Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ocular and rare
diseases, today announced clinical data for the first cohort of
patients (n=6) in the OPTIC phase 1 clinical trial of ADVM-022, the
company’s intravitreal injection gene therapy, in
treatment-experienced patients with wet age-related macular
degeneration (wet AMD). The data are being presented today by
Charles C. Wykoff M.D., Ph.D., director of research, Retina
Consultants of Houston, at the Atlantic Coast Retina Club Macula
20/20 Annual Meeting in New York, NY.
A copy of the presentation is available on the Adverum corporate
website under Events and Presentations in the Investors section,
available here.
In October 2019, Adverum presented data from the first cohort in
OPTIC at a median 34-week time point (28-44 week range). Today,
additional data for the first cohort are being presented, including
efficacy and safety data, with a median follow up of 44 weeks at a
range of 40-52 weeks, and included:
- Zero rescue injections for any patient. Additionally, the first
patient treated in OPTIC has reached 52 weeks post ADVM-022
administration.
- Vision was maintained as demonstrated by stable mean Best
Corrected Visual Acuity (BCVA) compared to baseline.
- Anatomical improvements were maintained as assessed by optical
coherence tomography (OCT) and central subfield thickness (CST)
measurements, compared to baseline.
As of December 1, 2019, ADVM-022 continues to be well-tolerated
in the first cohort with no drug-related or procedure-related
serious adverse events (SAEs), no drug-related systemic adverse
events and no adverse events meeting the criteria for dose-limiting
toxicities (DLTs). Low-grade inflammation was reported in all six
patients and was generally mild to moderate and responsive to
steroid eye drops. One ocular SAE, a retinal detachment, that was
not related to ADVM-022 or the administration procedure was
reported.
OPTIC Phase 1 Clinical Trial Data from Cohort 1
(n=6)
Results Following a Single ADVM-022 Dose: |
|
Median follow-up (weeks) |
44 |
Minimum/Maximum follow-up (weeks) |
40-52 |
Follow-up cutoff date |
December 1, 2019 |
|
|
Rescue Injections: |
|
Number of patients requiring anti-VEGF rescue injections |
0 patients |
Mean number of anti-VEGF rescue injections |
0 injections |
|
|
Change in BCVA1: |
|
Mean (ETDRS letters)3 |
-1.0 |
Minimum/Maximum (ETDRS letters) |
-7.0 / +7.0 |
|
|
Change in CRT2: |
|
Mean (mm)3 |
-25.5 |
Minimum/Maximum (mm)3 |
-117 / +32 |
|
|
Safety: |
|
Dose-limiting toxicities (DLTs) |
0 |
Serious adverse events (SAEs)4 |
1 |
1 Best corrected visual acuity (BCVA) as measured by Early
Treatment Diabetic Retinopathy Study (ETDRS) (i.e., sight charts) 2
Central retinal thickness (CRT), also referred to as central
subfield thickness (CST) assessed using Optical Coherence
Tomography (OCT) imaging and measured by an independent Central
Reading Center3 BCVA and CST values for patient with retinal
detachment (unrelated to study treatment) used last observations
prior to detachment 4 This event was deemed unrelated to
ADVM-022 or any study procedure
“These longer-term follow-up data demonstrate that patients in
this first cohort of OPTIC are achieving sustained benefits from
ADVM-022, a one-time intravitreal therapy, and have not required
any anti-VEGF rescue injections through a median of 44 weeks while
demonstrating impressive anatomic improvements,” said Charles C.
Wykoff M.D., Ph.D., director of research, Retina Consultants of
Houston and associate professor of clinical ophthalmology, Blanton
Eye Institute, Houston Methodist Hospital and Weill Cornell Medical
College, Houston Texas. “With a median follow-up period of 44
weeks, ADVM‑022 continues to control wet AMD disease activity in
all 6 patients and the low-grade intraocular inflammation appears
manageable with steroid eyedrops. Based on the data to date,
ADVM-022 has the potential to be a meaningful and potentially
transformative treatment for patients with wet AMD.”
Aaron Osborne, MBBS, chief medical officer of Adverum, added,
“These new clinical data are promising as they continue to support
the safety, efficacy, and durable clinical profile of ADVM-022 and
this therapy’s potential to change the treatment paradigm for
patients with wet AMD. Anti-VEGF injections, the current standard
of care, carry a significant treatment burden and real-world
outcomes data suggest that vision outcomes are suboptimal due to
undertreatment. In the first cohort of OPTIC, we continue to see
stable vision and anatomical improvements being maintained out to a
median of 44 weeks after a single ADVM-022 injection in these
difficult-to-treat patients who previously required frequent
anti-VEGF injections. We look forward to presenting longer-term
data from the first cohort and 24-week data from the second cohort
of OPTIC on February 8 at the Angiogenesis, Exudation, and
Degeneration 2020 symposium.”
About the OPTIC Phase 1 Trial of ADVM-022 in Wet
AMDThe multi-center, open-label, Phase 1, dose-escalation
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,
patients (n=6) received ADVM-022 at a dose of 2 x 10^11 vg/eye. In
the third cohort (n=9), patients also are receiving a dose of 2 x
10^11 vg/eye and in the fourth cohort (n=9), patients will receive
a dose of 6x10^11 vg/eye. Patients in the third and fourth cohorts
will receive prophylactic steroid eye drops instead of oral
steroids which were used in the first and second cohorts. The
primary endpoint of the trial is the safety and tolerability of
ADVM-022 after a single IVT administration. Secondary endpoints
include changes in best-corrected visual acuity (BCVA), measurement
of central retinal thickness (CRT), as well as mean number of
anti-VEGF rescue injections and percentage of patients needing
anti-VEGF rescue injections. Each patient enrolled will be followed
for a total of two years.
Eight leading retinal centers across the United
States (U.S.) 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 and reduce the burden of
frequent anti-VEGF injections, optimize patient compliance and
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 Wet Age-related Macular Degeneration (Wet
AMD)Age-related macular degeneration (AMD) is a
progressive disease affecting the macula, the region of the retina
at the back of the eye responsible for central vision. In patients
with wet AMD, an aggressive form of AMD, abnormal blood vessels
grow underneath and into the retina. These abnormal blood vessels
leak fluid and blood into and beneath the retina, causing vision
loss.
Wet AMD is a leading cause of vision loss in patients over 60
years of age, with a prevalence of approximately 1.2 million
individuals in the U.S. and 3 million worldwide. The incidence of
new cases of wet AMD in the U.S. is approximately 150,000 to
200,000 annually, and this number is expected to grow significantly
as the country’s population ages.
The current standard-of-care therapy for wet AMD is anti-VEGF
intravitreal injections. These are effective but typically require
eye injections every 4-12 weeks in order to maintain vision.
Compliance with this regimen can be difficult for patients,
caregivers, and healthcare systems, leading to undertreatment and
resulting in loss of vision.
About Adverum BiotechnologiesAdverum
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 to report additional clinical data for ADVM-022
from the OPTIC trial and to advance ADVM-022, including Adverum’s
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, and the
potential benefits of ADVM-022, 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 November 7, 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 Biotechnologies,
Inc.mlacy@adverum.com1-650-304-3892
Media:Cherilyn Cecchini, M.D.LifeSci
Communicationsccecchini@lifescicomms.com1-646-876-5196
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