Adverum Biotechnologies Provides 2019 Outlook
January 06 2019 - 8:00PM
Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ophthalmology and
rare diseases, today reviewed recent progress and provided an
outlook for 2019.
“For our lead gene therapy ADVM-022 for the treatment of wet
AMD, we are building off of last year’s momentum to execute our
ongoing OPTIC phase 1 clinical trial,” said Leone Patterson, chief
executive officer of Adverum Biotechnologies. “In a very short
period, we have dosed our first patient in the OPTIC phase 1 trial,
published long-term preclinical efficacy data in a leading
scientific journal, and received Fast Track designation for
ADVM-022. With this key groundwork complete, this year our primary
focus is on advancing this gene therapy for an initial indication
in wet AMD and evaluating additional anti-VEGF indications to
pursue. We have sufficient cash to fund operations at least through
the first half of 2020, including interim data from the three
cohorts in the OPTIC trial. Our team is excited to be working on
developing this novel single intravitreal injection therapy for
patients.”
Key Accomplishments for 2018
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In December
2018, long-term preclinical expression and efficacy data on
ADVM-022 in wet age-related macular degeneration (wet AMD) were
published in Molecular Therapy, a leading peer-reviewed scientific
journal. The data in this publication combined with two year
preclinical expression data presented in October 2018 at the
European Society of Gene and Cell Therapy (ESGCT) showed the
following: |
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- A single intravitreal injection of ADVM-022 in non-human
primates (NHPs) at dose ranges of 2 x 10 11 vg/eye to 2 x 10 12
vg/eye provided stable intraocular expression of aflibercept at
levels comparable with the levels measured in aflibercept
recombinant protein-injected eyes approximately 3 to 4 weeks
post-dose in all of the following: vitreous humor, aqueous humor,
retina and choroid
- A single intravitreal injection of ADVM-022 provided robust and
durable expression of aflibercept, sustained for approximately two
years post-dose in NHPs
- In a laser-induced choroidal neovascularization model in NHPs,
a single intravitreal injection 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 injection of ADVM-022 delivering a
continuous supply of aflibercept may provide an effective long-term
treatment option and prevent further vision loss for patients with
wet AMD
- The full online publication can be accessed at the following
link: https://doi.org/10.1016/j.ymthe.2018.11.003.
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In November
2018, Adverum dosed the first patient in the OPTIC phase 1 trial
evaluating a single intravitreal injection of ADVM-022 for patients
with wet AMD. ADVM-022 (AAV.7m8-aflibercept) is designed to provide
long-lasting therapy without the need of chronic or frequent
anti-VEGF injections |
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In
September 2018, Adverum received Fast Track designation for
ADVM-022 in wet AMD from the U.S. Food and Drug Administration
(FDA). Fast Track is a process designed to facilitate the
development and expedite the review of drugs to treat serious
conditions and fill an unmet medical need. Despite the availability
of anti-VEGF therapies, patients with wet AMD still have a
significant burden from the frequency of injections and
undertreatment may lead to vision loss |
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In late
August 2018, Adverum announced that the IND application for
ADVM-022 in patients went active |
2019 Outlook - Planned Pipeline
MilestonesADVM-022 for wet
AMD
- Provide an update on enrollment from the OPTIC phase 1 clinical
trial in the first half of 2019
- Provide interim data on the three cohorts from the OPTIC phase
1 clinical trial by the first quarter of 2020
Rare Disease Program
- Provide an update on rare disease program’s preclinical
development plan in the first half of 2019
Financial Guidance Adverum’s cash, cash
equivalents and marketable securities were $217.9 million as of
September 30, 2018. Adverum expects this quarter-end cash position
to fund operations at least through the first half of 2020.
Upcoming Events
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Adverum
plans to participate in the following upcoming conferences: |
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- J.P. Morgan’s 37th Annual Healthcare Conference in San
Francisco January 7-10, 2019. CEO Leone Patterson will present on
Thursday, January 10 at 8:00 am PT
- Leerink’s 8th Annual Global Healthcare Conference in New York,
February 27-March 1, 2019
- Cowen’s 39th Annual Health Care Conference in Boston, March
11-13, 2019
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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 StatementsStatements 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
Adverum’s plans for advancing ADVM-022 and the statements under the
headings “2019 Outlook - Planned Pipeline Milestones,” “Financial
Guidance” and “Upcoming Events”, 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 these 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
Form 10-Q filed with the SEC on November 8, 2018, particularly
under the heading “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.
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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