Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene
therapy company targeting unmet medical needs in ocular and rare
diseases, today announced the presentation of positive 24-week
clinical data from the first cohort of patients (n=6) treated with
a one-time intravitreal (IVT) dose of ADVM-022 in the OPTIC phase 1
clinical trial in wet age-related macular degeneration (wet AMD).
Patients treated in this cohort achieved vision maintenance and
improvements in retinal anatomy, with zero anti-VEGF rescue
injections required, after a one-time intravitreal dose of
ADVM-022, through week 24. These patients previously required
frequent anti-VEGF injections to control their wet AMD and to
maintain functional vision. ADVM-022 was safe and well tolerated.
These data are being presented on Thursday, September 12, 2019
at 7:41 a.m. BST by Szilárd Kiss, M.D. during a podium presentation
at the Retina Society 2019 Annual Meeting in London, UK. Adverum
will host a conference call and webcast beginning at 1:30 p.m. BST
(5:30 a.m. PT/8:30 a.m. ET) to review the data. Dr. Kiss’s
presentation slides will be available at the conclusion of the
company’s webcast presentation under the Events and Presentations
in the Investors section of the company’s website located at
www.adverum.com.
OPTIC Phase 1 Clinical Trial 24-week Data from First
Cohort (n=6)
Baseline Characteristics of Patients: |
First Cohort |
Dose of intravitreal injection ADVM-022 |
6 x 10^11 vg/eye |
Mean age |
79 years |
Mean number of years since diagnosis |
3.3 years |
Mean number of prior anti-VEGF injections |
35 injections (range 7-109) |
Mean number of anti-VEGF injections in 8 months prior to
screening |
6.2 injections |
Average annualized anti-VEGF injection frequency1 |
9.3 injections |
Mean BCVA2 study eyeApproximate Snellen equivalent |
65.8 letters20/50 |
Mean CRT3 study eye |
369 μm |
24-week Results Following One-time ADVM-022
Dose: |
First Cohort |
Rescue Injections: |
|
Number of patients requiring anti-VEGF rescue injections |
0 patients |
Mean number of anti-VEGF rescue injections |
0 injections |
|
|
Change in BCVA: |
|
Mean change in BCVA |
-2 letters |
Range change in BCVA |
-9.1 letters, +5.1 letters (90% CI) |
|
|
Change in CRT: |
|
Mean change in CRT |
-52.7 μm |
Range change in CRT |
-86.5 μm, -18.8 μm (90% CI) |
|
|
Safety: |
|
Serious adverse events (SAEs) |
0 |
Dose-limiting toxicities (DLTs) |
0 |
Systemic adverse events (AEs) potentially related to ADVM-022 |
0 |
Ocular AEs potentially related to ADVM-022 |
19 events in 6 patients:Mild (14)4Moderate (5)4
- Intermediate uveitis x2
- Vitreous cells
- Anterior chamber cells x2
|
1 |
Calculated based on number of anti-VEGF injections in past 8
months |
2 |
Best corrected visual acuity (BCVA) as measured by Early Treatment
Diabetic Retinopathy Study (ETDRS) (ie, sight charts) |
3 |
Central retinal thickness (CRT), also referred to as central
subfield thickness (CST), assessed using Optical Coherence
Tomography (OCT) imaging and measured by independent Central
Reading Center |
4 |
Common Terminology Criteria for Adverse Events v5.0, general
guidelines |
“Typically, patients with wet AMD require frequent anti-VEGF
injections to maintain vision, representing a substantial treatment
burden that often results in vision loss due to undertreatment. The
single largest unmet clinical need for these patients is for a
long-lasting anti-VEGF treatment,” said Szilárd Kiss, M.D., retinal
specialist, who is presenting the data at the Retina Society
meeting. “These data on ADVM-022 are compelling, as they
demonstrate for the first time that a one-time gene therapy
delivered by intravitreal injection has the potential to provide
sustained efficacy and transform the treatment paradigm for
patients with wet AMD.”
Aaron Osborne, MBBS, MRCOphth, chief medical officer of Adverum,
added, “We are excited that there were zero rescue injections
through 24 weeks in this cohort of patients, all of whom previously
required frequent injections to avoid losing vision. In addition to
being safe and well tolerated, a single injection of ADVM-022
resulted in sustained anatomical improvements and vision
maintenance. Given these positive results from OPTIC, we are
working with key stakeholders to continue development and seek
regulatory approval of ADVM-022 to meet our goal of delivering this
novel gene therapy candidate as soon as possible to patients with
wet AMD and diabetic retinopathy, our second indication for
ADVM-022. I’d like to thank the investigators, patients, and
caregivers for their ongoing participation in the OPTIC trial.”
Future Outlook – Planned Milestones
- On Friday, October 11, 2019 at 4:26 p.m. PT, Dr. Szilárd Kiss
will present 24-week data from the first cohort of the OPTIC trial
during a podium presentation at a late-breaking development session
during the Retina Subspecialty Day Program of the American Academy
of Ophthalmology 2019 Annual Meeting in San Francisco, CA.
- An update on further development plans for the OPTIC trial will
be provided in the fourth quarter of 2019.
- 52-week data from the first cohort of patients as well as
24-week data from the second cohort of patients in the OPTIC trial
will be presented in the first half of 2020.
- Submission of an investigational new drug application for
ADVM-022 in diabetic retinopathy is planned in the first half of
2020.
- Adverum expects to be able to occupy its new corporate
headquarters in Redwood City, CA by the end of this year, allowing
for the expansion of in-house process development capabilities to
the 1000-liter production scale.
Conference Call
InformationAdverum will host a conference call and webcast
to review the data presented on September 12, 2019 at 1:30 p.m. BST
(5:30 a.m. PT/8:30 a.m. ET). Individuals can participate in the
conference call by dialing 1-866-420-8347 (domestic) or
1-409-217-8241 (international), and referring to the “Adverum
Biotechnologies Conference Call.” The webcast will be accessible
under Events and Presentations in the Investors section of the
company’s website located at www.adverum.com. The archived audio
webcast will be available on Adverum’s website following the call
for 30 days.
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 a dose of 2 x 10^11 vg/eye due to the
robust preliminary anatomical response observed from patients in
the first cohort. 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 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 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 TherapyAdverum’s gene
therapy candidate for wet AMD and diabetic retinopathy, ADVM-022,
utilizes a proprietary 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. ADVM-022 is designed to deliver long-term
efficacy by significantly reducing the treatment burden of frequent
anti-VEGF injections and improving real-world vision outcomes for
patients with wet AMD and diabetic retinopathy.
In September 2018, Adverum received Fast Track designation for
ADVM-022 for the treatment of wet AMD from the FDA.
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. Disease
progression results in the death of retinal cells and loss of
vision. Wet AMD, also known as neovascular AMD, is an aggressive
form of AMD, affecting around 10-15% of patients living with AMD,
but accounting for approximately 90% of severe vision loss due to
the disease. In patients with wet 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
long-term eye injections every 4-8 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 Biotechnologies, Inc.Adverum is a
clinical-stage gene therapy company targeting unmet medical needs
in ocular and rare diseases. Adverum develops gene therapy product
candidates designed to provide durable efficacy by inducing
sustained expression of a therapeutic protein. Adverum’s core
capabilities include clinical development, novel vector discovery
and in-house manufacturing expertise, specifically in scalable
process development, assay development, and current Good
Manufacturing Practices quality control. 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 reporting clinical data; the
expected timing of submitting an IND for diabetic retinopathy; the
expected timing of occupying Adverum’s new facility; and the
expected growth in the incidence of wet AMD, 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 Lacy
Adverum Biotechnologies
Vice President, Investor Relations and Corporate Communications
mlacy@adverum.com
1-650-304-3892
Media:
Joshua R. Mansbach
Solebury Trout
jmansbach@troutgroup.com
1-646-378-2964
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