AGTC Announces Updated
Development Plan for its X-Linked Retinitis Pigmentosa Clinical Program,
Including Q1 2021 Start of Planned Phase 2/3 Trial
| Source:Applied Genetic Technologies Corporation
FDA feedback allows for forward program development
Company
reiterates that its favorable safety profile and its advanced
manufacturing and analytics capabilities enable rapid clinical
development
GAINESVILLE, Fla. and
CAMBRIDGE, Mass., July 22, 2020 (GLOBE NEWSWIRE) -- 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 next
steps in the clinical development of the Company’s potential treatment
of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR
gene following receipt of written feedback from the FDA. The revised
development plan, which includes immediate expansion of the current
trial in parallel with the planned Phase 2/3 trial, will be designed to
evaluate sustained efficacy across multiple measures of potential
benefit in patients with XLRP.
In lieu of an
in-person meeting likely due to limitations imposed by COVID-19, the FDA
provided comprehensive written feedback regarding the design and
execution of a registration trial and future regulatory submissions. The
Company continues to move forward as planned with manufacturing,
clinical site preparation and other activities to enable initiation of
the studies as quickly as possible.
“We are
pleased with the productive feedback from the FDA and are modifying our
development program based on their recommendations to advance our XLRP
gene therapy candidate,” said Sue Washer, President and CEO of AGTC.
“Based on data available to date, we believe we have the potential for a
best-in-class product when important factors such as visual sensitivity
improvements, BCVA and safety are considered, which could provide
meaningful benefit to patients with XLRP who today have no treatment
options.”
AGTC is expanding its ongoing Phase
1/2 trial immediately and plans to dose approximately 20 patients in two
masked dosing arms to collect additional functional data, including a
mobility test added as a supplemental endpoint. The Company expects to
begin dosing in Q4 2020.
For late stage
studies, the FDA has indicated in its written feedback, which is
consistent with how others in the XLRP gene therapy space are analyzing
data, that a change in visual sensitivity of 7 decibels or greater in at
least 5 loci would be clinically meaningful. AGTC has previously
reported visual sensitivity as a mean over an entire treated area, but
believes multiple patients already evaluated in the ongoing Phase 1/2
trial would meet the FDA’s definition. The Company’s revised Phase 2/3
trial design is also expected to include two masked active arms in
addition to a control group, with visual sensitivity as the primary
endpoint and several supplemental endpoints such as the mobility test.
AGTC expects to begin this trial in Q1 2021.
Further
information on the protocols for these trials including patient
numbers, timelines and corporate cash guidance will be provided in the
Company's 10K filing for the fiscal year ending June 30, 2020. In
addition, AGTC remains on track to provide multiple data readouts for
both its XLRP and ACHM clinical programs in the second half of 2020.
These readouts will include data from the two higher dose groups in the
XLRP Phase 1/2 trial.
About AGTC AGTC
is a clinical-stage biotechnology company developing genetic therapies
for people with rare and debilitating ophthalmic, otologic and central
nervous system (CNS) diseases. AGTC is a leader in designing and
constructing all critical gene therapy elements and bringing them
together to develop customized therapies that address real patient
needs. Initially focusing on ophthalmology, our goal is to preserve or,
hopefully, be able to improve vision in some cases. AGTC has active
clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM
CNGB3 & ACHM CNGA3). Our pre-clinical programs build on our
industry leading AAV manufacturing technology and expertise. AGTC is
advancing multiple important pipeline candidates to address substantial
unmet clinical need in optogenetics, otology and CNS disorders.
About X-linked Retinitis Pigmentosa (XLRP) 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. AGTC was granted U.S. Food and Drug Administration
(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.
Forward-Looking Statements This
release contains forward-looking statements that reflect AGTC's plans,
estimates, assumptions and beliefs, including statements regarding the
timing for and expected expansion of its XLRP clinical development
program, the timing for reporting data in its XLRP and ACHM clinical
programs, and its ability to enroll patients, effectively design and
successfully complete its ongoing clinical trials. Forward-looking
statements include information concerning possible or assumed
preclinical and clinical product development and regulatory progress,
future results of operations, financial guidance, business strategies
and operations, potential growth opportunities, potential market
opportunities, the effects of competition and the impact of the COVID-19
pandemic. 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; the direct and
indirect impacts of the ongoing COVID-19 pandemic on the Company’s
business, results of operations, and financial condition; 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 most recent annual or quarterly report and in
other reports AGTC has 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, AGTC assumes 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 Glenn Silver (PR) Lazar FINN Partners T: (212) 867-1768 or (646) 871-8485 [email protected] or [email protected]
BioFlorida is the voice of Florida's life sciences industry, representing 8,600 establishments and research organizations in the BioPharma, MedTech, Digital Health and Health Systems that collectively employ nearly 107,000 Floridians. Source: TEConomy/BIO (released 2022)
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