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 dosed the first
patient in the Company’s Phase 1/2 clinical trial evaluating the
safety and efficacy of an investigational AAV-based gene therapy
for the treatment of X-linked retinitis pigmentosa (XLRP). The
multicenter study will assess AGTC’s novel recombinant AAV vector
expressing a human RPGR gene (the rAAV2tYF-GRK1-RPGR) in patients
with XLRP. The patient is being followed by Dr. David Birch of
Retina Foundation of the Southwest and the surgery was performed by
Dr. Andreas Lauer of Oregon Health & Science University.
“Dosing the first patient in our Phase 1/2
clinical study is an important step forward in advancing a new gene
therapy in individuals with XLRP, a condition with no approved
treatment options,” said Sue Washer, president and CEO of AGTC. “We
are committed to advancing our clinical programs to deliver novel
gene-based therapies for inherited orphan diseases and would like
to express our deep appreciation to those patients participating in
this clinical trial and their contributions to finding a potential
treatment.”
The Phase 1/2 trial is an open-label, dose
escalation study designed to assess the safety and efficacy of
subretinal administration of the AAV-based gene therapy in
approximately 15 patients diagnosed with XLRP. Trial participants
will be enrolled sequentially in four groups. Individuals in Groups
1, 2 and 3 will receive a low, middle and high dose of the
investigational study agent, respectively. Patients in Group 4 will
receive the maximum tolerated dose as determined by the first three
groups. The primary focus of the study will be to assess the safety
of the vector through analysis of local (ocular) or systemic
treatment-emergent adverse events. Efficacy will be measured by
evaluation of changes in retinal structure, function and quality of
life.
Along with the XLRS program for which we
announced the completion of enrollment last week, the XLRP program
is part of AGTC’s collaboration with Biogen. Under the terms of the
collaboration, AGTC will receive a milestone payment of $2.5
million as a result of enrollment of the first patient in the XLRP
trial.
“This is an exciting clinical milestone for one
of the first potential treatments of XLRP,” said Stephen Rose,
Ph.D., chief scientific officer for the Foundation Fighting
Blindness (FFB). “The Foundation’s registry, My Retina Tracker,
which is supported in part by a grant from AGTC, continues to
provide invaluable support in identifying patients with XLRP, and
other inherited retinal diseases. It allows us to inform them of
clinical trials, such as this gene therapy in XLRP, that have the
potential to transform their lives.”
XLRP is an inherited condition that causes
progressive vision loss in boys and young men. Characteristics of
the disease include night blindness in early childhood and
progressive constriction of the visual field. In general, XLRP
patients experience a gradual decline in visual acuity over the
disease course, which results in legal blindness around the 4th
decade of life. Preclinical data in a canine model of XLRP caused
by mutations in the RPGR gene indicate that treatment with an
AAV-based gene therapy product slowed the loss of visual
function.
My Retina Tracker® is an online, confidential
patient registry for people affected by inherited retinal diseases.
The registry collects data with the intention of using the
aggregated knowledge to advance research and to help accelerate
clinical trial enrollment by providing a source of information
about people impacted by retinal diseases. The registry is free for
people affected by inherited orphan retinal diseases, and is
designed with state-of-the-art technology to protect user privacy.
To learn more about My Retina Tracker, visit
www.MyRetinaTracker.org or contact the registry’s coordinator at
coordinator@MyRetinaTracker.org.
AGTC was granted U.S. Food and Drug (FDA) orphan
drug designation in 2017, as well as European Commission orphan
medicinal product designation in 2016, for its gene therapy product
candidate to treat XLRP caused by mutations in the RPGR gene.
Orphan drug designation, covered by the U.S. Orphan Drug Act of
1983, is granted to drugs or biologics that treat a rare disease or
condition affecting fewer than 200,000 individuals. Products
receiving orphan drug designation are eligible to receive market
exclusivity for a period of seven years, an exemption from certain
taxes and user fees and additional clinical support from FDA.
For more information on AGTC and its pipeline of
AAV-based gene therapy candidates in rare disease, please visit
www.agtc.com/programs.
About AGTCAGTC is a clinical-stage
biotechnology company that uses a proprietary gene therapy platform
to develop transformational genetic therapies for patients
suffering from rare and debilitating diseases. Its initial
focus is in the field of ophthalmology, where it has active
clinical trials in X-linked retinoschisis (XLRS), X-linked
retinitis pigmentosa (XLRP), and achromatopsia (ACHM CNGB3 &
ACHM CNGA3). In addition to its clinical trials, AGTC has
preclinical programs in optogenetics, adrenoleukodystrophy (ALD),
which is a disease of the central nervous system, and otology. The
clinical-stage XLRS and XLRP programs, the discovery program in ALD
and two additional ophthalmology programs are being developed in
collaboration with Biogen. In addition to its product pipeline,
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. Information about the
Phase 1/2 clinical trial in XLRS can be found at ClinicalTrials.gov
under trial identifier number NCT02416622.
About Achromatopsia
(ACHM)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. Information about the Phase 1/2 clinical
trial in achromatopsia caused by CNGA3 can be found at
ClinicalTrials.gov under the trial identifier number NCT02935517,
while the Phase 1/2 clinical trial in achromatopsia caused by CNGB3
can be found under the trial identifier number NCT02599922.
About X-linked Retinitis Pigmentosa
(XLRP)XLRP is an inherited condition that causes boys to
develop night blindness by the time they are ten and progresses to
legal blindness by their early forties. Information about the Phase
1/2 clinical trial in XLRP can be found at ClinicalTrials.gov under
trial identifier number NCT03314207.
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: gene therapy is still novel with only a few approved
treatments so far; AGTC cannot predict when or if it will obtain
regulatory approval to commercialize a product candidate or receive
reasonable reimbursement; uncertainty inherent in clinical trials
and 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 September 13, 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.
Contact Info Received
IR/PR CONTACTS: David Carey (IR) or Tom
Vickery (PR)Lazar Partners Ltd.T: (212) 867-1768 or (646)
871-8482dcarey@lazarpartners.com or tvickery@lazarpartners.com
Corporate Contact:Bill SullivanChief Financial OfficerApplied
Genetic Technologies CorporationT: (617)
843-5728 bsullivan@agtc.com
Stephen PotterChief Business OfficerApplied Genetic Technologies
CorporationT: (617) 413-2754spotter@agtc.com
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