Applied Genetic Technologies Corporation (Nasdaq: AGTC), a
clinical stage biotechnology company focused on the development and
commercialization 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
updated interim three-month pediatric results and additional adult
safety results out to as long as 24 months from its ongoing Phase
1/2 dose escalation study of AGTC-401. AGTC-401 is
a recombinant AAV viral vector-based gene therapy targeting
mutations in the CNGB3 gene in patients with achromatopsia
(ACHMB3). Data from seven pediatric patients support previously
reported findings from adults treated with AGTC-401, which
demonstrated encouraging biologic activity and a favorable safety
profile. There were no new Suspected Unexpected Serious Adverse
Reactions (SUSARs) and for the three previously reported SUSARs,
the inflammation continues to improve with steroid treatment. Based
on the totality of the ACHMB3 data generated to date from 31
patients over as long as 24 months, AGTC plans to advance the
clinical development of AGTC-401 subject to consultation with the
U.S. Food and Drug Administration (FDA) at an End-of-Phase 2 (EOP2)
meeting in the first half of 2022.
The Company also reported updated results from a parallel study
of AGTC-402 targeting CNGA3 mutations in patients with
achromatopsia that are consistent with previously reported results
in adults, provide no indication of clinical improvements, and do
not support further clinical development. Most patients with CNGA3
mutations express a mutant protein that is not typically found in
patients with CNGB3 mutations, which the Company believes may have
impacted results seen in patients that received AGTC-402. AGTC will
continue to follow the ACHMA3 patients for long-term safety
observations.
“The three-month pediatric findings provide further evidence for
the strong potential of our product candidate for patients with
ACHMB3, and we look forward to continuing our discussions with the
regulatory agencies to determine the best path forward to bring
this important therapy to patients,” said Sue Washer, President and
Chief Executive Officer of AGTC. “We understand the challenges that
patients with achromatopsia face in their daily lives and our team
remains dedicated to advancing this program toward
commercialization. These additional data provide us the opportunity
to help inform the next phase of clinical development and we thank
the patients and investigators for their participation in this
important clinical trial.”
In the Phase 1/2 dose escalation study of AGTC-401 in ACHMB3
patients, a total of 21 adults were treated over a 100-fold dose
range in five groups and a total of ten pediatric patients were
treated at the three highest dose groups. The primary purpose of
any Phase1/2 clinical trial is to identify a well-tolerated dose
that provides clinical benefit to patients. The Company believes
that the data to date support that the 1.1E+12vg/mL dose is well
tolerated and provides clinical benefit in both adult and pediatric
patients.
“We’re pleased to have identified a generally safe and well
tolerated dose of AGTC-401 for both adults and pediatric patients
when inflammation is controlled,” said Dr. Robert Sisk, MD,
Director of Pediatric Vitreoretinal Surgery and Director of
Ophthalmic Genetics – Cincinnati Children’s Hospital and the
Cincinnati Eye Institute and an investigator in the ongoing AGTC
achromatopsia Phase 1/2 trials. “Based on the available pediatric
data at three months, which are consistent with previously reported
results in adults with ACHMB3, we are encouraged by the biologic
activity observed in patients treated with AGTC-401. The
achromatopsia community currently faces a significant unmet need
and the totality of this data reinforces the promise of AGTC-401 as
a potential treatment option if confirmed in future studies.”
As previously reported, in both the ACHMB3 and ACHMA3 trials,
treatment with the highest doses of AGTC-401 and AGTC-402,
respectively, led to three cases of severe ocular inflammation in
pediatric patients, which were reported as SUSARs. No new
additional SUSARs have been reported and the inflammation in all
previously reported SUSARs improved with an adjusted steroid
regimen. Two SUSARs (one in ACHMA3 and one in ACHMB3) have since
fully resolved and one (ACHMA3) continues to resolve, with all
three patients’ best corrected visual acuity returning to
baseline.
Secondary outcome measures evaluating efficacy were assessed by
standard visual function tests such as perimetry. We defined two
pediatric patients (17 and 7 years old) in the 1.1E+12 vg/mL dose
group as responders based on improvements in visual sensitivity.
Therefore, of the three adults and four children (total n=7) in the
1.1E+12 vg/mL dose group, four (>50%) are visual sensitivity
responders. These patients also had improvements in quality of life
as measured by a patient reported outcome survey developed
specifically for patients with achromatopsia.
The two other pediatric patients in the 1.1E+12 vg/mL dose group
and three pediatric patients ages 7 years and younger in the
3E+12vg/mL dose group (total n=5) could not sufficiently
concentrate and consistently complete the visual sensitivity
testing. Similar to other trials where endpoints are adapted for
young children, AGTC plans to work closely with clinicians and
regulators to develop potential adaptations for younger patients
for visual sensitivity testing.
The Company is currently working on an EOP2 meeting package for
the FDA and expects to receive feedback in the first half of 2022.
Subject to these discussions with FDA, the Company plans to
initiate the next phase of AGTC-401 clinical development. The
clinical program will be based on the favorable risk benefit
profile of the 1.1E+12 vg/mL dose, which had a generally safe and
well tolerated safety profile across all patients and signs of
biologic response based on improvements in visual sensitivity and
other measures of visual function.
Final data and analysis of both ACHMA3 and ACHMB3 studies will
be presented at relevant conferences and published for the
ophthalmology community.
Conference Call and Webcast
AGTC will host a conference call and webcast to review the
updated and previously reported interim study results today 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 Achromatopsia (ACHM)Achromatopsia (ACHM)
is an inherited condition caused by mutations in one of several
genes, with the two most common being mutations in either the CNGB3
or CNGA3 genes. ACHM is associated with extremely poor visual
acuity (most affected individuals are legally blind), extreme light
sensitivity resulting in daytime blindness, and complete loss of
color discrimination. AGTC is currently developing two separate AAV
gene therapy product candidates for the two most prevalent forms of
ACHM, caused by either a genetic mutation in the CNGB3 or CNGA3
genes. Together, these two genetic mutations account for up to 75%
of the ACHM patient population.
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 and ACHM CNGA3). 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 Otonomy, Inc., a
biopharmaceutical company dedicated to the development of
innovative therapeutics for neurotology, and Bionic Sight, LLC, an
innovator in the emerging field of optogenetics and retinal coding.
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 late-stage development program in
Achromatopsia (ACHM), including the potential for the clinical
development of AGTC-401 at the second highest dose, the continued
favorable safety profile, the timing of any discussions with the
FDA, and its ability to continue the clinical development of
AGTC-401. 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 our
most recent annual report on Form 10-K and 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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