AGTC and Collaborators Present New Data on the Natural History of X-Linked Retinoschisis (XLRS) at the Association for Resear...
May 09 2017 - 7:01AM
Researchers at the Casey Eye Institute, the Retina Foundation of
the Southwest, the Kellogg Eye Center and 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,
reported results from a study of the natural history of X-linked
retinoschisis (XLRS) and the impact of carbonic anhydrase
inhibitors (CAIs) on disease progression. The data were presented
this week at ARVO 2017, the Association for Research in Vision and
Ophthalmology Annual Meeting, taking place in Baltimore from May
7-11.
Maria A. Parker, M.D., Senior Project Manager at
the Casey Eye Institute at the Oregon Health and Science
University, presented the data in a poster titled “Natural History
and Effect of Carbonic Anhydrase Inhibitor Use in X-Linked
Retinoschisis” (Abstract #1490).
XLRS is characterized by abnormal splitting of
the layers of the retina, resulting in poor visual function in
young boys, which can ultimately result in legal blindness in adult
men. The study was designed to characterize the natural history of
XLRS and to determine the effect of CAIs on retinal function and
structure in XLRS patients.
This observational study enrolled 56 patients
six years of age and older (average 30.0 years) with a confirmed
mutation in the RS1 gene. Of the 56 patients, 18 had no CAI use
prior to or during the study (Group A), 18 had previously used CAIs
and continued to do so during the study (Group B) and 20 had no
history of CAI use but began these medications at the start of the
study (Group C). All patients underwent functional [best corrected
visual acuity (BCVA)] and structural [macular cystic cavity volume
(CCV) calculated from spectral domain optical coherence tomography]
evaluations at baseline, 6, 12 and 18 months, and Group C patients
underwent additional exams at 1 and 3 months after starting CAI
therapy.
There were no significant differences in BCVA or
CCV within each group at subsequent evaluations compared with
baseline values. Comparison of Group C with Groups A and B at each
follow up examination also showed no statistically significant
differences in BCVA or CCV, although there was a suggestion of
improved visual acuity in Group C earlier in the study. Researchers
conclude that these results demonstrate that XLRS is stable over an
18-month time period and that topical CAI use was not associated
with improvement in visual function or macular cyst volume at one
year. However, they also noted that some individuals treated with
CAIs demonstrated notable improvements at earlier time points,
suggesting that these medications may have more nuanced
effects.
“There are limited data available on the natural
course of XLRS, or the impact of using CAIs on disease progression,
likely because XLRS is a rare condition,” said Sue Washer,
President and CEO of AGTC. “This lack of information is one hurdle
to overcome as we develop new treatment approaches. We believe
these study results are an important advance in our understanding
of the natural progression of XLRS, and will enhance our efforts to
develop our AAV-based XLRS gene therapy candidate, which is
currently being evaluated in a Phase 1/2 clinical trial.”
AGTC is currently enrolling patients in a
clinical trial for its XLRS product candidates, as part of the
company's collaboration with Biogen. Patients and caregivers
interested in participating in or learning more about this trial
may learn more at www.agtc.com/patients-and-caregivers or by
contacting 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 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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