Adverum Biotechnologies Announces Publication of Preclinical Long-Term Expression and Efficacy Data in Molecular Therapy
December 03 2018 - 8:30AM
Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ophthalmology and
rare diseases, today announced the publication of preclinical
long-term expression and efficacy data in Molecular Therapy, the
leading international journal for research on the development of
molecular and cellular therapeutics to correct genetic and acquired
diseases.
The publication entitled, “Preclinical evaluation of ADVM-022, a
novel gene therapy approach to treating wet age-related macular
degeneration,” reported the following:
- A single intravitreal administration of ADVM-022 in non-human
primates (NHPs) provided stable and robust intraocular expression
of aflibercept and resulted in high levels of aflibercept within
the retina and choroid where wet age-related macular degeneration
(wet AMD) occurs
- Robust aflibercept expression in retinal and choroidal tissues
obtained at 16 months post-administration demonstrated that cells
transduced with ADVM-022 may continue to generate aflibercept for
an extended period of time
- In a laser-induced choroidal neovascularization model in NHPs,
a single intravitreal administration of ADVM-022 13 months before
lasering prevented the occurrence of clinically relevant choroidal
neovascularization lesions, similar to animals that received a
bolus of intravitreal aflibercept (standard-of-care) at the time of
lesioning
- A single intravitreal administration of ADVM-022 may provide a
safe and effective long-term treatment option for wet AMD
The full online publication can be accessed at the following
link: https://doi.org/10.1016/j.ymthe.2018.11.003
Adverum recently received Fast Track Designation from the U.S.
Food and Drug Administration (FDA) and announced initiation of a
phase 1 trial evaluating ADVM-022 in patients with wet AMD.
About the OPTIC Phase 1 Trial of ADVM-022 in Wet
AMDThe multi-center, open-label, phase 1, dose-escalation
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. At least six leading retinal centers across the
United States are expected to participate in the phase 1 trial. The
trial is expected to enroll 18 patients and evaluate three doses of
ADVM-022; first dose: 6 x 10^11 vg/eye, second dose: 2 x
10^12 vg/eye, and third dose: 6 x 10^12 vg/eye. Patients
will be administered a tapering prophylactic corticosteroid
regimen. The primary endpoint of the trial is the safety and
tolerability of ADVM-022 at 24 weeks after a single IVT
administration. Secondary endpoints include changes in
best-corrected visual acuity (BCVA), measurement of central retinal
thickness (CRT), as well as mean number of rescue anti-VEGF
injections and percentage of patients needing rescue anti-VEGF
injections. Each patient enrolled is expected to be followed for a
total of two years. For further details about the trial, enrollment
and eligibility please contact adverumopticstudy@adverum.com or
visit https://clinicaltrials.gov/ct2/show/NCT03748784.
About ADVM-022 Gene Therapy Candidate in Wet
AMDAdverum’s gene therapy candidate, 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. AAV.7m8
was discovered by directed evolution and is a variant of AAV2. The
vector was designed with the purpose of improved transduction
efficiency to the retina when administered intravitreally. ADVM-022
is designed to provide potentially sustained therapeutic levels of
aflibercept and to minimize the burden of frequent anti-VEGF
injections.
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. Approximately 10% of patients living with AMD have
an advance form of the disease called wet AMD, in which blood
vessels begin to invade the cellular space between the layers of
cells in the retina. These new blood vessels are often leaky, which
results in fluid and blood in the retina and causes vision
loss.
Wet AMD is a leading cause of vision loss in subjects over 60
years of age. A significant number of individuals are impacted by
this disease, which has a prevalence of approximately 1.2 million
individuals in the U.S. 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 based on the country’s
aging population.
The current treatment regimen can be burdensome, as patients
generally require intravitreal injections with anti-VEGF proteins
every 4-12 weeks. Compliance with this regimen can be difficult for
patients and their caregivers, leading to compliance deficiencies
and loss of vision from under dosing of treatment.
About Adverum Biotechnologies, Inc.Adverum is a
clinical-stage gene therapy company targeting unmet medical needs
in ophthalmology and rare diseases. Adverum develops gene therapy
product candidates designed to provide durable efficacy by inducing
sustained expression of a therapeutic protein. Adverum has
collaboration agreements with Regeneron Pharmaceuticals to
research, develop, and commercialize gene therapy products for
ophthalmic diseases and Editas Medicine to explore the delivery of
genome editing medicines for the treatment of inherited retinal
diseases. 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 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
AAV.7m8 vector designed to have improved transduction efficiency to
the retina, a single intravitreal administration of ADVM-022
may provide a safe and effective long-term treatment option for wet
AMD, Adverum’s OPTIC Phase 1 clinical trial of ADVM-022, and
expected growth in the number of new cases of wet AMD in the U.S.,
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. Actual
results and 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 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, as well as risks and uncertainties facing
Adverum described more fully in Adverum’s periodic reports filed
with the Securities and Exchange Commission (SEC), especially under
the caption “Risk Factors” in its latest Quarterly Report on Form
10-Q filed with the SEC on November 8, 2018. As a result, you
should not place undue reliance on these forward-looking
statements. 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:
Katherine Bock
Vice President Investor Relations & Corporate Communications
Adverum Biotechnologies, Inc.
650-656-9347
kbock@adverum.com
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