Adverum Biotechnologies Doses First Patient in the ADVANCE Phase 1/2 Clinical Trial of ADVM-043 for A1AT Deficiency
December 28 2017 - 9:00AM
-- Company Expects to Report Preliminary Data in
the Second Half of 2018 --
Adverum Biotechnologies, Inc. (Nasdaq:ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in serious rare and
ocular diseases, today announced the dosing of the first patient in
the ADVANCE Phase 1/2 clinical trial of ADVM-043 for alpha-1
antitrypsin (A1AT) deficiency. The ADVANCE clinical trial is
designed to evaluate the safety and protein expression following a
single administration of ADVM-043, Adverum’s novel gene therapy
candidate.
“We are excited to dose the first patient in the ADVANCE trial,
which is an important achievement for Adverum as we are now in the
clinic developing a potential new treatment option for individuals
with A1AT deficiency,” said Amber Salzman, Ph.D., president and
chief executive officer of Adverum Biotechnologies. “We are working
diligently to develop ADVM-043 as a single-administration gene
therapy for individuals living with this disease.”
“Patients with A1AT deficiency need new therapeutics to prevent
the progression of this disease,” said Charlie Strange, M.D.,
professor of pulmonary and critical care medicine at the Medical
University of South Carolina (MUSC) and principal investigator for
the Alpha-1 Foundation Research Registry, the largest registry of
individuals with A1AT deficiency. “We are excited to be part of
this Phase 1/2 study evaluating ADVM-043, a gene therapy approach
that has demonstrated promising potential in preclinical
studies.”
About the ADVANCE Phase 1/2 Clinical Trial of ADVM-043
for A1AT deficiencyThe ADVANCE Phase 1/2 clinical trial is
a multi-center, open-label, dose-escalation study of ADVM-043 in
patients with A1AT deficiency. The study will include up to 20
patients across up to four dosing cohorts of up to 5 patients each.
The first cohort will receive an intravenous (IV) low dose of
ADVM-043 of 8E13 total vg (equivalent to approximately 1E12 vg/kg
based on an 80-kg patient). The next two cohorts will receive an
intermediate IV dose or high IV dose, with the fourth cohort
potentially evaluating intrapleural (IP) delivery of ADVM-043.
The study will be conducted at 5 leading centers in the United
States. The primary endpoint is safety and tolerability and
secondary endpoints include changes in plasma concentrations of
both total and M-specific A1AT levels. Adverum expects to report
preliminary data from this trial in the second half of 2018.
Additional information about this clinical trial can be found at
ClinicalTrials.gov under trial identifier number NCT02168686.
About ADVM-043ADVM-043 (AAVrh.10-A1AT) is a
gene therapy candidate that has the potential to induce stable,
long-term A1AT protein following a single administration. In a
preclinical proof-of-concept study, ADVM-043 demonstrated robust
protein expression above therapeutic levels in mice following
either IV or IP administration. In another study in non-human
primates, evidence of stable long-term expression of hA1AT mRNA was
observed out to one year following IP administration of
ADVM-043.
About Alpha-1 Antitrypsin (A1AT) DeficiencyA1AT
deficiency is a fairly common orphan disease impacting
approximately 100,000 individuals in the United States. It is
estimated that 20 million people carry the gene for the disease,
which unfortunately can be passed to their children. A1AT
deficiency is associated with premature emphysema. The disease is
caused by mutations in the SERPINA1 gene, resulting in very low
levels of A1AT. The current standard-of-care treatment for patients
with A1AT deficiency with lung disease can be challenging, with
weekly IV infusions of an alpha-1 proteinase inhibitor. The current
treatment regimen can result in underdosing and lead to worsening
lung function.
About Adverum Biotechnologies,
Inc.Adverum is a clinical-stage gene therapy company
targeting unmet medical needs in serious rare and ocular diseases.
Adverum has a robust pipeline that includes product candidates
designed to treat rare diseases alpha-1 antitrypsin (A1AT)
deficiency and hereditary angioedema (HAE) as well as wet
age-related macular degeneration (wAMD). Leveraging a
next-generation adeno-associated virus (AAV)-based directed
evolution platform, Adverum generates 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 and in-house
manufacturing expertise, specifically in process development and
assay development. For more information please visit
www.adverum.com.
Forward-Looking StatementsStatements contained
in this press release regarding matters that are not historical
facts 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 Adverum’s product candidates, clinical studies, regulatory
filings and the therapeutic and commercial potential of its product
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circumstances. Adverum may not consummate any of these plans or
these product, clinical development or regulatory goals in a timely
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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
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or fail to receive required regulatory approvals. Risks and
uncertainties facing Adverum are described more fully in Adverum’s
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Contact for Adverum:
Leone Patterson
Chief Financial Officer
650-665-7222
lpatterson@adverum.com
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