AGTC Announces Topline Safety Data for X-Linked Retinoschisis Phase 1/2 Study
June 08 2017 - 4:01PM
Applied Genetic Technologies Corporation (NASDAQ:AGTC), a
biotechnology company conducting human clinical trials of
adeno-associated virus (AAV)-based gene therapies for the treatment
of rare diseases, today announced topline safety data for the dose
escalation phase of the company’s Phase 1/2 X-linked retinoschisis
(XLRS) clinical trial, a program partnered with Biogen. Clinical
investigator Mark Pennesi, M.D., Ph.D., Associate Professor in
Ophthalmic Genetics and Chief of the Ophthalmic Genetics division
at Oregon Health and Science University Casey Eye Institute, will
present the data at the Macula Society Annual Meeting in Singapore
on Saturday, June 10th after 8:15 a.m. local time.
Dr. Pennesi will present results for the first
12 subjects enrolled in the low, middle and high dose groups of the
Phase 1/2 dose escalation study. Several subjects have been
followed for more than one year. Mild to moderate ocular
inflammation was observed in the treated eye for the majority of
patients and resolved or was controlled either without further
intervention or after treatment with topical or oral
corticosteroids. No treatment related serious adverse events were
reported and the treatment was generally well tolerated.
“These findings are consistent with previous
results demonstrating that our investigational gene therapy for
XLRS was generally well tolerated and demonstrated a good safety
profile across treatment groups,” said Michael Goldstein, M.D.,
Chief Medical Officer of AGTC. “Based on these results, and in
accordance with a recent review by the Data Safety and Monitoring
Committee, we are continuing enrollment at the highest dose in the
expansion group and look forward to providing additional updates
with respect to safety, potential efficacy and biologic activity
endpoints.”
A copy of Dr. Pennesi’s presentation will be
available at https://www.agtc.com/products/x-Linked-retinoschisis
tomorrow, Friday evening by 8:15 p.m. Eastern Time.
Patients and caregivers interested in participating in or
learning about this trial may
visit www.agtc.com/patients-and-caregivers or
e-mail advocacy@agtc.com.
About AGTCAGTC is a
clinical-stage biotechnology company that uses its proprietary gene
therapy platform to develop products designed to transform the
lives of patients with severe diseases, with an initial focus in
ophthalmology. AGTC's lead product candidates are designed to treat
inherited orphan diseases of the eye, caused by mutations in single
genes that significantly affect visual function and currently lack
effective medical treatments.
AGTC's product pipeline includes ophthalmology
programs in X-linked retinoschisis (XLRS), X-linked retinitis
pigmentosa (XLRP), achromatopsia, wet age-related macular
degeneration, and our optogenetics program with Bionic Sight.
AGTC's non-ophthalmology programs include its adrenoleukodystrophy
program and its otology program, which is in pre-clinical
development, and the company expects to advance several otology
product candidates into clinical development in the next few years.
Each of AGTC's XLRS, XLRP and adrenoleukodystrophy programs is
partnered with Biogen. AGTC employs a highly-targeted approach to
selecting and designing its product candidates, choosing to develop
therapies for indications having high unmet medical need that it
believes are clinically feasible and present commercial
opportunities. AGTC has a significant intellectual property
portfolio and extensive expertise in the design of gene therapy
products including capsids, promoters and expression cassettes, as
well as, expertise in the formulation, manufacture and physical
delivery of gene therapy products.
About X-linked Retinoschisis
(XLRS)XLRS is an inherited retinal disease caused by
mutations in the RS1 gene, which encodes the retinoschisin protein.
It is characterized by abnormal splitting of the layers of the
retina, resulting in poor visual acuity in young boys, which can
progress to legal blindness in adult men.
Forward Looking StatementsThis release contains
forward-looking statements that reflect AGTC's plans, estimates,
assumptions and beliefs. Forward-looking statements include
information concerning possible or assumed future results of
operations, business strategies and operations, preclinical and
clinical product development and regulatory progress, potential
growth opportunities, potential market opportunities and the
effects of competition. 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: no gene therapy products have been approved
in the United States and only two such products have been approved
in Europe; AGTC cannot predict when or if it will obtain regulatory
approval to commercialize a product candidate; uncertainty inherent
in the regulatory review process; risks and uncertainties
associated with drug development and commercialization; 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 Annual Report on Form 10-K
for the fiscal year ended June 30, 2016, as 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.
IR/PR CONTACTS:
David Carey (IR) or Danielle Lewis (PR)
Lazar Partners Ltd.
T: (212) 867-1768 or (212) 843-0211
dcarey@lazarpartners.com or dlewis@lazarpartners.com
CORPORATE CONTACTS:
Larry Bullock
Chief Financial Officer
Applied Genetic Technologies Corporation
T: (386) 462-2204
lbullock@agtc.com
Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
spotter@agtc.com
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