Positive 18-Month Data from Ongoing Phase 1/2 Clinical Study of AGTC-501 Presented at the Association for Research in Vision and Ophthalmology 2022 Annual Meeting
May 04 2022 - 8:00AM
Applied Genetic Technologies Corporation (Nasdaq: AGTC), a
clinical stage biotechnology company focused on the development of
adeno-associated virus (AAV)-based gene therapies for the treatment
of rare and debilitating diseases with an initial focus on
inherited retinal diseases, today announced the presentation of
additional positive findings from the ongoing X-linked retinitis
pigmentosa (XLRP) Phase 1/2 study of AGTC-501, including 18-month
safety and efficacy data, at the Association for Research in Vision
and Ophthalmology (ARVO) 2022 Annual Meeting, May 1-4, 2022 in
Denver, Colorado.
The data presented at ARVO update previously reported data from
this non-randomized, open-label Phase 1/2 study that the Company
believes showed both safety and biological activity of AGTC-501 12
months after treatment. The 18-month data appear to show safety and
efficacy signals similar to the study’s 12-month findings,
including improvements to the ellipsoid zone (EZ). In patients with
XLRP, the EZ, a defined region within the photoreceptor layer of
the retina, degenerates over time and is eventually lost. Eighteen
months after treatment, of the eyes in the study with visible
foveal EZ at baseline that underwent subretinal administration of
AGTC-501, two thirds showed recovery of foveal EZ and nearly half
had improved EZ appearance, which correlated with improvement in
macular sensitivity.
“These results indicate sustained durability of improved visual
function over 18 months and suggest evidence of biological activity
for this XLRP gene therapy,” said Paul Yang, M.D., Assistant
Professor of Ophthalmology at Oregon Health & Science
University's Casey Eye Institute, and the trial’s principal
investigator. “Because there are currently no approved treatment
options for patients with XLRP, this data provides an important
step toward a potential treatment for patients with vision loss due
to XLRP.”
At 18 months post treatment, AGTC-501 appeared to be
well-tolerated across a wide dose range. The majority of adverse
events were mild to moderate in severity, including those related
to the subretinal injection procedure and importantly,
immunological assessments did not indicate safety concerns.
“We continue to be enthusiastic about our lead candidate
AGTC-501. The data presented today combined with the prior 12-month
data from this trial are a strong indicator of the potential of
both AGTC-501 and our gene therapy platform,” said Sue Washer,
President and Chief Executive Officer of AGTC. “These data, coupled
with other data from our ongoing clinical trials, reassure us that
we are on the right path to bringing lifechanging therapies for
rare retinal diseases to patients and we look forward to sharing
the three-month interim data from the SKYLINE Phase 2 expansion
portion of this trial in the second quarter of calendar 2022 and
24-month results from this Phase 1/2 trial in the third quarter of
calendar year 2022.”
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 unmet patient needs. AGTC’s most advanced clinical programs
leverage its best-in-class technology platform to potentially
improve vision for patients with inherited retinal diseases. AGTC
has active clinical trials in X-linked retinitis pigmentosa (XLRP)
and achromatopsia (ACHM CNGB3). 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 needs in
optogenetics, otology and CNS disorders, and has entered strategic
collaborations with companies including Bionic Sight, an innovator
in the emerging field of optogenetics, and retinal coding and
Otonomy, Inc., a biopharmaceutical company dedicated to the
development of innovative therapeutics for neurotology. For more
information, please visit https://agtc.com/.
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 gene therapy platform and the strength
of interim results from multiple clinical trials in XLRP, the
potential of AGTC-501 as a treatment for XLRP, and whether these
results will support future regulatory filings. 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; risks and uncertainties
related to funding sources for our development programs; the direct
and indirect impacts of the ongoing COVID-19 pandemic on the
Company’s business, results of operations, and financial condition;
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 the company’s most recent
annual report on Form 10-K, as it may be supplemented by 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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