Adverum Biotechnologies Doses First Patient in Third Cohort of OPTIC Phase 1 Clinical Trial of ADVM-022 Intravitreal Gene The...
October 24 2019 - 8:00AM
Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ocular and rare
diseases, today announced that the first patient was dosed in the
third cohort (n=9) of the ongoing OPTIC phase 1 clinical trial for
ADVM-022 for the treatment of neovascular or “wet” age-related
macular degeneration (wet AMD). Patients in this cohort are
receiving a single intravitreal injection of gene therapy candidate
ADVM-022 at a dose of 2 x 10 ^11 vg/eye.
“We are excited to report dosing the first patient in the third
cohort of OPTIC. This expansion of OPTIC will generate important
clinical data to support the further development of ADVM-022,” said
Aaron Osborne, MBBS, chief medical officer of Adverum. “Based on
the recently presented data from the first cohort of OPTIC,
which demonstrated a sustained response to a single injection of
ADVM-022 out to a median of 34 weeks, with no patient in the first
cohort requiring anti-VEGF rescue therapy, we believe that ADVM-022
has the potential to be a transformative treatment option for
patients with wet AMD.”
Dante Pieramici, M.D., co-director of the California Retina
Research Foundation, Managing Partner of The California Retina
Consultants and investigator in the OPTIC trial, said, “An
intravitreal gene therapy that can significantly reduce the number
of injections required to maintain vision would be welcomed by
patients with wet AMD as well as their caregivers and physicians.
I’m encouraged by the recently presented clinical data from the
first cohort of the OPTIC trial showing that the therapy was safe
and well tolerated with no rescue injections required in patients
who previously required frequent anti-VEGF injections to control
their wet AMD.”
About the OPTIC Phase 1 Trial of ADVM-022 in Wet
AMDThe multi-center, open-label, phase 1 trial is designed
to assess the safety and tolerability of a single intravitreal
(IVT) administration of ADVM-022 in patients with wet AMD who are
responsive to anti-vascular endothelial growth factor (VEGF)
treatment. In the first cohort, patients (n=6) received ADVM-022 at
a dose of 6 x 10^11 vg/eye and in the second cohort (n=6) patients
received ADVM-022 at a dose of 2 x 10^11 vg/eye. In the third
cohort (n=9), patients are receiving ADVM-022 at a dose of 2 x
10^11 vg/eye and in the fourth cohort (n=9), patients will receive
ADVM-022 at a dose of 6 x 10^11 vg/eye. Patients in the first and
second cohorts received prophylactic oral steroids, while patients
in the third and fourth cohorts will receive prophylactic steroid
eye drops. The primary endpoint of the trial is the safety and
tolerability of ADVM-022 after a single IVT administration.
Secondary endpoints include change in best-corrected visual acuity
(BCVA), change in central subfield thickness (CST) and macular
volume, as well as mean number of anti-VEGF rescue injections and
percentage of patients needing anti-VEGF rescue injections. Each
patient enrolled in the study will be followed for a total of two
years.
Eight leading retinal centers across the United
States are participating in the OPTIC phase 1 trial for
ADVM-022. For more information on the OPTIC phase 1 clinical
trial of ADVM-022 in wet AMD, please
visit https://clinicaltrials.gov/ct2/show/NCT03748784.
About ADVM-022 Gene TherapyADVM-022 utilizes a
propriety vector capsid, AAV.7m8, carrying an aflibercept coding
sequence under the control of a proprietary expression cassette.
ADVM-022 is administered as a one-time intravitreal injection,
designed to deliver long-term efficacy, reduce the burden of
frequent anti-VEGF injections, optimize patient compliance, and to
improve vision outcomes for wet AMD and diabetic retinopathy
patients.
In recognition of the need for new treatment options for wet
AMD, the U.S. Food and Drug Administration granted Fast Track
designation for ADVM-022 for the treatment of this disease.
Adverum is currently evaluating ADVM-022 in the OPTIC study, a
phase 1 clinical trial in patients 50 years and older with wet AMD.
Additionally, Adverum plans to submit an Investigational New Drug
Application for ADVM-022 for the treatment of diabetic retinopathy
to the U.S. Food and Drug Administration in the first half of
2020.
About Adverum Biotechnologies, Inc.Adverum
Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy
company targeting unmet medical needs for serious ocular and rare
diseases. Adverum is evaluating its novel gene therapy candidate,
ADVM-022, as a one-time, intravitreal injection for the treatment
of its lead indication, wet age-related macular degeneration. 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; the potential benefits of
ADVM-022: the expected timing of submitting an IND for diabetic
retinopathy, 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 achieve any of these in a timely
manner, or at all, or otherwise carry out the intentions or meet
the expectations 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 risks inherent to, without limitation: Adverum’s novel
technology, which makes it difficult to predict the time and cost
of product candidate development and obtaining regulatory approval;
the results of early clinical trials not always being predictive of
future results; the potential for future complications or side
effects in connection with use of ADVM-022; obtaining regulatory
approval for gene therapy product candidates; enrolling patients in
clinical trials; reliance on third parties for conducting the OPTIC
trial and vector production; and ability to fund operations through
completion of the OPTIC trial and thereafter. Risks and
uncertainties facing Adverum are described more fully in Adverum’s
Form 10-Q filed with the SEC on August 8, 2019 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:
Investors:Myesha LacyAdverum BiotechnologiesVice President,
Investor Relations and Corporate
Communicationsmlacy@adverum.com1-650-304-3892
Media:Cherilyn Cecchini, M.D.Account
Supervisorccecchini@lifescipublicrelations.com 1-646-876-5196
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