Benzinga | Aug 10, 2020 04:01PM EDT

Adverum Biotechnologies Announces Interim Data From Cohorts 1-4 From OPTIC Phase 1 Trial Of ADVM-022 Intravitreal Gene Therapy For wet AMD

REDWOOD CITY, Calif., Aug. 10, 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). The company also reported recent business progress and financial results for the second quarter ended June 30, 2020.

For the first time, interim data from all four cohorts of the OPTIC trial (July 23, 2020 cutoff date), including preliminary safety data from Cohort 4, are presented. These data further demonstrate the transformative potential of ADVM-022 to greatly reduce the treatment burden for patients with wet AMD:

* ADVM-022 continues to show robust treatment response Mean BCVA1 maintained Mean CRT2 maintained to improved

* Long-term durability beyond 15 months from single IVT injection with zero anti-VEGF rescue injections in Cohort 1 (high dose)

* Further evidence of a dose response: High dose: 6/63 patients rescue injection free Low dose: 10/154 patients rescue injection free

* Substantial reduction in annualized anti-VEGF injection rate following administration of ADVM-0225: High dose: 100% Low dose: 87%

* ADVM-022 continues to be well tolerated with a favorable safety profile in all 4 cohorts (n=30) All ADVM-022-related ocular adverse events (AE) were mild (78%) to moderate (22%) One AE of special interest of moderate recurrent uveitis deemed to be related to ADVM-022 was responsive to steroid eye drops (Cohort 1) Ocular inflammation, when observed, has been responsive to steroid eye drops No clinical or fluorescein6 evidence of posterior inflammation No vasculitis, retinitis, choroiditis, vascular occlusions, or endophthalmitis

* Early evidence from Cohort 4, consistent with Cohort 3, suggests that a prophylactic regimen of steroid eye drops results in fewer adverse events and less inflammation, when compared to a prophylactic regimen of oral steroids as used in Cohorts 1 and 2. In Cohorts 3 and 4, no patients have required more than steroid eye drops.