Adverum Biotechnologies Announces IND Active for ADVM-022, a Novel Gene Therapy to Treat Wet Age-related Macular Degeneration...
August 29 2018 - 4:30PM
Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in serious rare and
ocular diseases, today announced its Investigational New Drug (IND)
application is active for the planned multi-center, open-label,
Phase 1, dose-escalation study of ADVM-022, a novel gene therapy
candidate for the treatment of wet age-related macular degeneration
(wAMD).
“We are excited to have this IND active for
ADVM-022, currently the only intravitreal gene therapy candidate
entering the clinic for patients with wet AMD,” said Leone
Patterson, interim president and chief executive officer of Adverum
Biotechnologies. “We have partnered with leading retinal
specialists in the U.S. and look forward to initiating the new
OPTIC clinical trial in the fourth quarter of this year.”
“We are pleased that we have been able to advance
this unique, single dose treatment into the clinic,” said Mehdi
Gasmi, Ph.D., chief science and technology officer of Adverum
Biotechnologies. “Based on our IND enabling studies and animal data
presented earlier this year at ASGCT, ADVM-022 has the potential to
greatly improve the quality of life for patients living with wet
AMD.”
About the OPTIC Phase 1 Trial of ADVM-022
in wAMDThe multi-center, open-label, Phase 1,
dose-escalation trial is designed to assess the safety and
tolerability of a single intravitreal (IVT) injection of ADVM-022
in patients with wAMD who are responsive to anti-vascular
endothelial growth factor (VEGF) treatment. 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 will
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 injection. Secondary endpoints include changes
in best-corrected visual acuity (BVCA) at 24 weeks, measurement of
central retinal thickness (CRT), and number of rescue aflibercept
injections. Each patient enrolled will be followed for a total of 2
years.
About ADVM-022 Gene Therapy
CandidateAdverum’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
injection. Excess VEGF activity can lead to wAMD progression and
vision loss and current anti-VEGF therapies need to be administered
frequently to patients (every 4-12 weeks). Reduced compliance with
the current approved regimen is associated with decreased efficacy.
Treatment with ADVM-022 is designed to provide potentially
sustained therapeutic levels of aflibercept and to minimize the
burden of frequent anti-VEGF injections.
In May 2018, long-term preclinical efficacy data on
ADVM-022 was presented at the American Society of Gene & Cell
Therapy 21st Annual Meeting. In this preclinical study in
non-human primate models of wAMD, the efficacy of ADVM-022 at 13
months post-administration was consistent with earlier reported
data, demonstrating that single intravitreal injection of ADVM-022
was found to be safe and statistically significant (p<0.0001) in
preventing the development of Grade IV lesions compared to the
untreated vehicle control group. ADVM-022 induced long-term
efficacy that was comparable to aflibercept, an anti- VEGF
standard-of-care therapy. In this preclinical study, ADVM-022 was
well-tolerated, with no serious adverse events.
In the same preclinical study, ADVM-022 induced
sustained intraocular expression of aflibercept for up to 16 months
following a single intravitreal injection. Robust levels of
aflibercept protein were detected up to 16 months in aqueous and
vitreous humor and, more importantly, in retina and choroid
tissues, where neovascularization occurs in wAMD.
About Wet Age-related Macular Degeneration
(wAMD)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 of the disease called wAMD, 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.
wAMD 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
wAMD 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 underdosing of treatment.
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 Statements
Statements contained in this press release regarding matters 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 expectations regarding the new
OPTIC clinical trial, including expected timing and structure, the
benefits ADVM-022 may have to patients with wAMD, and the
expectation that new cases of wAMD in the U.S. is expected to grow
significantly, all of which are based on certain assumptions made
by Adverum, expected future developments and other factors Adverum
believes are appropriate in the circumstances. Actual results may
differ from those set forth in these forward-looking statements as
a result of various risks and uncertainties, which include, without
limitation, the risk of unexpected delays in the enrollment of
patients in Adverum’s new OPTIC clinical trial or in the
manufacturing of ADVM-022 to be used in the OPTIC clinical trial,
as well as the 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 August 8, 2018. 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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