXLI was generally well-tolerated and did not result in any
reported incidence of intraocular
inflammation, iritis, vitritis, or vasculitis.
- Cash runway remains into 2028 with a
prioritization of resources on the clinical development of AXPAXLI
for wet AMD. Based on the Company’s current
operating plan, which includes the completion of the SOL-1 and
SOL-R clinical trials, the assessment of AXPAXLI for NPDR and
PAXTRAVA™ for ocular hypertension and glaucoma, the cessation of
substantive development activities for other clinical programs, the
recent personnel changes in research and technical operations, the
anticipated revenues from DEXTENZA® product sales, and the
Company’s observance of its minimum liquidity covenant, the Company
believes its current cash and cash equivalents continue to enable
it to fund its planned operating expenses, its debt service
obligations and its capital expenditure requirements into
2028.
Dilsher S. Dhoot, MD, of California Retina
Consultants commented, “While SOL-1 enrollment has
exceeded our expectations, perhaps we should not have been
surprised. We are recruiting treatment naïve wet AMD patients with
good vision, the largest de novo patient population we see, and
there is no competing clinical trial for these patients. Many
patients know someone with wet AMD and understand how burdensome
treatment is. When I talk to my patients about the SOL-1 study,
they are excited about the potential durability of AXPAXLI. In
addition, they like the study design because if they are
randomized, they are guaranteed to receive an active agent, not a
sham. They find it comforting to know that I will see them monthly,
at a minimum, and if the drug they are receiving appears to be
losing strength, they can be rescued with aflibercept, as needed,
for the remainder of the study.”
Baruch D. Kuppermann, MD, PhD, Chair of the Department
of Ophthalmology, and Director of the Gavin Herbert Eye Institute
at the University of California, Irvine noted, “I believe
the start of the SOL-R study will be good news for patients and the
retina community. Clearly, SOL-1 was designed as a regulatory
study. It is exactly what the FDA is asking for in their latest
guidance documents and is being conducted under an SPA, which has
become increasingly important in today’s regulatory landscape.
SOL-R is different because it is a large, ‘real-world’
repeat-dosing study designed to provide physicians with data on how
we may ultimately use AXPAXLI, if approved. I believe having the
results of this study will be important for the retina community
and so I am enthusiastic about the clinical trial. Further, I
expect the SOL-R design feature to initially enroll patients who
failed loading or randomization in SOL-1 to enhance enrollment in
both studies by giving patients twice the opportunity to
participate in a study.”
Dr. Dhoot added, “After
reviewing the expanded HELIOS Phase 1 data set in NPDR, I am even
more comfortable that AXPAXLI has the potential to be safe and
effective, with durable activity in the back of the eye. The
current approved anti-VEGF agents for DR necessitate frequent
injections, and for that reason I have very few patients receiving
treatment. In the HELIOS study, patients receiving AXPAXLI
sustained or improved DRSS to week 48 without the development of a
single vision threatening complication, unlike patients in the sham
control arm. In NPDR, I care about maintaining or improving my
patients’ vision, and if I can do this with a 6 to 12-month
treatment, I believe many of my patients would be very excited.
This is a differentiated result compared to other benchmark agents
and it fortifies my confidence in the potential use of AXPAXLI in
both wet AMD and NPDR.”
A replay of the webcast and presentation can be accessed through
the "Events and Presentations" section of the Company’s investor
website at investors.ocutx.com. A replay of the webcast will be
archived for at least 30 days.
About the SOL-1 StudyThe pivotal Phase 3 SOL-1
trial is designed to evaluate the safety and efficacy of AXPAXLI in
a multi-center, double-masked, randomized (1:1), parallel group
study that involves sites primarily located in the U.S. The trial
is intended to randomize approximately 300 evaluable
treatment-naïve patients with a diagnosis of wet AMD in the study
eye.
The superiority study begins with an eight-week loading segment,
a 9-month treatment segment followed by a safety follow-up. During
the loading segment, subjects who have 20/80 vision or better and
who satisfy other enrollment criteria receive two doses of
aflibercept (at week -8 and week -4). Subjects that achieve visual
acuity of 20/20 at Day 1 or gain at least 10 early treatment
diabetic retinopathy (ETDRS) letters at Day 1 are then randomized
to receive a single dose of AXPAXLI or a single dose of aflibercept
and assessed monthly for the entire study. The clinical trial
protocol requires that, during the study, subjects in any arm
meeting pre-specified rescue criteria will receive a supplemental
dose of aflibercept.
The primary endpoint of SOL-1 is the proportion of subjects who
maintain visual acuity, defined as <15 ETDRS letters of BCVA
loss, at Week 36.
About the SOL-R StudyThe Phase 3 SOL-R trial is
designed to evaluate the safety and efficacy of AXPAXLI in a
multi-center, double-masked, randomized (2:2:1), three-arm study
that will involve sites located in the U.S. and the rest of the
world. The trial is intended to randomize approximately 825
patients diagnosed with wet AMD within three months prior to
enrollment in the study eye.
The one-year, non-inferiority study reflects a patient
enrichment strategy that includes multiple loading doses of
aflibercept and monitoring to exclude those with significant
retinal fluid fluctuations. In the first arm, patients will be
randomized to receive a single dose of AXPAXLI at Day 1 and
re-dosed at Week 24. In the second arm, patients will receive
aflibercept (2mg) on label every 8 weeks. In a third arm, patients
will receive a single dose of the comparator at Day 1 and re-dosed
at Week 24, similar to the first arm. The clinical trial protocol
requires that, during the study, subjects in any arm meeting
pre-specified rescue criteria will receive a supplemental dose of
aflibercept.
The primary endpoint is non-inferiority in mean best corrected
visual acuity (BCVA) change from baseline between the AXPAXLI and
on-label aflibercept (2mg) arms at one year.
About the HELIOS studyThe Phase 1 HELIOS trial
is a multi-center, double-masked, randomized (2:1), parallel group
study conducted in the U.S. The study was designed to evaluate
the safety, tolerability, and efficacy of AXPAXLI compared to a
sham control in subjects with moderately severe to severe
non-proliferative diabetic retinopathy (NPDR) without
center-involved diabetic macular edema (CI-DME). The data set
reported here is based on 21 evaluable subjects (one patient of the
22 enrolled subjects died from an unrelated event). The primary
endpoint of the study is frequency of treatment emergent adverse
events (TEAEs). Secondary study endpoints include changes in the
diabetic retinopathy severity score (DRSS), changes in best
corrected visual acuity (BCVA) compared to baseline, changes in
central subfield thickness (CSFT) compared to baseline, and the
portion of subjects receiving rescue therapy.
About AXPAXLI™AXPAXLI is an investigational
bioresorbable, hydrogel implant incorporating axitinib, a small
molecule, multi-target, tyrosine kinase inhibitor with
anti-angiogenic properties, being evaluated for the treatment of
wet AMD, DR, and other retinal diseases.
About Ocular Therapeutix, Inc.Ocular
Therapeutix, Inc. is a biopharmaceutical company committed to
enhancing people’s vision and quality of life through the
development and commercialization of innovative therapies for wet
age-related macular degeneration (wet AMD), diabetic retinopathy
(DR), and other diseases and conditions of the
eye. AXPAXLI™ (axitinib intravitreal implant, also known
as OTX-TKI), Ocular’s product candidate for retinal disease, is
based on its ELUTYX™ proprietary bioresorbable hydrogel-based
formulation technology. AXPAXLI is currently in a Phase 3 clinical
trial for wet AMD. The clinical portfolio also
includes PAXTRAVA™ (travoprost intracameral implant, also
known as OTX-TIC), currently in a Phase 2 clinical trial for the
treatment of open-angle glaucoma or ocular hypertension.
Ocular’s expertise in the formulation, development, and
commercialization of innovative therapies of the eye and the ELUTYX
platform supported the development and launch of its first
commercial drug product, DEXTENZA®, an FDA-approved
corticosteroid for the treatment of ocular inflammation and pain
following ophthalmic surgery and ocular itching associated with
allergic conjunctivitis.
Follow us on our website, LinkedIn, or X.
The Ocular Therapeutix logo and DEXTENZA® are registered
trademarks of Ocular Therapeutix, Inc. AXPAXLI™, PAXTRAVA™,
ELUTYX™, and Ocular Therapeutix™ are trademarks of Ocular
Therapeutix, Inc.
Forward-Looking Statements: Any statements in
this press release about future expectations, plans, and prospects
for the Company, including the development and regulatory status of
the Company’s product candidates; the timing, design, and
enrollment of the Company’s SOL-1 Phase 3 clinical trial of AXPAXLI
(also called OTX-TKI) for the treatment of wet AMD; the timing,
design, and initiation of the Company’s SOL-R Phase 3 clinical
trial of AXPAXLI; the Company’s plans to advance the development of
AXPAXLI and its other product candidates; the potential utility of
any of the Company’s product candidates; the Company’s objective to
become a leader in retinal care; the Company’s cash runway and the
sufficiency of the Company’s cash resources; and other statements
containing the words “anticipate”, “believe”, “estimate”, “expect”,
“intend”, “goal”, “may”, “might”, “plan”, “predict”, “project”,
“target”, “potential”, “will”, “would”, “could”, “should”,
“continue”, and similar expressions, constitute forward-looking
statements within the meaning of The Private Securities Litigation
Reform Act of 1995. Actual results may differ materially from those
indicated by such forward-looking statements as a result of various
important factors. Such forward-looking statements involve
substantial risks and uncertainties that could cause the Company’s
preclinical and clinical development programs, future results,
performance or achievements to differ significantly from those
expressed or implied by the forward-looking statements. Such risks
and uncertainties include, among others, the timing and costs
involved in commercializing DEXTENZA or any product or product
candidate that receives regulatory approval; the ability to retain
regulatory approval of DEXTENZA or any product or product candidate
that receives regulatory approval; the initiation, design, timing,
conduct and outcomes of ongoing and planned clinical trials; the
risk that the FDA will not agree with the Company’s interpretation
of the written agreement under Special Protocol Assessment for the
SOL-1 trial; the risk that even though the FDA has agreed with the
overall design of the SOL-1 trial, the FDA may not agree that the
data generated by the SOL-1 trial supports potential marketing
approval; uncertainty as to the FDA’s view of the Company’s
proposed design for the SOL-R trial; uncertainty as to whether the
data from earlier clinical trials will be predictive of the data of
later clinical trials, particularly later clinical trials that have
a different design or utilize a different formulation than the
earlier trials, or whether preliminary or interim data from a
clinical trial will be predictive of final data from such trial;
availability of data from clinical trials and expectations for
regulatory submissions and approvals; the Company’s scientific
approach and general development progress; uncertainties inherent
in estimating the Company’s cash runway, future expenses and other
financial results, including its ability to fund future operations,
including clinical trials; the Company’s existing indebtedness and
the ability of the Company’s creditors to accelerate the maturity
of such indebtedness upon the occurrence of certain events of
default; the Company’s ability to enter into strategic alliances or
generate additional funding on a timely basis, on favorable terms,
or at all; and other factors discussed in the “Risk Factors”
section contained in the Company’s quarterly and annual reports on
file with the Securities and Exchange Commission. In addition, the
forward-looking statements included in this press release represent
the Company’s views as of the date of this press release. The
Company anticipates that subsequent events and developments may
cause the Company’s views to change. However, while the Company may
elect to update these forward-looking statements at some point in
the future, the Company specifically disclaims any obligation to do
so, whether as a result of new information, future events or
otherwise, except as required by law. These forward-looking
statements should not be relied upon as representing the Company’s
views as of any date subsequent to the date of this press
release.
Investors & MediaOcular Therapeutix,
Inc.Bill SlatteryVice President, Investor
Relationsbslattery@ocutx.com
Ocular Therapeutix (NASDAQ:OCUL)
Historical Stock Chart
From Jul 2024 to Aug 2024
Ocular Therapeutix (NASDAQ:OCUL)
Historical Stock Chart
From Aug 2023 to Aug 2024