Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage
company pioneering the use of gene therapy as a new standard of
care for highly prevalent ocular diseases, today announced
preliminary safety and efficacy data from the ongoing LUNA Phase 2
trial in patients with wet age-related macular degeneration (AMD).
These data are being presented by Dr. Arshad Khanani today at the
47th Annual Meeting of the Macula Society. The presentation titled
“Ixoberogene soroparvovec (Ixo-vec) Intravitreal Gene Therapy for
Neovascular Age-Related Macular Degeneration: Preliminary Results
from the LUNA Phase 2 Study" is scheduled for 10:56am ET.
“Wet AMD is a leading cause of blindness in people over the age
of 65, requiring life-long anti-VEGF injections. Our goal with
Ixo-vec is to provide patients virtually injection-free management
of their wet AMD lasting years and potentially for life,” stated
Laurent Fischer, M.D., president and chief executive officer of
Adverum Biotechnologies. “We are pleased with the emerging
treatment profile of Ixo-vec, showing potential for best-in-class
efficacy and over 90% of subjects having no or minimal inflammation
with our go-forward local prophylactic regimen. Underscoring the
potential best-in-class profile of Ixo-vec, these early activity
results were seen in hard-to-treat patients, who received over 9
annualized injections in the year prior to receiving Ixo-vec. Going
into LUNA, we looked to replicate the robust clinical activity seen
in OPTIC, in which patients continued to see clinical benefit
through 3 years, with measured stable aflibercept through 4.5
years. We are proud to build upon our pioneering efforts with the
most mature dataset and longest follow-up of all wet AMD
intravitreal gene therapies currently in development.”
Dr. Fischer continued, “Going into LUNA, we also looked to
establish that an enhanced prophylactic regimen combined with
highly active doses of Ixo-vec would further enhance its long-term
benefit-risk profile. With these early data, we are pleased with
the promising profile of Ozurdex plus difluprednate which we expect
to be our go-forward regimen. We look forward to sharing the
maturing LUNA dataset with the goal of confirming the optimal
dose(s) and prophylactic regimen for pivotal studies and with the
plan to present our 26-week interim analysis in the middle of
2024.”
“Based on my experience as an investigator for over five years
across multiple clinical trials with Ixo-vec for wet AMD, it’s
promising to see the sustained disease control and a significant
treatment burden reduction after a single intravitreal injection of
Ixo-vec in previously treated patients with wet AMD,” said Arshad M
Khanani, M.D., M.A., FASRS, Managing Partner and Director of
Clinical Research at Sierra Eye Associates, Clinical Associate
Professor at University of Nevada, Reno and Principal Investigator
in LUNA trial. “Ixo-vec has the potential to shift the treatment
paradigm for patients with wet AMD and I look forward to working
with the Adverum team to advance Ixo-vec into pivotal studies.”
LUNA Trial Background and Baseline
Demographics
The LUNA trial is an ongoing double-masked, randomized, Phase 2
trial. 60 patients with wet AMD were enrolled equally across two
dose cohorts, 2E11 and 6E10 vg/eye. The study is designed to assess
optimized prophylactic regimens, with patients receiving one and/or
two locally administered corticosteroid regimens, with or without
oral prednisone.
The LUNA trial builds on our experience with the OPTIC study,
where landmark 2-year data was recently published in The Lancet’s
eclinicalmedicine.
The data cut-off for these data is November 15, 2023, except for
treatment burden reduction, which is of January 2, 2024.
LUNA Preliminary Efficacy and Safety Data
Summary
Both the 2E11 and 6E10 doses demonstrated maintenance of visual
and anatomic outcomes. Notably, both doses resulted in a
potentially best-in-class reduction in annualized anti-vascular
endothelial growth factor (VEGF) injections and the percentage of
patients remaining free of annualized injections, with data
trending similar to or better than the OPTIC study.
- Treatment Burden Reduction
- At 26 weeks, Ixo-vec demonstrated
annualized reduction in anti-VEGF injection rates of 90% (n=19) at
6E10 and 94% (n=20) at 2E11.
- At 26 weeks, Ixo-vec demonstrated
injection free rates of 68% (n=19) at 6E10 and 85% (n=20) at
2E11.
- Visual (BCVA) and Anatomic (CST)
Outcomes:
- Visual acuity was maintained at both
dose levels - mean BCVA change from baseline to last visit (95%
CI):
- 2E11: -1.7 (-4.5,
1.2)
- 6E10: +0.5 (-2.2, 3.3)
- Anatomic endpoints were maintained
at both dose levels - mean CST (μm) change from baseline to last
visit (95% CI):
- 2E11: -16.4 (-31.5, -1.3)
- 6E10: -7.9 (-30.9, 15.0)
- In a sub-group analysis of patients
with higher baseline CST, a greater reduction in CST was
demonstrated, indicating the robust efficacy potential of Ixo-vec
gene therapy.
- Safety
- Preliminary data suggest
corticosteroid prophylaxis optimization at both the 2E11 and 6E10
doses appears to result in improved inflammatory profiles in LUNA
as compared to OPTIC study results.
- Ixo-vec was generally
well-tolerated, and when present intraocular inflammation was
responsive to local corticosteroids.
- No Ixo-vec related serious adverse
events were reported.
- No episcleritis, vasculitis,
retinitis, choroiditis, vascular occlusion or hypotony were
reported.
- Preliminary data indicate that
select prophylactic regimens are outperforming others that do not
provide sustained prophylaxis.
- Early on in the
study, the company implemented a protocol amendment to augment the
Ozurdex containing regimens with a course of Durezol eye
drops.
- Preliminary data
indicate that the amended Ozurdex + Durezol regimen may be the
favorable prophylactic regimen for future pivotal studies.
- In this potential
“go-forward” regimen, the vast majority of patients had no
inflammation, with over 90% of these patients having no or minimal
inflammation. Oral corticosteroids showed no incremental
benefit.
Upcoming Ixo-vec Milestones
- LUNA 26-week interim
analysis expected mid-2024
- Continued FDA and
EMA formal and informal regulatory interactions
- Initiation of Phase
3 trial expected H1’25
Webcast Details
The live webcast will be accessible under Events and
Presentations in the Investors section of the company's website.
Listeners can register for the webcast via this webcast link.
Analysts wishing to participate in the question and answer session
should use this dial-in link. A replay of the webcast will be
available on the company’s website shortly after the conclusion of
the webcast.
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 two to three 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-VEGF, optimize patient
compliance and improve vision outcomes for patients with wet AMD.
In recognition of the need for new treatment options for wet AMD,
FDA granted Fast Track designation for Ixo-vec for the treatment of
wet AMD. Ixo-vec has also received PRIME designation from the EMA
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 as a
one-time IVT injection for the treatment of wet AMD, including its
potential to improve vision outcomes, the potential best-in-class
efficacy of Ixo-vec, and anticipated timing of preliminary and
interim data from the Phase 2 LUNA trial and initiation of a Phase
3 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 September 30, 2023 filed with the SEC on
November 9, 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 Relations
Email: ir@adverum.com
Adverum Biotechnologies (NASDAQ:ADVM)
過去 株価チャート
から 8 2024 まで 9 2024
Adverum Biotechnologies (NASDAQ:ADVM)
過去 株価チャート
から 9 2023 まで 9 2024