Adverum Biotechnologies Provides Update on Alpha-1 Antitrypsin Deficiency Program
October 14 2016 - 7:01AM
Adverum Biotechnologies, Inc. (Nasdaq:ADVM) today provided an
update on its development program for ADVM-043 (formerly
“ANN-001”), a novel gene therapy for Alpha-1 Antitrypsin (A1AT)
Deficiency, a rare genetic disorder that may result in serious
respiratory and liver disease. Adverum has decided to upgrade
the ADVM-043 manufacturing process by implementing its
proprietary baculovirus-based production system and plans to
transfer the third-party contract manufacturing for ADVM-043 to a
large-scale contract manufacturer. This upgrade is designed
to increase the production scale, and comply with industry
standards so that the same production process is used from clinical
trials through commercial stage. The company now expects to
begin enrolment of patients in the Phase 1/2 clinical trial for
ADVM-043 in the fourth quarter of 2017.
“Adverum has industry-leading capabilities in process
development and manufacturing, consisting of a
baculovirus-based AAV production system and state-of-the-art
purification technology to manufacture AAV vectors of various
serotypes. This allows us to deliver a turn-key large scale process
to third-party cGMP manufacturers. We plan to leverage our leading
manufacturing capabilities and upgrade our ADVM-043 manufacturing
process now, to prepare for both our anticipated clinical and
commercial product needs,” said Amber Salzman, Ph.D., Chief
Executive Officer of Adverum Biotechnologies. “Our goal is to
meet with the FDA to review our modified plans in the first quarter
of 2017, to allow us to move ahead with patient enrolment in our
planned Phase 1/2 clinical trial for ADVM-043 in the fourth quarter
of 2017. We are committed to advancing this novel gene therapy for
patients with A1AT deficiency, and are planning to be ready to
deliver this potential product through a robust commercial-ready
manufacturing process.”
The company’s other lead programs for wet age-related macular
degeneration (wAMD) and hereditary angioedema (HAE) remain on
target to initiate toxicology studies in the first half of
2017.
About Adverum’s Manufacturing
CapabilitiesAdverum’s industrialized manufacturing process
is based on a proprietary baculovirus expression system and
state-of-the-art purification technology. This process is highly
efficient and scalable, applicable to various serotypes, with
current production yields per run up to one hundred times greater
than those obtained using conventional AAV production systems. The
company has the capability to develop manufacturing processes up to
200L and transfer them turn-key to third party cGMP manufacturers.
Further scale up is expected to allow commercial-grade
material for diseases with large patient populations. In addition,
Adverum has also developed formulations that allow for the
production of highly concentrated purified vectors.
About Adverum Biotechnologies, Inc. Adverum is
a gene therapy company committed to discovering and developing
novel medicines that can offer life-changing benefits to patients
living with rare diseases or diseases of the eye who currently have
limited or burdensome treatment options. Adverum has a robust
pipeline that includes product candidates to treat wet AMD, A1AT
deficiency, and hereditary angioedema, among other diseases.
We are leveraging our next-generation adeno-associated virus
(AAV)-based directed evolution platform to generate product
candidates designed to provide durable efficacy by inducing
sustained expression of a therapeutic protein. Our focus on the
patient is supported by clinical development expertise and core
capabilities in vector optimization, process development,
manufacturing, and assay development. For more information
please visit www.adverumbio.com.
Forward-Looking Statements for Adverum
Biotechnologies Statements 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 Adverum’s plans,
potential opportunities, expectations, projections, goals,
objectives, milestones, strategies, product pipeline, the
sufficiency of its resources to fund the advancement of any
development program or the completion of any clinical trials, and
the safety, efficacy, and projected development timeline and
commercial potential of products under development, all of which
are based on certain assumptions made by us on current conditions,
expected future developments and other factors we believe are
appropriate in the circumstances. Adverum may not consummate any
plans or product development goals in a timely manner, or at all,
or otherwise carry out the intentions or meet the expectations or
projections disclosed in our 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, risks related to regulatory review of Adverum’s
development plans and planned clinical trials and the risk that
Adverum will not be able to successfully develop or commercialize
any of its product candidates. 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:
Tricia Truehart
Vice President
The Trout Group LLC
646-378-2953
ttruehart@troutgroup.com
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