Adverum Biotechnologies Announces Positive Interim Data from Cohorts 1-4 from OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD
2020-11-14 · Adverum Biotechnologies, Inc. · original adverum.com ↗
— Maintained efficacy and greatly reduced anti-VEGF treatment burden after a single IVT injection of ADVM-022 — — R obust preliminary aqueous a nti-VEGF protein expression observed at 18 months in Cohort 1 – — Well tolerated with ocular cell grades and steroid eye drop use decreasing over time — — First p ivotal trial planned for mid-2021 following regulatory agency discussions — — Company to host webcast with Key Opinion Leaders to discuss new OPTIC data today, Saturday, November 14, 2020 at 7:30 am PT / 10:30 am ET — REDWOOD CITY, Calif., Nov. 14, 2020 (GLOBE NEWSWIRE) — Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced positive new interim data from Cohorts 1-4 of the OPTIC Phase 1 clinical trial of ADVM-022 intravitreal (IVT) injection gene therapy in patients requiring frequent anti-VEGF injections for their wet age-related macular degeneration (AMD). “With these impressive OPTIC data and the removal of the partial clinical hold on ADVM-022 by the FDA, our goal is to continue to advance into pivotal trials to demonstrate the transformative potential of our gene therapy,” said Laurent Fischer, M.D., chief executive officer of Adverum. “We are excited that ADVM-022 has the potential to be a “one and done” IVT injection that may dramatically reduce the treatment burden for the millions of patients with wet AMD and DME worldwide. Particularly during COVID-19, we are reminded of the benefits that ADVM-022, a novel gene therapy that has demonstrated long-term treatment benefit after one in-office IVT injection, could deliver to patients. Our Adverum team is laser-focused on accelerating the development and future commercial launch plans for ADVM-022. I am humbled by the dedication of the retina specialists and their staff, and our employees, to help progress our clinical trials which generate the data necessary to drive our mission of helping patients with severe ocular diseases.” Adverum reported new interim data from the OPTIC trial (October 15, 2020 cutoff date) that further demonstrate the transformative potential of ADVM-022 to greatly reduce the anti-VEGF injection burden for patients with wet AMD: ADVM-022 continues to maintain efficacy at both high and low doses (n=30) Mean BCVA 1 maintained Mean CRT 2 maintained to improved Durability out to 92 weeks from a single IVT injection with zero supplemental injections in Cohort 1 (high dose) Robust preliminary aqueous anti-VEGF protein expression observed at 18 months in Cohort 1 (high dose) Mean aqueous anti-VEGF protein level 1840 ng/mL Substantial reduction in annualized anti-VEGF injection frequency 3 following ADVM-022 in patients who previously required frequent injections: High dose: 99% reduction Low dose: 85% reduction Most patients are supplemental anti-VEGF injection free in OPTIC: High dose: 14/15 4 patients injection free Low dose: 10/15 5 patients injection free ADVM-022 continues to be well tolerated with a favorable safety profile at both high and low doses All ADVM-022-related ocular adverse events (AE) were mild (78%) to moderate (22%) Ocular inflammation, when observed, has been responsive to steroid eye drops and overall is decreasing over time No clinical or fluorescein evidence of posterior inflammation No vasculitis, retinitis, choroiditis, vascular occlusions, or endophthalmitis OPTIC Phase 1 Clinical Trial Data: Results Following a Single ADVM-022 Dose: Cohort 1 Cohort 2 Cohort 3 Cohort 4 Patients n=6 n=6 n=9 n=9 M edian (Range) F ollow-up V isit ( W eeks) 8 6 (64 to 92) 64 (64 to 68) 4 8 (32 to 48) 1 6 (12 to 24) ADVM-022 Dose High dose 6 x 10^11 vg/eye Low dose 2 x 10^11 vg/eye Low dose 2 x 10^11 vg/eye High dose 6 x 10^11 vg/eye Prophylactic S teroid R egimen 13-day oral 13-day oral 6-week eye drops 6-week eye drops Supplemental Anti-VEGF Injection Use : Number of patients supplemental injection free 6/6 patients 3/6 patients 7/9 patients 8/9 patients Total supplemental anti-VEGF injections 0 injections 17 injections in 3 patients 8 injections in 2 patients 1 injection in 1 patient Follow-up BCVA 1 and CRT 2 : All Patients All Patients Supp. IVT-free Patients 50% (3/6) All Patients Supp. IVT-free Patients 78% (7/9) N/A BCVA mean change from baseline (letters) -2.5 +0.2 +1.0 -0.9 +4.1 N/A CRT mean change from baseline (μm) -19.7 μm -1.0 μm -23.7 μm -113.4 μm -132.7 μm N/A Ocular Inflammation and Management: % patients with any cellular inflammation at most recent visit 33% 0% 11% 22% Average # of daily steroid eye drops 6 1.2 0.5 0.8 1.9
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