AGTC Files Investigational New Drug Application for the Treatment of Achromatopsia Caused by Mutations in the CNGA3 Gene
October 19 2016 - 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 that it has filed an
Investigational New Drug application (IND) with the U.S. Food and
Drug Administration to conduct a Phase I/II clinical trial of the
Company's gene therapy product candidate for the treatment of
achromatopsia caused by mutations in the CNGA3 gene. The Company
previously initiated a Phase I/II clinical trial of its gene
therapy product for the treatment of achromatopsia caused by
mutations in the CNGB3 gene.
"Achromatopsia severely impairs vision of
affected individuals which significantly impacts their daily
activities,” said Sue Washer, President and CEO. “The filing of an
IND for our second achromatopsia therapeutic candidate reflects our
commitment to advancing therapies that address the unmet needs of
patients with the two mutations that are the most common causes of
this disease. We believe this filing validates the flexibility and
broad utility of our AAV-based gene therapy platform to develop
multiple gene-based therapies for rare, inherited ophthalmic
diseases and this program will be the fourth ophthalmic gene
therapy candidate we have advanced to human trials.”
Achromatopsia results from mutations in one of
several genes. Two of these genes, CNGA3 and CNGB3, encode the
alpha and beta subunits, respectively, of an ion channel that is
essential for proper function of cone cells within the retina.
About 75 percent of achromatopsia patients have mutations in one of
these two genes; the remainder of cases result from mutations in
one of several other genes. Preclinical studies in dog and sheep
models of achromatopsia demonstrate that the animals are better
able to navigate a maze after receiving gene therapy that delivers
a functional copy of CNGA3 or CNGB3.
The Company plans to initiate a clinical study
evaluating the safety and efficacy of its proprietary gene therapy
for CNGA3-related achromatopsia in the United States in the coming
months.
About Achromatopsia
Achromatopsia is an inherited retinal disease,
which is present from birth and is characterized by the lack of
cone photoreceptor function. The condition results in markedly
reduced visual acuity, extreme light sensitivity causing day
blindness, and complete loss of color discrimination.
Best-corrected visual acuity in persons affected by achromatopsia,
even under subdued light conditions, is usually about 20/200, a
level at which people are considered legally blind. The incidence
rate for achromatopsia is approximately one in 30,000 people, and
it is estimated that there are approximately 10,000 people in the
United States and 17,000 people in Europe with achromatopsia.
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 six named
ophthalmology development programs across five targets (X-linked
retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP),
achromatopsia, wet age-related macular degeneration and blue cone
monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin
deficiency and adrenoleukodystrophy) and AGTC is continuing to
develop early research studies in additional indications. The
company is also exploring genetic defects in cells in the inner ear
that lead to deafness and expects to advance several product
candidates into development within the next few years. AGTC employs
a highly targeted approach to selecting and designing its product
candidates, choosing to develop therapies for indications having
high unmet medical need, clinical feasibility and commercial
potential. 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, reliance on
third parties over which AGTC may not always have full control;
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) 843-5775
spotter@agtc.com
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