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, MD, 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, MD, FACS, 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.