Benzinga | Oct 1, 2021 04:03PM EDT

Adverum Presents 2-Year OPTIC Data Demonstrating Sustained Durability And Promising Safety Profile From Single Intravitreal Injection Of ADVM-022 In Wet AMD Patients Who Previously Required Frequent Anti-VEGF Injections

REDWOOD CITY, Calif., Oct. 01, 2021 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (NASDAQ:ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced new long-term data from the OPTIC clinical trial of ADVM-022 single, in-office intravitreal (IVT) injection gene therapy in patients requiring frequent anti-VEGF injections for their neovascular or wet age-related macular degeneration (wet AMD). Safety and efficacy data from patients followed through 2 years post injection are being presented at the Retina Society's 54th Annual Scientific Meeting in Chicago, Illinois. The presentation is available on the Publications page in the Pipeline section of Adverum's website.

"We have now followed all wet AMD patients in the OPTIC trial for a minimum of a year, and in some cases beyond two years, after treatment with a single in-office intravitreal injection of ADVM-022. We are very pleased to report that both the promising safety profile and durability of treatment effect continue to be maintained in this patient population," said Laurent Fischer, M.D., president and chief executive officer at Adverum Biotechnologies. "We are excited to see that ADVM-022 has the potential to extend the treatment benefit from weeks to years."

Dr. Fischer stated, "We continue to analyze all data from patients receiving ADVM-022 in OPTIC and our INFINITY study in diabetic macular edema. We will present data from the latter on October 9 at the American Society of Retina Specialists Annual Scientific Meeting, followed by an encore presentation of OPTIC data on October 11. We look forward to completing our analysis of the data from our ADVM-022 program to meet with investigators and regulators as we outline our path forward in wet AMD. We are planning a Phase 2 trial to evaluate doses of ADVM-022 at 2 x 10^11 and lower with enhanced prophylaxis, with the goal of further improving the safety profile of ADVM-022 as we aim to deliver this novel therapy to patients."

Key Efficacy Data (July 16, 2021 cutoff date) - 2 x 10^11 vg/eye dose (n=15), planned to be evaluated in a future Phase 2 trial:

* >80% reduction in annualized anti-VEGF injection frequency1 in patients who previously required frequent injections

* >50% of patients (8/152) after median follow-up of 1.7 years remained entirely free of any supplemental anti-VEGF injection

* Robust aflibercept expression levels continue to be sustained through 2 years after a single injection of ADVM-022

* Vision (mean BCVA3 versus baseline) maintained over time

* Retinal anatomy (mean CST4 versus baseline) maintained to improved

Key Safety Data (July 16, 2021 cutoff date) - 2 x 10^11 vg/eye (n=15) and 6 x 10^11 vg/eye (n=15) doses:

* All ADVM-022-related ocular adverse events (AE) were mild (81%) to moderate (19%) across all wet AMD patients in OPTIC, and patients have reached a follow-up period of between 1 and 2 years post treatment No dose-limiting toxicities No clinically relevant decreases in intraocular pressure No vasculitis, retinitis, choroiditis, vascular occlusions, or endophthalmitis

* At the 2 x 10^11 vg/eye dose, ocular inflammation was minimal and responsive to steroid eye drops

1 Annualized rate (Before) = (number of IVTs in 12 months prior to ADVM-022) / (days from the first IVT in the past 12 months to ADVM-022 / 365.25)

Annualized rate (After) = (number of aflibercept IVTs since ADVM-022) / (days from ADVM-022 to the last study follow-up / 365.25)

2 All patients from Cohort 2 (n=6) and Cohort 3 (n=9) treated with the 2 x 10^11 vg/eye dose

3 Best corrected visual acuity (BCVA)

4 Central subfield thickness (CST)

"As the highest enroller in OPTIC, I have observed the potential of ADVM-022, a novel intravitreal gene therapy, to significantly reduce the treatment burden for my patients with neovascular age-related macular degeneration. In the latest data from the OPTIC trial, we have seen a manageable safety profile, robust aflibercept expression and sustained anatomical improvements 21 months after a single 2 x 10^11 dose of ADVM-022," said Arshad M. Khanani, M.D., M.A., Managing partner and Director of Clinical Research, Sierra Eye Associates; Clinical Associate Professor, University of Nevada, Reno School of Medicine and a member of Adverum's Scientific Advisory Board. "I look forward to continuing to provide input into the future development plans for ADVM-022 in patients with neovascular AMD."