AGTC Announces Completion of Enrollment in the Two Highest Dose Groups of its Ongoing Phase 1/2 Clinical Trial in Patients wi...
19 February 2020 - 11:00PM
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 completed enrollment
in the two highest dose groups of its Phase 1/2 clinical trial
evaluating the safety and efficacy of sub-retinal injection of
AGTC-501 for the treatment of X-linked retinitis pigmentosa (XLRP)
caused by mutations in the RPGR gene. The patients in these
additional groups received a higher or highest dose of AGTC’s XLRP
candidate.
“We expect that the information that will be
obtained from the two additional dose groups will help to reinforce
the data generated to date and previously reported in September
2019 and January 2020,” said Sue Washer, president and CEO of AGTC.
“We plan to report interim data from these two new dose groups and
to report top-line 12-month data for the first four dose groups in
the second half of 2020 and intend to initiate a pivotal trial by
the end of the year. Combined with the ongoing progress in our two
achromatopsia clinical studies, we expect to have multiple data
read-outs in 2020 that will build on the momentum we created in
January.”
AGTC most recently reported data from the
ongoing XLRP Phase 1/2 clinical trial in January 2020. Results at
the six-month time point in patients dosed centrally were
indicative of durable and meaningful improvements in central visual
sensitivity, encouraging improvements in Best Corrected Visual
Acuity and a favorable safety profile.
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 trial programs in X-linked retinitis pigmentosa (XLRP) and
achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its
clinical trials, AGTC has preclinical programs in optogenetics,
otology and adrenoleukodystrophy (ALD), frontotemporal dementia
(FTD) and amyotrophic lateral sclerosis (ALS), which are diseases
of the central nervous system (CNS), and other ophthalmology
indications. The optogenetics program is being developed in
collaboration with Bionic Sight. The otology program is being
developed in collaboration with Otonomy. 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 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 (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 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, financial guidance, 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 June 30, 2018, 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.
For AGTC
David Carey (IR) or Glenn Silver (PR)Lazar FINN
PartnersT: (212) 867-1768 or (646)
871-8485david.carey@finnpartners.com or
glenn.silver@finnpartners.com
Corporate Contact:Bill SullivanChief Financial
OfficerApplied Genetic Technologies CorporationT: (617)
843-5728bsullivan@agtc.com
Stephen PotterChief Business OfficerApplied
Genetic Technologies CorporationT: (617)
413-2754spotter@agtc.com
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