Adverum Biotechnologies Presents Positive End of Study

– Data demonstrating that Ixo-vec was usually nicely tolerated and {that a} single intravitreal (IVT) injection can result in steady and protracted aflibercept protein ranges by way of three years in contributors within the OPTIC extension research

– 81% – 98% discount in imply annualized anti-VEGF injections after a single IVT injection of Ixo-vec, together with 80% and 53% of contributors supplemental injection free over two years throughout the 6×10^11 (6E11) and 2×10^11 (2E11) doses, respectively, regardless of neutralizing antibodies

– Mean best-corrected visible acuity (BCVA) and central subfield thickness (CST) had been maintained or improved throughout each 6E11 and 2E11 doses

– All 2E11 contributors had been irritation free on the finish of the research

REDWOOD CITY, Calif., Nov. 04, 2022 (GLOBE NEWSWIRE) — Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage firm that goals to ascertain gene remedy as a brand new commonplace of take care of extremely prevalent ocular ailments, at the moment introduced long-term follow-up information from the OPTIC research treating moist age-related macular degeneration (moist AMD) throughout The Retina Society Annual Meeting in Pasadena, California. New information introduced embrace last two-year security and efficacy evaluation of Ixo-vec (ixoberogene soroparvovec, previously known as ADVM-022), together with discount in annualized anti-VEGF injections, clinically related aflibercept protein ranges and upkeep to enchancment of imply best-corrected visible acuity (BCVA) and central subfield thickness (CST). OPTIC research contributors might enroll in an extension research for an extra three years for a complete of 5 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,” said 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 6×10^10 (6E10) dose, along with enhanced prophylactic steroid regimens.”

Data Highlights

• Ixo-vec was usually nicely tolerated with dose-dependent irritation that was aware of topical steroids.
• All 2E11 contributors had been irritation free and didn’t require steroids to deal with irritation on the finish of the research.
• OPTIC trial contributors had an 81% – 98% discount in annualized anti-VEGF injections, in addition to steady therapeutic aflibercept protein ranges demonstrated by way of three years in extension research contributors.
• 80% and 53% of the contributors within the 6×10^11 (6E11) and 2×10^11 (2E11) dose teams, respectively, had been supplemental injection free over two years.
• BCVA and CST had been maintained to improved by way of at the very least two years at each the 6E11 and 2E11 dose ranges.

“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 companies at Wills Eye Hospital, and presenter of the information 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 presently underway in LUNA, a double-masked, randomized, Phase 2 trial. Adverum expects to conduct the trial at roughly 40 websites within the U.S. and Europe. Up to 72 topics shall be randomized equally between the 2E11 dose and new decrease 6E10 dose and throughout 4 prophylactic steroid regimens. Specific regimens being evaluated embrace topical difluprednate (Durezol®), dexamethasone intravitreal implant (Ozurdex®), or a mix of both topical Durezol® or IVT Ozurdex® with oral prednisone, with the intention of establishing a prophylactic routine with minimal want for irritation administration put up prophylaxis.

About Wet Age-Related Macular Degeneration

Wet AMD, often known as neovascular AMD or nAMD, is a sophisticated type of AMD, affecting roughly 10% of sufferers dwelling with AMD. Wet AMD is a number one trigger of blindness in sufferers over 65 years of age, with a prevalence of roughly 20 million people worldwide dwelling with this situation. The incidence of new instances of moist AMD is predicted to develop considerably worldwide as populations age. AMD is projected to affect 288 million individuals worldwide by 2040, with moist AMD accounting for about 10% of these instances.

About OPTIC Trial of ADVM-022 in Wet AMD

ADVM-022, ixoberogene soroparvovec (Ixo-vec), is Adverum’s clinical-stage gene remedy product candidate being developed for the remedy of moist AMD. Ixo-vec makes use of a proprietary vector capsid, AAV.7m8, carrying an aflibercept coding sequence below the management of a proprietary expression cassette. Unlike different ophthalmic gene therapies that require surgical procedure to manage the gene remedy below the retina (sub-retinal strategy) Ixo-vec has the benefit of being administered as a one-time IVT injection within the workplace and is designed to ship long-term efficacy and cut back the burden of frequent anti-VEGF injections, optimize affected person compliance, and enhance imaginative and prescient outcomes for sufferers with moist AMD.

The OPTIC trial is designed as a multi-center, open-label, dose-ranging, security, and efficacy trial of Ixo-vec in contributors with moist AMD who’ve demonstrated responsiveness to anti-VEGF remedy. Patients in OPTIC are treatment-experienced, and beforehand required frequent anti-VEGF injections to handle their moist AMD and to keep up practical imaginative and prescient.

About LUNA Trial of Ixo-vec in Wet AMD

Ixoberogene soroparvovec (Ixo-vec) is Adverum’s clinical-stage gene remedy product candidate being developed for the remedy of moist AMD. Ixo-vec makes use of an engineered, proprietary vector capsid, AAV.7m8, carrying an aflibercept coding sequence below the management of a proprietary expression cassette. Unlike different ophthalmic gene therapies that require surgical procedure to manage the gene remedy below the retina (sub-retinal strategy), Ixo-vec has the benefit of being administered as a one-time IVT injection within the workplace and is designed to ship long-term efficacy and cut back the burden of frequent anti-vascular endothelial progress issue (VEGF) injections, optimize affected person compliance, and enhance imaginative and prescient outcomes for sufferers with moist AMD.

The LUNA trial is a multicenter, double-masked, randomized, parallel-group Phase 2 research evaluating two doses of Ixo-vec, together with the 2×10^11 vg/eye (2E11) dose and a brand new, decrease 6×10^10 vg/eye (6E10) dose, in moist AMD. The trial will randomize as much as 72 contributors equally throughout two doses and 4 prophylactic steroid regimens in 40 websites within the U.S. and Europe. The major endpoints embrace the imply change in best-corrected visible acuity (BCVA) from baseline to 1 12 months and the incidence and severity of hostile occasions.

About Adverum Biotechnologies

Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage firm that goals to ascertain gene remedy as a brand new commonplace of take care of extremely prevalent ocular ailments with the aspiration of growing practical cures to revive imaginative and prescient and forestall blindness. Leveraging the analysis capabilities of its proprietary, intravitreal (IVT) platform, Adverum is growing sturdy, single-administration therapies, designed to be delivered in physicians’ workplaces, to get rid of the necessity for frequent ocular injections to deal with these ailments. Adverum is evaluating its novel gene remedy candidate, ixoberogene soroparvovec (Ixo-vec, previously known as ADVM-022), as a one-time, IVT injection for sufferers with neovascular or moist age-related macular degeneration. By overcoming the challenges related to present remedy paradigms for these debilitating ocular ailments, Adverum aspires to rework the usual of care, protect imaginative and prescient, and create a profound societal affect across the globe. For extra data, please go to www.adverum.com.

Forward-looking Statements

Statements contained on this press launch concerning occasions or outcomes which will happen sooner or later are “forward-looking statements” throughout the which means of the Private Securities Litigation Reform Act of 1995. Such statements embrace however should not restricted to statements concerning the potential advantages of Ixo-vec, in addition to the design of and affected person enrollment within the ongoing LUNA trial evaluating the 2×10^11 (2E11) dose and a brand new, decrease 6×10^10 (6E10) dose, together with enhanced prophylactic steroid regimens. Actual outcomes might differ materially from these anticipated in such forward-looking statements in consequence of numerous dangers and uncertainties, together with dangers inherent to, with out limitation: Adverum’s novel expertise, which makes it tough to foretell the timing of graduation and completion of medical trials; regulatory uncertainties; enrollment uncertainties; the outcomes of early medical trials not all the time being predictive of future medical trials and outcomes; and the potential for future issues or unwanted side effects in reference to use of Ixo-vec. Additional dangers and uncertainties going through Adverum are set forth below the caption “Risk Factors” and elsewhere in Adverum’s Securities and Exchange Commission (SEC) filings and reviews, together with 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 on this press launch communicate solely as of the date on which they had been made. Adverum undertakes no obligation to replace such statements to mirror occasions that happen or circumstances that exist after the date on which they had been made.

Corporate & Investor Inquiries

Anand Reddi
Vice President, Head of Corporate Strategy, External Affairs and Engagement
Adverum Biotechnologies, Inc.
T: 650-649-1358
E: [email protected]

Media

Megan Talon
Associate Director, Corporate Communications
Adverum Biotechnologies, Inc.
T: 650-649-1006
E: [email protected]