Adverum Biotechnologies Announces 3-Year Efficacy and Safety Results from the OPTIC Extension Study in Patients with Wet AMD at AAO 2023
November 04 2023 - 4:54PM
Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage
company that aims to establish gene therapy as a new standard of
care for highly prevalent ocular diseases, today announced updated
data from the OPTIC extension study of patients with wet AMD during
the Retina Subspecialty Day at the American Academy of
Ophthalmology (AAO) 2023 Annual Meeting in San Francisco,
California. A summary of previously announced aflibercept protein
levels from the LUNA study was also presented.
“We are pleased to be leading innovation for patients with wet
AMD through the development of a treatment option that has the
potential to be safe, efficacious and much less burdensome for
patients,” stated Laurent Fischer, M.D., president and chief
executive officer of Adverum Biotechnologies. “These efforts are
now supported by the most mature dataset in wet AMD IVT gene
therapy, with the OPTIC safety and efficacy data now sustained out
to 3 years. Ixo-vec demonstrated a robust improvement in outcome
measures while maintaining a favorable safety profile, particularly
at the 2E11 dose, which we have advanced to our Phase 2 LUNA trial.
We are particularly encouraged by the continuous and consistent
aflibercept protein levels observed at up to 4.5 years
post-treatment. These data highlight the potential for patients to
experience life-long clinical benefit after a single IVT treatment
with minimal or no need for additional injections and showcase the
potentially transformative impact of gene therapy.”
OPTIC 3-year Extension Data Highlights
- An oral presentation
titled “ADVM-022 Intravitreal Gene Therapy for Neovascular AMD:
Preliminary Data from the Phase 2 LUNA Trial and 3-Year Results
From the Phase 1 OPTIC-Extension Trial” was presented today by Carl
D. Regillo, M.D. FACS at the AAO 2023 Annual Meeting.
- Patients in the
OPTIC extension trial continue to experience long-term benefit from
Ixo-vec through 3 years of follow-up, including maintenance of
vision, durability of anatomical improvements and sustained
reduction in anti-VEGF treatment burden.
- Patients at the 2E11
dose had an 84% reduction in annualized anti-VEGF injections, with
53% of the participants at the 2E11 dose receiving no supplemental
injections through three years.
- Aflibercept protein
levels have been sustained through follow-up, which is up to 4.5
years post-treatment.
- BCVA was maintained
and CST was improved through 3 years.
- Ixo-vec was
generally well tolerated with dose-dependent inflammation that was
responsive to topical steroids.
LUNA Aflibercept Protein Data Highlights, Baseline
Characteristics and Clinical Program Milestones
- Aflibercept protein data suggest
Ixo-vec at the 2E11 and 6E10 doses deliver similar levels of
aflibercept that are both within the therapeutically active range
based on OPTIC and non-human primate data.
- The LUNA trial population has
comparable injection frequency and other baseline characteristics,
with slightly better visual acuity and fluid control, compared to
the study population of OPTIC.
- In particular, the mean annualized
anti-VEGF injection rate in the 12 months prior to Ixo-vec
treatment in LUNA is 9.9 injections, indicating that these subjects
require frequent injections, administered approximately every six
weeks.
- Clinical Program Milestones:
- Q4 2023 - LUNA preliminary efficacy and
safety data.
- Mid-2024 - Additional LUNA data,
including the 26-week interim analysis.
“The long-term follow-up data from the OPTIC trial continue to
affirm that Ixo-vec offers a potentially transformational treatment
for wet AMD,” commented Carl Regillo, M.D., F.A.C.S., Chief of the
Retina Service at Wills Eye Hospital and presenter of the data at
the AAO Annual Meeting. “A favorable benefit-risk profile resulting
in an 84% reduction in annualized anti-VEGF injections was
demonstrated in participants receiving the 2E11 dose, with 53% of
patients continuing to be free from supplemental injections at 3
years after a single IVT injection. Taken together, the efficacy
and safety at the 2E11 dose in the OPTIC trial underscore Ixo-vec's
potential as a promising treatment option for wet AMD patients
addressing real-world unmet needs. I look forward to learning more
from the ongoing Phase 2 LUNA trial.”
About Wet Age-Related Macular Degeneration
Wet AMD, also known as neovascular AMD or nAMD, is a VEGF driven
advanced form of AMD affecting approximately 10% of patients living
with AMD associated with the build-up of fluid in the macula and
the retina. Wet AMD is a leading cause of blindness in people over
65 years of age, with approximately 20 million individuals
worldwide living with this condition. New cases of wet AMD are
expected to grow significantly worldwide as populations age. AMD is
expected to impact 288 million people worldwide by 2040, with wet
AMD accounting for approximately 10% of those cases. Additionally,
wet AMD is a bilateral disease, and incidence of nAMD in the second
eye is up to 42% in the first 2 to 3 years. The current standard of
care requires frequent life-long repeated bolus injections of
anti-VEGF in the eye. IVT gene therapy has the promise to preserve
vision and reduce most or all injections for the life of the
patient by delivering stable therapeutic levels of anti-VEGF to
control macular fluid.
About Ixo-vec in Wet AMD
Adverum is developing ixoberogene soroparvovec (Ixo-vec,
formerly referred to as ADVM-022), its clinical-stage gene therapy
product candidate, for the treatment of wet AMD. Ixo-vec utilizes a
proprietary vector capsid, AAV.7m8, carrying an aflibercept coding
sequence under the control of a proprietary expression cassette.
Unlike other ophthalmic gene therapies that require surgery to
administer the gene therapy under the retina (sub-retinal
approach), Ixo-vec is designed to be administered as a one-time IVT
injection in the physician’s office, deliver long-term efficacy,
reduce the burden of frequent anti-vascular endothelial growth
factor (VEGF) injections, optimize patient compliance and improve
vision outcomes for patients with wet AMD. In recognition of the
need for new treatment options for wet AMD, the U.S. Food and Drug
Administration granted Fast Track designation for Ixo-vec for the
treatment of wet AMD. Ixo-vec has also received PRIME designation
from the European Medicines Agency and the Innovation Passport from
the United Kingdom’s Medicines and Healthcare Products Regulatory
Agency for the treatment of wet AMD.
About Adverum Biotechnologies
Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage
company that aims to establish gene therapy as a new standard of
care for highly prevalent ocular diseases with the aspiration of
developing functional cures to restore vision and prevent
blindness. Leveraging the capabilities of its proprietary
intravitreal (IVT) platform, Adverum is developing durable,
single-administration therapies, designed to be delivered in
physicians’ offices, to eliminate the need for frequent ocular
injections to treat these diseases. Adverum is evaluating its novel
gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly
referred to as ADVM-022), as a one-time, IVT injection for patients
with neovascular or wet age-related macular degeneration.
Additionally, by overcoming the challenges associated with current
treatment paradigms for debilitating ocular diseases, Adverum
aspires to transform the standard of care, preserve vision, and
create a profound societal impact around the globe. For more
information, please visit www.adverum.com.
Forward-looking Statements
Statements 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 the potential benefits of Ixo-vec in the
treatment of wet AMD, the design of and enrollment in the LUNA
trial, including the prophylactic corticosteroid regimens, and
anticipated preliminary and interim data from the LUNA trial.
Actual results could differ materially from those anticipated in
such forward-looking statements as a result of various risks and
uncertainties, including risks inherent to, without limitation:
Adverum’s novel technology, which makes it difficult to predict the
timing of commencement and completion of clinical trials;
regulatory uncertainties; enrollment uncertainties; the results of
early clinical trials not always being predictive of future
clinical trials and results; and the potential for future
complications or side effects in connection with use of Ixo-vec.
Additional risks and uncertainties facing Adverum are set forth
under the caption “Risk Factors” and elsewhere in Adverum’s
Securities and Exchange Commission (SEC) filings and reports,
including Adverum’s Quarterly Report on Form 10-Q for the quarter
ended June 30, 2023 filed with the SEC on August 10, 2023. 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.
Inquiries:
Adverum Investor RelationsEmail: ir@adverum.com
Adverum Biotechnologies (NASDAQ:ADVM)
Historical Stock Chart
From Apr 2024 to May 2024
Adverum Biotechnologies (NASDAQ:ADVM)
Historical Stock Chart
From May 2023 to May 2024