Adverum Biotechnologies Provides Update on OPTIC Phase 1 Trial for ADVM-022 in Wet AMD
May 16 2019 - 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 U.S. Food and Drug
Administration (FDA) has lifted the clinical hold on ADVM-022 for
the second cohort in the company’s OPTIC Phase 1 trial for wet
age-related macular degeneration (wet AMD), allowing dose
escalation to 2 x 10^12 vg/eye. This dose would be three
times higher than the dose of 6 x 10^11 vg/eye evaluated in the
first cohort. However, based on the robust preliminary anatomical
response observed to date in the first cohort (n=6), Adverum will
begin dosing the second cohort at a lower dose of 2 x 10^11 vg/eye,
three times lower than the dose used in the first cohort. The
dosing of patients in this second cohort is expected to begin in
June 2019. Adverum believes that ADVM-022 at a dose of 6 x 10^11
vg/eye has demonstrated the potential to provide sustained efficacy
following a single intravitreal injection.
As previously announced, the independent data
monitoring committee (DMC) unanimously voted to proceed with dose
escalation per protocol following their review of the safety data
from the first cohort. To date, no patient in the first cohort has
experienced a serious adverse event (SAE), with the first patient
completing the 24-week (6 month) assessment.
While the FDA has lifted the clinical hold on the
second cohort, ADVM-022 remains on partial clinical hold for dosing
patients in the third cohort, with the highest dose of 6 x 10^12
vg/eye. Adverum currently does not plan to dose at this highest
level, based on the robust preliminary anatomical response observed
in the first cohort of patients. Adverum will continue to work
closely with the FDA to resolve the remaining CMC comments. “The
preliminary results from the first cohort of patients in the OPTIC
trial are exciting. We look forward to presenting 24-week primary
and secondary outcomes from this cohort of patients at a scientific
meeting in the second half of this year,” said Leone Patterson,
chief executive officer of Adverum. “We are pleased that the FDA
and the DMC independently cleared us to dose escalate in the OPTIC
trial. Given the encouraging robust anatomical response from
patients in the first cohort, we will begin dosing patients in the
second cohort at a lower dose of 2 x 10^11 vg/eye, instead of dose
escalating as previously planned.”
About ADVM-022 Gene Therapy Candidate for
Wet AMD
Adverum’s gene therapy candidate for wet AMD,
ADVM-022, utilizes a proprietary vector capsid (AAV.7m8) carrying
an aflibercept coding sequence under the control of a proprietary
expression cassette and is administered as a single intravitreal
administration. ADVM-022 is designed to provide sustained
therapeutic levels of aflibercept, minimize the burden of frequent
anti-VEGF injections, and improve real- world vision outcomes for
patients with wet AMD. For more information on the OPTIC phase 1
clinical trial of ADVM-022 in wet AMD, please visit
https://clinicaltrials.gov/ct2/show/NCT03748784.
In September 2018, Adverum received Fast Track
designation for ADVM-022 in wet AMD from the FDA.
About Wet Age-related Macular Degeneration
(Wet AMD)
Age-related macular degeneration (AMD) is a
progressive disease affecting the retinal cells in the macula, the
region of the eye responsible for central vision. Disease
progression results in the death of retinal cells and the gradual
loss of vision. Wet AMD is an advanced form of AMD, affecting
approximately 10% of patients living with AMD. In patients with wet
AMD, abnormal blood vessels grow underneath and into the retina.
These abnormal blood vessels leak fluid and blood into and beneath
the retina, causing vision loss.
Wet AMD is a leading cause of vision loss in
patients over 60 years of age, with a prevalence of approximately
1.2 million individuals in the U.S. and 3 million worldwide. The
incidence of new cases of wet AMD in the U.S. is approximately
150,000 to 200,000 annually, and this number is expected to grow
significantly as the country’s population ages.
The current standard-of-care therapy for wet AMD is
anti-VEGF intravitreal injections. These are effective but
typically require long-term eye injections every 4-8 weeks in order
to maintain vision gains. Compliance with this regimen can be
difficult for patients, caregivers, and healthcare systems, leading
to suboptimal dosing and loss of vision from undertreatment.
About Adverum Biotechnologies,
Inc.
Adverum is a clinical-stage gene therapy company
targeting unmet medical needs in ocular and rare diseases. Adverum
develops gene therapy product candidates designed to provide
durable efficacy by inducing sustained expression of a therapeutic
protein. The company has collaboration agreements with Regeneron
Pharmaceuticals and Editas Medicine. Adverum’s core capabilities
include clinical development, novel vector discovery and in-house
manufacturing expertise, specifically in scalable process
development, assay development, and current Good Manufacturing
Practices quality control. For more information, please visit
www.adverum.com.
Forward-looking Statements
Statements contained in this press release
regarding matters, 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 plans related to
proceeding to dose additional patients in Adverum’s OPTIC Phase 1
trial for ADVM-022, the safety of ADVM-022, and the potential
therapeutic and commercial potential of its product candidates, 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 consummate any of its clinical development, regulatory or
commercialization goals in a timely manner, or at all, or otherwise
carry out the intentions or meet the expectations or projections
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, without limitation,
the risk that Adverum’s resources will not be sufficient for
Adverum to conduct or continue planned development programs and
planned clinical trials, the risk of a delay in the enrollment of
patients in Adverum’s clinical studies or in the manufacturing of
products to be used in such clinical studies, the risk that Adverum
will not be able to successfully develop or commercialize any of
its product candidates and the risk that Adverum will be delayed in
receiving or fail to receive required regulatory approvals. Risks
and uncertainties facing Adverum are described more fully in
Adverum’s periodic reports filed with the SEC, especially under the
caption “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, except as required by law.
Investor and Media Inquiries:
Amy Figueroa, CFA
Investor Relations Consultant
afigueroa@adverum.com
650-823-2704
Adverum Biotechnologies (NASDAQ:ADVM)
Historical Stock Chart
From Apr 2024 to May 2024
Adverum Biotechnologies (NASDAQ:ADVM)
Historical Stock Chart
From May 2023 to May 2024