AGTC Exceeds Enrollment Target in SKYLINE Trial of AGTC-501 for the Treatment of X-linked Retinitis Pigmentosa (XLRP)
January 11 2022 - 08:00AM
Applied Genetic Technologies Corporation (Nasdaq: AGTC), a clinical
stage biotechnology company focused on the development and
commercialization of adeno-associated virus (AAV)-based gene
therapies for the treatment of rare and debilitating diseases with
an initial focus on inherited retinal diseases (IRDs), today
announced it had exceeded the enrollment target in the Company’s
SKYLINE trial of AGTC-501, a recombinant AAV vector-based gene
therapy developed for the treatment of X-linked retinitis
pigmentosa (XLRP). A total of 14 patients have been enrolled across
the high and low dose groups, exceeding the planned target
enrollment of 12.
“We are pleased to have reached this important milestone in the
clinical development of AGTC-501, which positions us to announce
interim three-month trial results in the second quarter of 2022,”
said Sue Washer, President and Chief Executive Officer of AGTC.
“We’re looking forward to adding to the growing body of evidence
that supports the potential of our product candidate and further
validates the meaningful and durable benefits seen to date in
treated patients. Specifically, we believe the mobility data, in
conjunction with the visual acuity and visual sensitivity data from
the high dose group in the SKYLINE trial, will potentially serve as
a strong indicator of success for not only the final 12-month
endpoint for the SKYLINE trial, but also for the Company's VISTA
trial. We are extremely grateful to the patients and investigators
participating in our clinical trials for their help completing
enrollment with a goal of bringing this potential therapeutic
option to patients suffering from the progressive vision loss
resulting from XLRP.”
SKYLINE is a multi-site expansion of the ongoing Phase 1/2 study
in which patients are randomized to either a high or low dose of
AGTC-501 with the primary objective to identify the proportion of
treated eyes that demonstrate improvement from baseline in measures
of visual sensitivity and visual acuity as well as the patient’s
ability to navigate a mobility maze more successfully under varying
light and challenge conditions. Importantly, this is the first
trial that has the potential to demonstrate a correlation of visual
sensitivity and visual acuity with the maze outcome, a key
functional endpoint in the VISTA trial.
Earlier this year, AGTC released updated data from the ongoing
Phase1/2 clinical trial of AGTC-501, which demonstrated that at 12
months, 50% of patients in the high dose groups were considered
responders to treatment based on visual sensitivity and that
treatment resulted in a statistically significant improvement in
best-corrected visual acuity (BCVA) across all treatment groups in
patients where the macula was treated. Additionally, key findings
on a patient’s baseline characteristics, combined with a novel
application of machine learning techniques, are being applied to
the SKYLINE and VISTA trials to potentially further enhance the
ability to identify likely responders. Importantly, at 12 months
post treatment, primary outcome measures of safety continued to
indicate AGTC-501 to be generally safe and well-tolerated at all
doses. The SKYLINE and VISTA trials are intended to support
registration if they are able to show continued safety and
comparable response rates.
The Company anticipates releasing the following, subject to any
continuing impact of COVID-19:
- 24-month results from the ongoing
Phase 1/2 clinical trial of AGTC-501 in the third quarter of
calendar year 2022;
- 12-month results from the SKYLINE
trial in the first quarter of calendar year 2023; and
- interim results from the VISTA trial
in the first half of calendar year 2023.
About X-linked Retinitis Pigmentosa
(XLRP)X-linked Retinitis Pigmentosa (XLRP) is an inherited
condition that causes progressive vision loss in boys and young
men. Characteristics of the disease include night blindness in
early childhood and progressive constriction of the visual field.
In general, XLRP patients experience a gradual decline in visual
acuity over the disease course, which results in legal blindness
around the 4th or 5th decade of life.
About AGTC-501AGTC-501 (laruparetigene
zosaparvovec), the Company’s lead gene therapy development program,
uses an engineered AAV vector to insert a stabilized and functional
copy of the Retinitis Pigmentosa GTSase Regulator (RPGR) gene into
a patient’s photoreceptor cells. AGTC-501 is comprised of that
stabilized RPGR gene and a promoter that was specifically selected
due to its ability to drive efficient gene expression in rods and
cones, maintain photoreceptor function and delay disease
progression in large animal, naturally occurring preclinical models
of XLRP. In addition, published NHP studies have demonstrated that
AGTC’s proprietary AAV capsid has as much as twice the transfection
efficiency in photoreceptors compared to capsids used in competing
programs. AGTC was granted U.S. Food and Drug Administration (FDA)
orphan drug designation in 2017, as well as European Commission
orphan medicinal product designation in 2016, for its gene therapy
product candidate to treat XLRP caused by mutations in the RPGR
gene.
About AGTCAGTC is a clinical-stage
biotechnology company developing genetic therapies for people with
rare and debilitating ophthalmic, otologic and central nervous
system (CNS) diseases. AGTC is a leader in designing and
constructing all critical gene therapy elements and bringing them
together to develop customized therapies with the potential to
address real patient needs. AGTC’s most advanced clinical programs
leverage its best-in-class technology platform to potentially
improve vision for patients with an inherited retinal disease. AGTC
has active clinical trials in X-linked retinitis pigmentosa (XLRP)
and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical
programs build on the company’s industry leading AAV manufacturing
technology and scientific expertise. AGTC is advancing multiple
important pipeline candidates to address substantial unmet clinical
need in optogenetics, otology and CNS disorders, and has entered
into strategic collaborations with companies including Otonomy,
Inc., a biopharmaceutical company dedicated to the development of
innovative therapeutics for neurotology, and Bionic Sight,
LLC, an innovator in the emerging field of optogenetics and retinal
coding.
Forward-Looking Statements This release
contains forward-looking statements that reflect AGTC's plans,
estimates, assumptions and beliefs, including statements about the
potential of the Company’s late-stage development programs in
X-Linked Retinitis Pigmentosa (XLRP), including the expected timing
for the release of data in its SKYLINE and VISTA trials and the
likelihood that these results further support the potential of
AGTC-501. Forward-looking statements also include information
concerning preclinical and clinical product development and
regulatory progress, potential growth opportunities, and potential
market opportunities. Forward-looking statements include all
statements that are not historical facts and can be identified by
terms such as "anticipates," "believes," "could," "seeks,"
"estimates," "expects," "intends," "may," "plans," "potential,"
"predicts," "projects," "should," "will," "would" or similar
expressions and the negatives of those terms. Actual results could
differ materially from those discussed in the forward-looking
statements, due to a number of important factors. Risks
and uncertainties that may cause actual results to differ
materially include, among others: gene therapy is still novel with
only a few approved treatments so far; AGTC cannot predict when or
if it will obtain regulatory approval to commercialize a product
candidate or receive reasonable reimbursement; uncertainty inherent
in clinical trials and the regulatory review process; risks and
uncertainties associated with drug development and
commercialization; the direct and indirect impacts of the ongoing
COVID-19 pandemic on our business, results of operations, and
financial condition, including impacts on supply chain and
availability of clinical trial material; factors that could cause
actual results to differ materially from those described in the
forward-looking statements are set forth under the heading "Risk
Factors" in our most recent annual report on Form 10-K and
subsequent periodic reports filed with the SEC. Given these
uncertainties, you should not place undue reliance on these
forward-looking statements. Also, forward-looking statements
represent management's plans, estimates, assumptions and beliefs
only as of the date of this release. Except as required by law, we
assume no obligation to update these forward-looking statements
publicly or to update the reasons actual results could differ
materially from those anticipated in these forward-looking
statements, even if new information becomes available in the
future.
PR Contact:
Kerry Sinclair
Spectrum Science Communications
ksinclair@spectrumscience.com
Corporate Contact:
Jonathan Lieber
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (617) 843-5778
jlieber@agtc.com
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