Adverum Biotechnologies Begins Patient Enrollment in the ADVANCE Phase 1/2 Clinical Trial for A1AT Deficiency
December 05 2017 - 9:00AM
-- Patients to Receive Single-administration of
Gene Therapy Candidate ADVM-043 ---- Company Expects to Report
Preliminary Data in the Second Half of 2018 --
Adverum Biotechnologies, Inc. (Nasdaq:ADVM), a leading gene therapy
company targeting unmet medical needs in serious rare and ocular
diseases, today announced the initiation of patient enrollment in
the ADVANCE Phase 1/2 clinical trial of ADVM-043 in patients with
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.
“Our team has been laser-focused on initiating the ADVANCE
clinical trial, which is an important milestone that transforms
Adverum into a clinical-stage company,” said Amber Salzman, Ph.D.,
president and chief executive officer of Adverum Biotechnologies.
“We are thrilled to collaborate with The Alpha-1 Foundation and
gain from their expertise and insight into patient needs. Given
their reach and robust Patient Registry, we are grateful for their
support in patient recruitment and look forward to working together
to develop a new treatment option for patients living with A1AT
deficiency.”
“Current approved therapy for A1AT deficiency patients with lung
disease is weekly IV plasma-derived protein replacement,” said
Henry Moehring, chief executive officer of the Alpha-1 Foundation.
“Until new and innovative therapies become available, the weekly
infusions unfortunately are lifelong for patients.”
“We are excited to see the ADVANCE clinical trial open for
patient enrollment to be able to evaluate ADVM-043 as a novel,
single-administration approach for treating A1AT deficiency,” added
Jean-Marc Quach, chief executive officer of The Alpha-1 Project
(TAP), a wholly-owned subsidiary of the Alpha-1 Foundation.
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. The Company 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 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 leading 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 and clinical studies and the
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 plans or product or clinical development 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. 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.
Contact for Adverum:
Leone Patterson
Chief Financial Officer
650-665-7222
lpatterson@adverum.com
Adverum Biotechnologies (NASDAQ:ADVM)
Historical Stock Chart
From Mar 2024 to Apr 2024
Adverum Biotechnologies (NASDAQ:ADVM)
Historical Stock Chart
From Apr 2023 to Apr 2024