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
– Data reconfirm previously highlighted
safety and efficacy potential of AGTC-501 as a treatment for X-linked
retinitis pigmentosa (XLRP) –
GAINESVILLE,
Fla., and CAMBRIDGE, Mass., May 04, 2022 (GLOBE NEWSWIRE) -- 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 AGTC AGTC
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.
BioFlorida is the voice of Florida's life sciences industry, representing 8,600 establishments and research organizations in the BioPharma, MedTech, Digital Health and Health Systems that collectively employ nearly 107,000 Floridians. Source: TEConomy/BIO (released 2022)
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