Applied Genetic Technologies Corporation (Nasdaq: AGTC), a
clinical stage biotechnology company focused on the development of
adeno-associated virus (AAV)-based gene therapies for the treatment
of rare and debilitating diseases with an initial focus on
inherited retinal diseases, today reported positive three-month
interim data from its ongoing Phase 2 Skyline trial of AGTC-501, a
recombinant AAV viral vector-based gene therapy targeting mutations
in the RPGR gene in patients with X-linked retinitis pigmentosa
(XLRP).
There were robust improvements in visual
sensitivity, the trial’s primary efficacy endpoint, in multiple
patients three months after dosing, with a 62.5% response rate in
dose group B and a 25% response rate in dose group A. This is well
above the statistically significant 50% response rate the Vista
Phase 2/3 trial for XLRP is powered to detect. The Company
considers a responder to be a patient who has an improvement in
visual sensitivity as assessed by microperimetry of at least 7
decibels (dB) in at least 5 loci. Importantly, for responders in
this trial, the number of loci that improved by at least 7 dB was
between 9 and 17. It is also notable that the mean visual
sensitivity of the entire macula increased and the area of the
macula with visual sensitivity also increased in the patients who
were responders. Although these results exceeded the current
standard set by the United States Food and Drug Administration
(FDA) that at least 5 loci increase by at least 7 dB, the improved
loci were not pre-specified, as also required in the FDA
standard.
There was a significant difference in visual
sensitivity as assessed by MAIA microperimetry in the treated eyes
compared with the untreated eyes of all Skyline responders at 3
months. In the Phase 1/2 trial, patients who were responders at 3
months remained responders at 12 months. Based on the durability of
the Phase 1/2 improvements in visual sensitivity, we believe the
results observed at 3 months in the Skyline trial will be
consistent at 12 months. Additional data related to the Skyline
trial is available on the AGTC website or by clicking here.
Consistent with the previously reported Phase 1/2
results, AGTC-501 was generally well-tolerated in this trial with
no clinically relevant safety concerns attributed to the study
agent. The majority of adverse events were non-serious and mild to
moderate in severity. The Company believes that the favorable
safety profile is largely the result of a strong focus on product
design, extensive pre-clinical testing, enhanced inclusion and
exclusion criteria, and surgical consistency.
“The meaningful response rate in visual sensitivity
seen in this interim Skyline data is very encouraging, as are the
favorable safety data,” said Dr. Robert Sisk, MD, FACS, FASRS,
Director of Pediatric Vitreoretinal Surgery and Director of
Ophthalmic Genetics – Cincinnati Children’s Hospital and the
Cincinnati Eye Institute and an investigator in the trial.
“Patients with XLRP have no FDA-approved options to treat this
devastating disease and we believe the results presented today show
great promise to provide outcomes that are truly meaningful and
potentially life-changing to patients.”
Skyline is a 14 patient Phase 2 expansion of the
Company’s Phase 1/2 clinical trial, in which patients are
randomized to either a high or low dose of AGTC-501 with the main
objective to evaluate the efficacy, safety and tolerability of
AGTC-501. The primary endpoint is the proportion of treated eyes
that demonstrate improvement from baseline in visual sensitivity at
12 months with secondary endpoints (also at 12 months) that include
improvements in best-corrected visual acuity (BCVA) and the
patient’s ability to navigate a mobility maze more successfully
under varying light and challenge conditions. The interim analysis
includes 13 male pediatric and adult patients that were treated
over a 10-fold dose range in two groups. The fourteenth patient had
not yet reached the three-month time point at the cut-off date for
the interim analysis. Patients in the Skyline trial were younger
than the patients in the comparable dose groups from the Phase 1/2
clinical trial with better BCVA and better mean visual sensitivity
at baseline than in the previously reported Phase 1/2 study. Given
the higher baseline BCVA, improvements in BCVA at three months were
less pronounced than in the Phase 1/2 clinical trial. More than
half of the patients, including four of the six responders in
visual sensitivity, showed improvement in the mobility maze based
on light levels passed, improved speed and/or reduced errors.
“We are incredibly excited by the compelling
interim results seen in the Skyline trial, including strong safety
data and robust improvements in visual sensitivity with a clear
difference between the two dose groups. These results add to the
growing body of evidence supporting the best-in-class potential of
AGTC-501 for the treatment of XLRP,” said Sue Washer, President and
Chief Executive Officer of AGTC. “We believe that if we achieve
similar results in the Vista Phase 2/3 clinical trial, we will have
a broad and compelling body of data to support the submission of a
BLA to the FDA, and to enable a differentiated and highly
competitive product profile.”
Conference Call and Webcast AGTC
will host a conference call and webcast to review the updated and
previously reported interim study results tomorrow, May 17, at 8:00
a.m. ET. To access the call, dial 877-407-6184 (US) or 201-389-0877
(outside of the US). A live webcast will be available in the Events
and Presentations section of AGTC’s Investor Relations site at
http://ir.agtc.com/events-and-presentations.
Please log in approximately 10 minutes prior to the
scheduled start time. The archived webcast will be available in the
Events and Presentations section of the Company's website.
About X-Linked Retinitis Pigmentosa
(XLRP)X-linked Retinitis Pigmentosa (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 or 5th decade of life.
About AGTCAGTC 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 with the potential to
address unmet patient needs. AGTC’s most advanced clinical programs
leverage its best-in-class technology platform to potentially
improve vision for patients with inherited retinal diseases. AGTC
has active clinical trials in X-linked retinitis pigmentosa (XLRP)
and achromatopsia (ACHM CNGB3). Its preclinical programs build on
the company’s industry leading AAV manufacturing technology and
scientific expertise. AGTC is advancing multiple important pipeline
candidates to address substantial unmet clinical needs in
optogenetics, otology and CNS disorders, and has entered strategic
collaborations with companies including Bionic Sight, an innovator
in the emerging field of optogenetics, and retinal coding and
Otonomy, Inc., a biopharmaceutical company dedicated to the
development of innovative therapeutics for neurotology. For more
information, please visit https://agtc.com/.
Forward-Looking
Statements This release contains forward-looking
statements that reflect AGTC's plans, estimates, assumptions and
beliefs, including statements about the potential of the company’s
gene therapy platform and the strength of interim results from the
Skyline Trial in XLRP, the potential of AGTC-501 as a treatment for
XLRP, the ability to use the interim Skyline results as a predictor
of the success of the final Skyline and Vista clinical trial
results and whether these results will support future regulatory
filings for AGTC-501. 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; risks and uncertainties related to funding
sources for our development programs; 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 report on Form
10-K, as it may be supplemented by subsequent periodic reports
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.
PR Contact:Kerry SinclairSpectrum
Science Communicationsksinclair@spectrumscience.com
Corporate Contact:Jonathan
LieberChief Financial OfficerApplied Genetic Technologies
CorporationT: (617) 843-5778jlieber@agtc.com
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