Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ocular and rare
diseases, today announced new long-term data from the OPTIC
clinical trial of ADVM-022 single intravitreal (IVT) injection gene
therapy in patients requiring frequent anti-VEGF injections for
their neovascular or wet age-related macular degeneration (wet
AMD). Safety and efficacy data from patients followed for a median
of 88 and 68 weeks at the 2 x 10^11 vg/eye dose (for Cohorts 2
& 3, respectively) and 104 and 36 weeks at the 6 x 10^11 vg/eye
dose (for Cohorts 1 & 4, respectively) are being presented at
the Association for Research in Vision and Ophthalmology (ARVO)
2021 Virtual Meeting with a pre-recorded presentation uploaded on
April 16, 2021.
“The long-term OPTIC data show the potential for ADVM-022 to
offer disease modifying treatment for patients with wet AMD,” said
Laurent Fischer, M.D., chief executive officer of Adverum
Biotechnologies. “Patient safety is our absolute priority and
following the unexpected adverse event we reported this past week
in a patient treated with the 6e11 high dose in the INFINITY study
in diabetic patients with macular edema, we are unmasking the
INFINITY study in order to analyze all data available and monitor
every patient who has received our gene therapy. We are also
working closely with our data monitoring committee and scientific
advisors and conducting a thorough review of all the data from our
ADVM-022 program. We will report our findings as the analysis
progresses to inform next steps for development.”
Dr. Fischer continued, “In OPTIC, ADVM-022 with wet AMD has
demonstrated durability out to two years with the ease of a single,
in-office intravitreal injection. We believe that we are well
within the therapeutic window with the 2e11 dose with 60% of
patients supplemental injection free beyond one year. Additionally,
the aflibercept protein levels at the 2e11 dose were within the
modeled therapeutic range and sustained out to at least one year,
consistent with levels observed 4-6 weeks after an aflibercept
injection.” The data reported in this press release and the related
statements relate only to the OPTIC clinical trial evaluating
ADVM-022 gene therapy for the treatment of wet AMD.
Adverum reported new interim data from the OPTIC trial (March
10, 2021 cutoff date, n=30) that continue to demonstrate the
potential of ADVM-022 to greatly reduce the anti-VEGF injection
burden for patients with wet AMD:
- All ADVM-022-related ocular adverse events (AE) were mild (80%)
to moderate (20%) in OPTIC patients with wet AMD. No clinical or
fluorescein evidence of posterior inflammation
- No vasculitis, retinitis, choroiditis, vascular occlusions, or
endophthalmitis
- Inflammation when observed was mild and responsive to steroid
eye drops
- At 2 x 10^11 vg/eye dose, ocular inflammation was minimal and
responsive to steroid eye drops; 87% of patients (13/151) have
discontinued steroid eye drops
Cohort 3 Safety Data for 2 x 10^11 vg/eye
Dose:
- A photo
accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/b6f22004-1612-4316-a481-9ac63157b54f
- A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/cbe15798-cd87-444f-b81a-f989dd73fc4a
- A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/7e3ef263-ebd4-4a27-a783-b41515ed8bdd
- Durable expression of aflibercept following a single, in-office
IVT injection of ADVM-022, for both doses (2 x 10^11 vg/eye and 6 x
10^11 vg/eye)
- Maintained or gained vision (mean BCVA2)
- Maintained to improved retinal anatomy (mean CRT3)
- Majority of patients are supplemental anti-VEGF injection free:
- 60% of patients (9/151) injection free following 2 x 10^11
vg/eye dose beyond one year
- 73% of patients (11/151) required zero or one injection
following 2 x 10^11 vg/eye at one-year
- 87% of patients (13/154) injection free following 6 x 10^11
vg/eye dose
- A photo accompanying this announcement is available
at: https://www.globenewswire.com/NewsRoom/AttachmentNg/f20337ce-b5a4-45de-8895-404bcd79b843
- Substantial reduction in annualized anti-VEGF injection
frequency5 following ADVM-022 in patients who previously required
frequent injections:
- 85% reduction for 2 x 10^11 vg/eye
- 96% reduction for 6 x 10^11 vg/eye
- Robust sustained aflibercept expression levels within
therapeutic range was observed for both doses and reaching the top
of the dose response curve.
- A photo accompanying this announcement is available
at: https://www.globenewswire.com/NewsRoom/AttachmentNg/e83dc5f6-25da-4922-96f3-49e30b1c7811
OPTIC Clinical Trial Data:
Results Following a Single ADVM-022 Dose: |
Cohort 1 |
Cohort 2 |
Cohort 3 |
Cohort 4 |
Patients |
n=6 |
n=6 |
n=9 |
n=9 |
Median (Range) Follow-up Visit (Weeks) |
104(All) |
88(646 to 92) |
68(48 to 72) |
36(32 to 44) |
ADVM-022 Dose |
High dose6 x 10^11 vg/eye |
Low dose2 x 10^11 vg/eye |
Low dose2 x 10^11 vg/eye |
High dose6 x 10^11 vg/eye |
Prophylactic Steroid Regimen |
13-day oral |
13-day oral |
6-week eye drops |
6-week eye drops |
Supplemental Anti-VEGF Injection Use: |
|
Number of patients supplemental injection free |
6/6 patients |
3/6 patients |
6/9 patients |
7/9 patients |
Follow-up BCVA2 and
CRT3: |
|
|
All Patients |
All Patients |
Supp. IVT-free Patients 50% (3/6) |
All Patients |
Supp. IVT-free Patients67% (6/9) |
All Patients |
Supp. IVT-free Patients 78% (7/9) |
BCVA mean change from baseline (letters) |
-1.3 |
-1.5 |
-1.0 |
+1.4 |
+4.3 |
-0.2 |
-0.4 |
CRT mean change from baseline (μm) |
-8.7 μm |
-28.2 μm |
-30.3 μm |
-134.4 μm |
-181.7 μm |
-77.1 μm |
-77.3 μm |
1 |
All patients from Cohort 2 (n=6) and Cohort 3 (n=9) |
2 |
Best corrected visual acuity
(BCVA) |
3 |
Central retinal thickness
(CRT) |
4 |
All patients from Cohort 1 (n=6)
and Cohort 4 (n=9) |
5 |
Annualized rate (Before) =
(number of IVTs in 12 months prior to ADVM-022) / (days from the
first IVT in the past 12 months to ADVM-022 / 365.25)Annualized
rate (After) = (number of aflibercept IVTs since ADVM-022) /
(days from ADVM-022 to the last study follow-up / 365.25) |
6 |
A patient missed visits after
week 64 due to worsening of COPD and died of a severe pneumonia due
to lung malignancy at ~76 weeks |
Brandon Busbee, M.D., partner, Tennessee Retina Physicians, and
investigator in OPTIC, said, “I appreciate Adverum putting patient
safety first as they seek to thoroughly review the data from the
ADVM-022 program. I look forward to partnering with the company on
ADVM-022’s future development for patients with wet AMD.”
ARVO 2021
PresentationsPresentation Title: Phase
1 Study of Intravitreal Gene Therapy with ADVM-022
for Neovascular AMD (OPTIC
Trial)Session: AMD: Clinical research
- New Therapies and TechnologiesDate
and Time: May 3, 2021 from 4:30 PM to 6:00 PM
ETPresenter: Brandon G. Busbee,
M.D., partner, Tennessee Retina Physicians
Poster Title: Preclinical Evaluation
of ADVM-062, a Novel Intravitreal Gene Therapy for the Treatment of
Blue Cone MonochromacyADVM-062 is a one-time intravitreal (IVT)
gene therapy utilizing AAV.7m8 to provide cone-specific expression
of human
L-opsin. Session: Drug Delivery and
Gene Therapy Date and Time: May 3,
2021 from 11:15 AM to 1:00 PM
ETPresenter: Ruslan Grishanin, director
translational science, Adverum Biotechnologies
These data presentations are available to ARVO participants
and are posted on the Publications section of the
Adverum’s website. Adverum is focused on conducting a thorough
review of data from the ADVM-022 program in the context of the
recent unexpected adverse event in the ADVM-022 INFINITY DME study
and is therefore canceling its webcast to review these new OPTIC
data, which was previously scheduled for Sunday, May 2, 2021.
About the OPTIC Phase 1 Trial of ADVM-022 in Wet
AMDThis multi-center, open-label, dose-ranging trial is
designed to assess the safety and tolerability of a single
intravitreal (IVT) administration of ADVM-022 in patients with wet
AMD. Patients in OPTIC are difficult-to-treat and had previously
received frequent anti-vascular endothelial growth factor (VEGF)
treatment. Patients received a 6 x 10^11 vg/eye of ADVM-022 in
Cohort 1 (n=6) and Cohort 4 (n=9) and patients received a 2 x 10^11
vg/eye of ADVM-022 in Cohort 2 (n=6) and Cohort 3 (n=9). Patients
in Cohorts 3 and 4 received six weeks of prophylactic steroid eye
drops rather than 13 days of prophylactic oral steroids which were
used in Cohorts 1 and 2. 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 the need for supplemental anti-VEGF
injections. Each patient enrolled will be followed for a total of
two years.
For more information, please visit
https://clinicaltrials.gov/ct2/show/NCT03748784.
About ADVM-022 Gene TherapyADVM-022 utilizes
Adverum’s propriety vector capsid, AAV.7m8, carrying a codon
optimized aflibercept coding sequence under the control of a
proprietary expression cassette. ADVM-022 is administered as a
one-time intravitreal injection (IVT), designed to deliver
long-term efficacy and reduce the burden of frequent anti-VEGF
injections, and improve real-world vision outcomes for patients
with wet age-related macular degeneration (wet AMD) and diabetic
macular edema (DME).
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 wet AMD.
About Wet AMDAge-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 worldwide1. 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 ages2,3.
The current standard-of-care therapies for wet AMD are anti-VEGF
proteins. These therapies can be burdensome, as patients generally
require chronic intravitreal (IVT) injection of anti-VEGF protein
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. It is estimated that these
standard-of-care branded anti-VEGF therapies used for the treatment
of wet AMD, DR, retinal vein occlusion, and other ocular diseases
generated in excess of $11 billion in sales worldwide in 20204.
1 Arch Ophthalmol. 2004;122(4):564-572.
doi:10.1001/archopht.122.4.564.2 Brown GC, Brown MM, Sharma S, et
al. The Burden of Age-Related Macular Degeneration: A Value-Based
Medicine Analysis. Transactions of the American Ophthalmological
Society. 2005.3 California Retina Consultants. Advances in Wet AMD.
Available at:
https://www.californiaretina.com/advances-in-wet-amd/4 Year-end
2020 financial statements from Regeneron, Roche, and Novartis.
About Adverum BiotechnologiesAdverum
Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy
company targeting unmet medical needs in serious ocular and rare
diseases. Adverum is advancing the clinical development of its
novel gene therapy candidate, ADVM-022, as a one-time, intravitreal
injection for the treatment of patients with wet age-related
macular degeneration and diabetic macular edema. For more
information, please visit www.adverum.com.
Forward-looking Statements Statements contained
in this press release regarding the 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
the potential for ADVM-022 in treating wet AMD and DME. Actual
results 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; and the potential for
future complications or side effects in connection with use of
ADVM-022. Risks and uncertainties facing Adverum are described more
fully in Adverum’s Annual Report on Form 10-K for the year ended
December 31, 2020 and any subsequent filings with the SEC 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 Relations Contacts
Myesha Lacy
Adverum Biotechnologies, Inc.
T: 650-649-1257
E: mlacy@adverum.com
Amy Figueroa
Adverum Biotechnologies, Inc.
T: 650-823-2704
E: afigueroa@adverum.com
Media Contact
Andrea Cohen
Sam Brown Inc.
T: 917-209-7163
E: andreacohen@sambrown.com
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