AGTC Announces Completion of Enrollment of Phase 1 / 2 Clinical Study of Investigational Gene Therapy in Patients with X-link...
April 10 2018 - 9:30AM
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 completion of enrollment in a
clinical study of the company's gene therapy product candidate, in
collaboration with Biogen, for the treatment of x-linked
retinoschisis (XLRS). This multicenter study is designed to
evaluate the collaboration’s AAV vector expressing retinoschisin
(rAAV2tYF-CB-hRS1) in patients with XLRS caused by mutations in the
RS1 gene. Topline data are anticipated by Q4 2018 with the final
analysis at the twelve-month time point.
“There are currently no FDA approved treatment
options for XLRS, a leading cause of macular degeneration in young
men,” said Sue Washer, president and CEO of AGTC. “The completion
of enrollment in this AAV trial represents another significant
achievement in our gene therapy clinical development program- a
milestone that may improve the lives of individuals affected by
XLRS.”
The Phase 1/2 trial is an open-label, dose
escalation study designed to assess the safety and efficacy of
intravitreal administration of the AAV-based gene therapy in
approximately 27 patients diagnosed with XLRS caused by mutations
in the RS1 gene. Trial participants were enrolled sequentially in
four groups. Individuals in Groups 1, 2 and 3 received a low,
middle or high dose of the investigational study agent. Patients in
Group 4 received the maximum tolerated dose as determined by the
first three groups. In addition, a group of pediatric patients were
enrolled at the middle dose. Although the primary endpoint of this
study is designed to evaluate safety, efficacy will also be
measured by an evaluation of changes in visual structure, function
and quality of life.
XLRS is an inherited early onset retinal
degenerative disease and the leading cause of juvenile macular
degeneration in males. Characterized by abnormal splitting of the
layers of the retina, the disease begins early in childhood and
causes poor visual acuity in young boys which may materialize into
legal blindness in adulthood. The disease begins early in childhood
and affected boys typically have best-corrected visual acuity of
20/60 to 20/120 at initial diagnosis. Severe complications such as
retinal hemorrhage or retinal detachment occur in up to 40% of
patients, especially in older individuals.
For more information on AGTC and its pipeline of
AAV-based gene therapy candidates in rare disease, please visit
www.AGTC.com/products.
About AGTC
AGTC 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 (CNS), 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 StatementsThis
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.
IR/PR CONTACTS: David Carey (IR) or Tom
Vickery (PR) Lazar Partners Ltd. T: (212) 867-1768 or (646)
871-8482 dcarey@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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