Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ocular and rare
diseases, today announced new interim clinical data from the OPTIC
Phase 1 dose-ranging clinical trial of ADVM-022 intravitreal
injection gene therapy. OPTIC includes treatment-experienced
patients with wet age-related macular degeneration (AMD). The data
are being presented today by David S. Boyer, M.D., senior partner,
Retina-Vitreous Associates Medical Group and adjunct clinical
professor of ophthalmology with the University of Southern
California/Keck School of Medicine in Los Angeles, at the
Angiogenesis, Exudation, and Degeneration 2020 Annual Meeting in
Miami.
A copy of the presentation is available on the Adverum corporate
website under Events and Presentations in the Investors
section.
For the first time, data are being presented from patients in
cohort 2 (n=6) at 24 weeks following treatment with a single
intravitreal injection of a three-fold lower dose of ADVM-022 (2 x
10^11 vg/eye) compared to the cohort 1 dose (6 x 10^11
vg/eye). New data as detailed in the table below include:
- ADVM-022 demonstrated a robust efficacy signal and evidence of
a dose response:
- Cohort 1: 6 of 6 patients remain rescue-injection-free at
a median follow up of 50 weeks, with 3 patients at 52 weeks.
- Cohort 2: 4 of 6 patients remain rescue-injection-free at
24 weeks at the lower dose.
- In both cohorts combined, 10 of 12 (83%) patients remain
rescue-injection-free. For these patients:
- Vision was generally maintained as demonstrated by stable mean
best corrected visual acuity (BCVA) compared to baseline.
- Retinal anatomy improvements were achieved and maintained as
demonstrated by mean central subfield thickness (CST) compared to
baseline.
- ADVM-022 continues to demonstrate a favorable safety
profile and be well tolerated 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).
- ADVM-022-related adverse events (AEs) have been mild (71%) to
moderate (29%).
- Low-grade ocular inflammation was commonly reported and was
responsive to steroid eye drops.
OPTIC Phase 1 Clinical Trial Data:
Results Following a Single ADVM-022 Dose: |
Cohort 1 |
Cohort 2 |
Patients |
6 |
6 |
Dose ADVM-022 |
Higher Dose6 x 10^11 vg/eye |
Lower Dose 2 x 10^11 vg/eye |
|
|
|
Follow-up (median) |
50 weeks |
24 weeks |
Rescue Injections: |
|
|
Number of patients requiring anti-VEGF rescue injections |
0/6 patients |
2/6 patients |
Total anti-VEGF rescue injections |
0 injections |
6 injections |
Safety: |
|
|
Systemic adverse events |
0 |
0 |
Dose-limiting toxicities (DLTs) |
0 |
0 |
Serious adverse events (SAEs)1 |
1 |
0 |
Drug/procedure related SAEs |
0 |
0 |
|
|
|
Follow-up |
44 weeks (median) |
24 weeks |
Change in BCVA2: |
|
Full cohort |
Rescue-free patients |
Mean (ETDRS letters)3 |
-1.0 |
-4.8 |
-0.8 |
Range (ETDRS letters) |
-7 / +7 |
-19 / +16 |
-14 / +16 |
Change in CRT3: |
|
|
Mean (mm)4 |
-25.5 |
-27.8 |
-30.8 |
Range (mm)4 |
-117 / +32 |
-61 / -8 |
-61 / -8 |
1 |
This event (retinal detachment) was deemed unrelated to ADVM-022 or
any study procedure. |
2 |
Best corrected visual acuity (BCVA) as measured by Early Treatment
Diabetic Retinopathy Study (ETDRS) (i.e., sight charts). Data
through December 1, 2019 (Cohort 1). |
3 |
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 Center Data through December 1, 2019 (Cohort 1). |
4 |
BCVA and CST values for patient with retinal detachment (unrelated
to study treatment) used last observations prior to
detachment. |
|
|
“I am very encouraged that this difficult-to-treat patient
population enrolled in OPTIC is maintaining vision and anatomical
improvements for an extended period of time,” said David S. Boyer,
M.D., senior partner, Retina-Vitreous Associates Medical Group and
adjunct clinical professor of ophthalmology with the University of
Southern California/Keck School of Medicine in Los Angeles,
California. “Additionally, ADVM-022 continues to be safe and
well tolerated, with ocular inflammation that is manageable with
steroid eye drops. Patients with wet AMD and their caregivers carry
a significant treatment burden from the current standard-of-care
anti-VEGF injections, and real-world vision outcomes are suboptimal
due to undertreatment. ADVM-022 as a one-time intravitreal
injection therapy could transform the treatment paradigm for
patients and their caregivers.”
Aaron Osborne, MBBS, chief medical officer of Adverum, added,
“ADVM-022 has demonstrated a robust efficacy signal and evidence of
a dose response in the OPTIC Phase 1 trial with data from 12
patients and two doses now available. Patients in cohort 2 received
a three-fold lower dose of ADVM-022 than in cohort 1, and 4 of 6 of
these patients are rescue injection-free through 24 weeks, whilst
all 6 patients in cohort 1 remain rescue free with a median follow
up of 50 weeks. OPTIC is progressing well, with the key objectives
for cohorts 3 and 4 being to further evaluate dose response and to
assess a 6-week prophylactic course of steroid eye drops instead of
the 13-day oral steroid prophylaxis used in cohorts 1 and 2. We
look forward to presenting clinical data from all four cohorts of
OPTIC during this important year in the clinic for our novel gene
therapy, ADVM-022.”
KOL Discussion Tomorrow:In addition, Adverum
will host an event with expert retinal specialists to discuss the
OPTIC data presented at Angiogenesis and the potential opportunity
for ADVM-022. The discussion will be held on Sunday, February 9,
2020 beginning at 10:00 am EST. The event will be webcast live from
Adverum’s website at www.adverum.com in the Investors section under
the Events and Presentations page. A replay of the webcast will be
archived and available for replay following the event. A copy of
the slide presentation will also available on the Adverum corporate
website under Events and Presentations in the Investors
section.
About the OPTIC Phase 1 Trial of ADVM-022 in Wet
AMDThe multi-center, open-label, Phase 1, 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 who are responsive to anti-vascular endothelial growth factor
(VEGF) treatment. In cohort 1, patients (n=6) received ADVM-022 at
a higher dose of 6 x 10^11 vg/eye and in cohort 2, patients (n=6)
received ADVM-022 at a lower dose of 2 x 10^11 vg/eye. In cohort 3,
patients (n=9) also are receiving a dose of 2 x 10^11 vg/eye and in
cohort 4, patients (n=9) will receive a dose of 6 x 10^11 vg/eye.
Patients in cohorts 3 and 4 will receive prophylactic steroid eye
drops instead of 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 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
(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 in 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; the uncertainty of
obtaining regulatory approval for gene therapy product candidates;
potential delays in 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,
except as required by law.
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
Adverum Biotechnologies (NASDAQ:ADVM)
Historical Stock Chart
From Mar 2024 to Apr 2024
Adverum Biotechnologies (NASDAQ:ADVM)
Historical Stock Chart
From Apr 2023 to Apr 2024