Adverum Biotechnologies Doses First Patient in Cohort 4 of OPTIC Phase 1 Clinical Trial of ADVM-022 Intravitreal Gene Therapy...
April 27 2020 - 8:00AM
Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ocular and rare
diseases, today announced that the first patient was dosed in
Cohort 4 of the ongoing OPTIC Phase 1 clinical trial for ADVM-022
for the treatment of wet age-related macular degeneration (AMD).
Patients in Cohort 4 (n=9) are receiving a single intravitreal
injection of gene therapy candidate ADVM-022 at a dose of 6 x 10
^11 vg/eye (same as Cohort 1) and are receiving steroid eye drop
prophylaxis for six weeks (same as Cohort 3).
David S. Boyer, M.D., senior partner,
Retina-Vitreous Associates Medical Group and adjunct professor of
ophthalmology with the University of Southern California/Keck
School of Medicine in Los Angeles said, “The current
standard-of-care requires patients with wet AMD to receive frequent
anti-VEGF injections to maintain their vision. A one-time treatment
such as ADVM-022, which, similar to standard of care, is
administered as an in-office intravitreal injection, could
transform the treatment paradigm for wet AMD, particularly at this
time when it is more important than ever to reduce the need for
frequent injections and clinic visits. The data demonstrated in
OPTIC have been positive and underscore the potential of ADVM-022
to be a long-lasting treatment option for patients.”
“We are pleased to have enrolled our first patient
in Cohort 4, furthering our execution of the OPTIC trial,” said
Aaron Osborne, MBBS, chief medical officer of Adverum. “Patients in
this Cohort are receiving the higher dose of ADVM-022, which has
demonstrated outstanding efficacy and durability in Cohort 1, as
has been presented. We believe that utilizing the higher dose of
ADVM-022 with the use of steroid eye drop prophylaxis, will further
support that our gene therapy candidate, ADVM-022, has the
potential to be an important treatment option for patients living
with wet AMD. Additionally, I’m grateful for the continued support
of our clinical trial sites as we all manage through the global
pandemic. Due to COVID-19, sites quickly implemented extra safety
precautions for patients and their staff, allowing us to proceed
with enrollment. It’s a pleasure to partner with investigators in
OPTIC who share our commitment to develop a novel
single-administration approach for treating patients with wet AMD
and we look forward to sharing data from all four cohorts later
this year.”
About the OPTIC Phase 1 Trial of ADVM-022
in Wet AMDThe multi-center, open-label, Phase 1,
dose-ranging trial is designed to assess the safety and
tolerability of a single intravitreal (IVT) administration of
ADVM-022 in patients with wet AMD who are responsive to
anti-vascular endothelial growth factor (VEGF) treatment. In Cohort
1, patients (n=6) received ADVM-022 at a higher dose of 6 x 10^11
vg/eye and in Cohort 2, patients (n=6) received ADVM-022 at a lower
dose of 2 x 10^11 vg/eye. In Cohort 3, patients (n=9) also received
a dose of 2 x 10^11 vg/eye and in Cohort 4, patients (n=9) are
receiving a dose of 6 x 10^11 vg/eye. Patients in Cohorts 3 and 4
receive prophylactic steroid eye drops instead of oral steroids
which were used in Cohorts 1 and 2. The primary endpoint of the
trial is the safety and tolerability of ADVM-022 after a single IVT
administration. Secondary endpoints include changes in
best-corrected visual acuity (BCVA), measurement of central retinal
thickness (CRT), as well as the need for anti-VEGF rescue
injections. Each patient enrolled will be followed for a total of
two years.
Ten leading retinal centers across the United
States (U.S.) are participating in the OPTIC Phase 1 trial for
ADVM-022. For more information on the OPTIC Phase 1 clinical trial
of ADVM-022 in wet AMD, please
visit https://clinicaltrials.gov/ct2/show/NCT03748784.
About ADVM-022 Gene
TherapyADVM-022 utilizes a propriety vector capsid,
AAV.7m8, carrying an aflibercept coding sequence under the control
of a proprietary expression cassette. ADVM-022 is administered as a
one-time intravitreal injection, designed to deliver long-term
efficacy and reduce the burden of frequent anti-VEGF injections,
optimize patient compliance and improve vision outcomes for wet AMD
and diabetic retinopathy patients.
In recognition of the need for new treatment
options for wet AMD, the U.S. Food and Drug Administration granted
Fast Track designation for ADVM-022 for the treatment of this
disease.
Adverum is currently evaluating ADVM-022 in the
OPTIC study, a Phase 1 clinical trial in patients 50 years and
older with wet AMD. Additionally, Adverum plans to initiate a Phase
1/2 clinical trial of ADVM-022 for the treatment of diabetic
retinopathy in the second half of 2020.
About Adverum Biotechnologies,
Inc.Adverum Biotechnologies (Nasdaq: ADVM) is a
clinical-stage gene therapy company targeting unmet medical needs
in serious ocular and rare diseases. Adverum is evaluating its
novel gene therapy candidate, ADVM-022, as a one-time, intravitreal
injection for the treatment of its lead indication, wet age-related
macular degeneration. For more information, please visit
www.adverum.com.
Forward-looking
StatementsStatements contained in this press release
regarding events or results that may occur in the future are
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995. Such statements include,
but are not limited to statements regarding: Adverum’s plans for
advancing ADVM-022; the potential benefits of ADVM-022: the
expected timing of submitting an IND for diabetic retinopathy, all
of which are based on certain assumptions made by Adverum on
current conditions, expected future developments and other factors
Adverum believes are appropriate in the circumstances. Adverum may
not achieve any of these in a timely manner, or at all, or
otherwise carry out the intentions or meet the expectations
disclosed in its forward-looking statements, and you should not
place undue reliance on these forward-looking statements. Actual
results and the timing of events could differ materially from those
anticipated in such forward-looking statements as a result of
various risks and uncertainties, which include risks inherent to,
without limitation: Adverum’s novel technology, which makes it
difficult to predict the time and cost of product candidate
development and obtaining regulatory approval; the results of early
clinical trials not always being predictive of future results; the
potential for future complications or side effects in connection
with use of ADVM-022; obtaining regulatory approval for gene
therapy product candidates; enrolling patients in clinical trials;
reliance on third parties for conducting the OPTIC trial and vector
production; and ability to fund operations through completion of
the OPTIC trial and thereafter. Risks and uncertainties facing
Adverum are described more fully in Adverum’s Form 10-K filed with
the SEC on March 12, 2020 under the heading “Risk Factors.” All
forward-looking statements contained in this press release speak
only as of the date on which they were made. Adverum undertakes no
obligation to update such statements to reflect events that occur
or circumstances that exist after the date on which they were
made.
Investor and Media Inquiries:
Investors:Myesha LacyAdverum Biotechnologies,
Inc.mlacy@adverum.com1-650-304-3892
Media:Cherilyn Cecchini, M.D.LifeSci
Communicationsccecchini@lifescicomms.com1-646-876-5196
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