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
long-term follow-up data from the OPTIC study treating wet
age-related macular degeneration (wet AMD) during The Retina
Society Annual Meeting in Pasadena, California. New data presented
include final two-year safety and efficacy assessment of Ixo-vec
(ixoberogene soroparvovec, formerly referred to as ADVM-022),
including reduction in annualized anti-VEGF injections, clinically
relevant aflibercept protein levels and maintenance to improvement
of mean best-corrected visual acuity (BCVA) and central subfield
thickness (CST). OPTIC study participants could enroll in an
extension study for an additional three years for a total of five
years of follow-up.
“We are pleased to present our final two-year analysis from our
OPTIC trial of Ixo-vec for the treatment of wet AMD. Ixo-vec
demonstrated a robust treatment effect while maintaining a
favorable safety profile, particularly at the 2E11 dose which was
advanced to our Phase 2 LUNA trial,” stated Richard Beckman, M.D.,
chief medical officer of Adverum Biotechnologies. “We are
particularly encouraged by the continuous and consistent
aflibercept protein levels, through three years, as well as the
maintenance to improvement in BCVA and CST from baseline while
dramatically reducing the anti-VEGF treatment burden for patients.
The longer-term follow-up data from OPTIC further strengthens our
confidence in the design of the ongoing LUNA trial where we are
evaluating the 2E11 dose and a new, lower 6x10^10 (6E10) dose,
along with enhanced prophylactic steroid regimens.”
Data Highlights
- Ixo-vec was generally well tolerated with dose-dependent
inflammation that was responsive to topical steroids.
- All 2E11 participants were inflammation free and did not
require steroids to treat inflammation at the end of the
study.
- OPTIC trial participants had an 81% – 98% reduction in
annualized anti-VEGF injections, as well as continuous therapeutic
aflibercept protein levels demonstrated through three years in
extension study participants.
- 80% and 53% of the participants in the 6x10^11 (6E11) and
2x10^11 (2E11) dose groups, respectively, were supplemental
injection free over two years.
- BCVA and CST were maintained to improved through at least two
years at both the 6E11 and 2E11 dose levels.
“The complete data set from the OPTIC trial affirms that Ixo-vec
may offer a potentially transformational treatment for wet AMD,”
commented Carl Regillo, M.D., F.A.C.S., chief of retina services at
Wills Eye Hospital, and presenter of the data at The Retina
Society’s Annual Meeting. “A favorable benefit-risk profile
resulting in an 81% reduction in annualized anti-VEGF injections
was demonstrated in participants receiving the 2E11 dose regardless
of baseline neutralizing antibodies, 53% of whom were supplemental
injection free over two years. That is very promising and
meaningful to patients, physicians, and the overall healthcare
system. I look forward to serving as a LUNA Phase 2 investigator
and gaining a further understanding of the safety and efficacy
profile of Ixo-vec.”
Patient enrollment and dosing are currently underway in LUNA, a
double-masked, randomized, Phase 2 trial. Adverum expects to
conduct the trial at approximately 40 sites in the U.S. and Europe.
Up to 72 subjects will be randomized equally between the 2E11 dose
and new lower 6E10 dose and across four prophylactic steroid
regimens. Specific regimens being evaluated include topical
difluprednate (Durezol®), dexamethasone intravitreal implant
(Ozurdex®), or a combination of either topical Durezol® or IVT
Ozurdex® with oral prednisone, with the aim of establishing a
prophylactic regimen with minimal need for inflammation management
post prophylaxis.
About Wet Age-Related Macular Degeneration
Wet AMD, also known as neovascular AMD or nAMD, is an advanced
form of AMD, affecting approximately 10% of patients living with
AMD. Wet AMD is a leading cause of blindness in patients over 65
years of age, with a prevalence of approximately 20 million
individuals worldwide living with this condition. The incidence of
new cases of wet AMD is expected to grow significantly worldwide as
populations age. AMD is projected to impact 288 million people
worldwide by 2040, with wet AMD accounting for approximately 10% of
those cases.
About OPTIC Trial of ADVM-022 in Wet AMD
ADVM-022, ixoberogene soroparvovec (Ixo-vec), is Adverum’s
clinical-stage gene therapy product candidate being developed 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 has
the advantage of being administered as a one-time IVT injection in
the office and is designed to deliver long-term efficacy and reduce
the burden of frequent anti-VEGF injections, optimize patient
compliance, and improve vision outcomes for patients with wet
AMD.
The OPTIC trial is designed as a multi-center, open-label,
dose-ranging, safety, and efficacy trial of Ixo-vec in participants
with wet AMD who have demonstrated responsiveness to anti-VEGF
treatment. Patients in OPTIC are treatment-experienced, and
previously required frequent anti-VEGF injections to manage their
wet AMD and to maintain functional vision.
About LUNA Trial of Ixo-vec in Wet AMD
Ixoberogene soroparvovec (Ixo-vec) is Adverum’s clinical-stage
gene therapy product candidate being developed for the treatment of
wet AMD. Ixo-vec utilizes an engineered, 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 has the advantage of
being administered as a one-time IVT injection in the office and is
designed to deliver long-term efficacy and reduce the burden of
frequent anti-vascular endothelial growth factor (VEGF) injections,
optimize patient compliance, and improve vision outcomes for
patients with wet AMD.
The LUNA trial is a multicenter, double-masked, randomized,
parallel-group Phase 2 study evaluating two doses of Ixo-vec,
including the 2x10^11 vg/eye (2E11) dose and a new, lower 6x10^10
vg/eye (6E10) dose, in wet AMD. The trial will randomize up to 72
participants equally across two doses and four prophylactic steroid
regimens in 40 sites in the U.S. and Europe. The primary endpoints
include the mean change in best-corrected visual acuity (BCVA) from
baseline to one year and the incidence and severity of adverse
events.
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 research 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. By
overcoming the challenges associated with current treatment
paradigms for these 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 well as
the design of and patient enrollment in the ongoing LUNA trial
evaluating the 2x10^11 (2E11) dose and a new, lower 6x10^10 (6E10)
dose, along with enhanced prophylactic steroid regimens. 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, 2022 filed with the SEC on August 11, 2022. 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.
Corporate & Investor Inquiries
Anand ReddiVice President, Head of Corporate Strategy, External
Affairs and EngagementAdverum Biotechnologies, Inc.T:
650-649-1358E: areddi@adverum.com
Media
Megan TalonAssociate Director, Corporate CommunicationsAdverum
Biotechnologies, Inc.T: 650-649-1006E: mtalon@adverum.com
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