Adverum Biotechnologies, Inc. announced that it has gained alignment with the U.S. Food and Drug Administration (FDA) on the clinical development path for ADVM-022, an investigational single, in-office intravitreal (IVT) injection gene therapy for the treatment of wet age-related macular degeneration (AMD). Two Phase 3 trials are expected to commence later this year.
“Based on the results in our OPTIC trial to date and our discussion with the FDA, we can now initiate two global Phase 3 trials and have alignment on both clinical development and CMC requirements to support a BLA submission anticipated in 2024,” said Laurent Fischer, M.D., chief executive officer at Adverum Biotechnologies.
Based on the favorable safety and efficacy profiles observed to date from ADVM-022 in the OPTIC trial as well as interactions with the FDA, Adverum is planning to accelerate its clinical development program by conducting two global Phase 3 trials targeted to initiate in parallel in the fourth quarter of 2021. Pending successful outcomes of these trials, Adverum anticipates being able to submit its Biologics License Application for ADVM-022 for wet AMD in 2024.
The most recent OPTIC data presented at Angiogenesis 2021 demonstrated that patients who received 2 x 10^11 vg/eye of ADVM-022 experienced an 85% reduction in annualized anti-VEGF injections and two-thirds remained supplemental anti-VEGF injection free with median follow up of 48 weeks. Patients completing two years in OPTIC are being enrolled into an extension trial to be followed for up to five years.
The two Phase 3 trials will study the efficacy and safety of two doses of ADVM-022 straddling the 2 x 10^11 vg/eye dose used in the OPTIC trial. In the Phase 3 trials of approximately 450 patients each, patients newly diagnosed with wet AMD will be randomized to one of three arms, receiving a single IVT injection of either 3 x 10^11 vg/eye or 1 x 10^11 vg/eye of ADVM-022 compared to aflibercept IVT every 8 weeks. The primary endpoint will be non-inferiority to aflibercept based on change from baseline in Best Corrected Visual Acuity (BCVA) at one year.
“The remarkable durability and anatomical improvements seen with a single IVT injection of ADVM-022 in the OPTIC trial suggest that our therapy could provide clinically important benefits over currently marketed therapies and other late-stage programs in development for wet AMD,” said Aaron Osborne, MBBS, chief medical officer at Adverum Biotechnologies. “In our OPTIC trial, we enrolled difficult-to-treat wet AMD patients who required frequent anti-VEGF injections to maintain their vision. Our Phase 3 clinical trials will enroll newly diagnosed wet AMD patients and will be comparable to the designs of previous intravitreal anti-VEGF agents’ Phase 3 trials.”
Full press release: investors.adverum.com/news-releases/news-release-details/adverum-announces-shortened-timelines-and-clear-development-path
Source: Adverum Biotechnologies