AGTC Files Investigational New Drug Application for the Treatment of X-linked Retinitis Pigmentosa Caused by Mutations in the...
10 August 2017 - 9:00PM
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 that it has filed an
Investigational New Drug application (IND) with the U.S. Food and
Drug Administration to conduct a Phase 1/2 clinical trial of the
company's gene therapy product candidate for the treatment of
X-linked retinitis pigmentosa (XLRP) caused by mutations in the
RPGR gene.
XLRP is an inherited condition that causes
progressive vision loss, beginning with night blindness in young
boys followed by progressive constriction of the field of vision.
Affected men progress to legal blindness by their early forties.
Preclinical data indicated that treatment with an investigational
gene therapy product slowed the loss of visual function in canines
with XLRP caused by mutations in the RPGR gene. XLRP and X-linked
retinoschisis (XLRS) are the two lead development programs within
AGTC's collaboration and license agreement with Biogen, Inc.
(NASDAQ:BIIB), to develop gene-based therapies for multiple
ophthalmic diseases.
“We are deeply committed to advancing therapies
to address the unmet needs of patients with rare inherited retinal
disorders, and filing our fifth IND in this field represents a
significant milestone that may ultimately improve the lives of
individuals affected by XLRP,” said Sue Washer, President and CEO
of AGTC. “Gene therapy clinical trials continue to rapidly
progress, making this an exciting time for AGTC, the clinical
community and patients affected by ocular conditions with no
currently available treatments.”
AGTC plans to initiate a clinical study evaluating the safety and
efficacy of its proprietary gene therapy for XLRP caused by
mutations in the RPGR gene in the United States in the coming
months.
AGTC is currently scheduling patients for
enrollment in clinical trials for XLRS as well as for achromatopsia
caused by mutations in the CNGB3 and CNGA3 genes. Patients and
caregivers interested in participating in or learning about AGTC’s
current and upcoming clinical trials may
visit www.agtc.com/patients-and-caregivers or
e-mail advocacy@agtc.com.
About AGTC
AGTC 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 XLRS, XLRP, achromatopsia, wet age-related macular
degeneration, and an 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 part of its
collaboration and license agreement 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.
Forward Looking Statements
This 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 updated by the Company's Quarterly
Report on Form 10-Q for the fiscal quarter ended March 31, 2017, 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)
Lazar Partners Ltd.
T: (212) 867-1768
dcarey@lazarpartners.com
Lazar Partners for AGTC (PR)
T: (212) 867-1762
AGTC_PR@lazarpartners.com
CORPORATE CONTACTS:
Stephen Potter
Chief Business Officer
Applied Genetic Technologies Corporation
T: (617) 413-2754
spotter@agtc.com
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