Results support AGTC’s clinical development
strategies in both conditions
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 the presentation of new data from
studies in animal models of achromatopsia (ACHM) and X-linked
retinitis pigmentosa (XLRP) that support the company’s clinical
development programs in these indications. The data were presented
at the American Society of Gene and Cell Therapy 20th Annual
Meeting, taking place in Washington, D.C., May 10-13.
ACHM and XLRP are rare inherited retinal
diseases. ACHM results from mutations in either of the CNGB3 or
CNGA3 genes. Mutations in these genes account for approximately 75
percent of the total achromatopsia patient population. Individuals
with achromatopsia have markedly reduced visual acuity, extreme
light sensitivity, and complete loss of color discrimination.
AGTC is currently enrolling patients in a
clinical trial for its CNGB3 gene-related ACHM treatment candidate,
and is currently scheduling patients to be enrolled in a clinical
trial for its CNGA3 gene-related ACHM treatment candidate. Patients
and caregivers interested in participating in or learning more
about these trials may find more information at
www.agtc.com/patients-and-caregivers or by contacting
advocacy@agtc.com.
XLRP affects boys, causing night blindness by
the time they are ten, and progresses to legal blindness by their
early forties. AGTC is developing a gene-based therapy for XLRP in
collaboration with Biogen and expects to file an Investigational
New Drug (IND) Application with the U.S. Food and Drug
Administration for this product candidate this year.
Lisa R. Keyes, Ph.D., Research Scientist at
AGTC, will present the ACHM data in an abstract titled, “Evaluating
Safety and Efficacy of the AAV2tYF-PR1.7-CNGA3 Vector in
CNGA3-Deficient Sheep” (Abstract #299) today in an oral session
from 4:15 p.m. to 4:30 p.m. EDT. These data are from a study that
assessed toxicity, CNGA3 expression and efficacy of two
subretinally administered vectors [AAV2tYF-PR1.7-hCNGA3 and
AAV5-PR2.1-hCNGA3 (a vector previously shown to rescue cone
photoreceptor responses)] in an animal model of ACHM, over a
12-week evaluation period.
No systemic toxicity was associated with
treatment and no consistent test article-related effects were
observed. Two out of five animals treated with the higher dose of
AAV2tYF-PR1.7-CNGA3 had microscopic findings of outer retinal
atrophy, with or without inflammatory cells in the retina and
choroid that were considered procedural- and/or test
article-related. All vector-treated eyes demonstrated CNGA3
expression, and developed cone-mediated electroretinogram (ERG)
responses with no change in rod-mediated ERG responses.
Improvements in maze navigation times and obstacle collisions were
observed in all vector-treated eyes compared with control eyes and
with pre-dose results in the treated eyes. The researchers conclude
that these results support the use of AAV2tYF-PR1.7-hCNGA3 in
clinical studies in patients with achromatopsia caused by mutations
in CNGA3.
“The improvements in maze navigation times,
obstacle collisions and ERG responses observed with the product
candidate in this study suggest that AAV-based gene therapy has
important potential in the treatment of ACHM resulting from
mutations in the CNGA3 gene,” said Sue Washer, President and CEO of
AGTC. “The favorable tolerability profile observed in this study
also supports the use of this vector construct in human clinical
trials. These study findings provided the basis for the design of
the Phase 1/2 clinical trial of our gene-based therapy for ACHM
resulting from CNGA3 mutations, which is currently scheduling
patients for enrollment.”
Jilin Liu, Associate Scientist at AGTC, will
present the XLRP data in an abstract titled, “Evaluation of
AAV2tYF-GRK1-RPGR Vectors in a Canine Model of RPGR-XLRP”
(Abstract #692) in a poster session May 12, from 5:45 p.m. to 7:45
p.m. EDT.
The poster will include results from a study
evaluating the efficacy of two vectors (AAV2tYF-GRK1-RPGRco and
AAV2tYF-GRK1-RPGRstb) containing the AAV2tYF capsid, human GRK1
promoter and a codon-optimized or stabilized version of the human
RPGR gene administered subretinally in an animal model of mid-stage
XLRP resulting from mutations in the RPGR gene. In this model,
mid-stage disease occurs when animals are approximately 12 weeks of
age and is associated with an approximate 40% loss of
photoreceptors. Two animals per group received RPGRco in the right
eye and RPGRstb in the left eye at each of three dose levels.
Rescue of photoreceptor structure was assessed by clinical
examination and histology and/or immunohistochemistry on retinal
cryosections eight weeks post injection.
No abnormal ophthalmic findings were noted in
any eyes at the middle- or low-dose levels. Fundoscopic examination
at 8 weeks post-dosage showed signs of retinal detachment and
inflammation in the eyes injected with the high dose of either
RPGRco or RPGRstb. Dose-dependent RPGR transgene expression was
observed with both vectors, with greater RPGR expression noted in
eyes injected with RPGRco compared with contralateral eyes injected
with RPGRstb at the same dose levels. Correction of rod opsin and
middle/long wavelength cone opsin mislocalization was demonstrated
in all AAV-RPGR treated eyes. Researchers conclude that the results
demonstrate greater RPGR expression with RPGRco compared with
RPGRstb, and that the middle doses of both vectors resulted in
optimal correction at mid-stage disease with limited inflammation
in this animal model of XLRP.
Data from both the ACHM and XLRP studies were
also presented earlier in the week at ARVO 2017, the Association
for Research in Vision and Ophthalmology Annual Meeting, which took
place in Baltimore from May 7-11.
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 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.
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 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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