ADVM-022 for Wet AMD
For wet AMD, we are advancing our gene therapy candidate ADVM-022 (AAV.7m8-aflibercept) in the OPTIC Phase 1 clinical trial. The multi-center, open-label, Phase 1, dose-ranging trial is designed to assess the safety and tolerability of a single intravitreal (IVT) administration of ADVM-022 in treatment-experienced patients with wet AMD who are responsive to anti-vascular endothelial growth factor (VEGF) treatment. For more information on this trial, please visit www.clinicaltrials.gov.
ADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection, designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve vision outcomes for wet AMD patients.
In recognition of the need for new treatment options for wet AMD, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for ADVM-022 for the treatment of wet AMD.
About Wet AMD
Age-related macular degeneration (AMD) is a progressive disease affecting the macula, the region of the retina at the back of the eye responsible for central vision. In patients with wet AMD, an aggressive form of AMD, abnormal blood vessels grow underneath and into the retina. These abnormal blood vessels leak fluid and blood into and beneath the retina, causing vision loss.
Wet AMD is a leading cause of vision loss in patients over 60 years of age, with a prevalence of approximately 1.2 million individuals in the U.S. and 3 million worldwide1. The incidence of new cases of wet AMD in the U.S. is approximately 150,000 to 200,000 annually, and this number is expected to grow significantly as the country’s population ages2,3.
Although the underlying molecular causes of wet AMD are not completely known, VEGF is known to play a central role in the growth of new blood vessels in wet AMD.
The current standard-of-care therapies for wet AMD are proteins that bind VEGF and neutralize its activity. These therapies can be burdensome, as patients generally require chronic intravitreal injection of anti-VEGF protein every 4-12 weeks. Compliance with this regimen can be difficult for patients and their caregivers, leading to compliance deficiencies and loss of vision from underdosing. It is estimated that these standard-of-care branded anti-VEGF therapies used for the treatment of wet AMD, DR, retinal vein occlusion, and other ocular diseases generated in excess of $11 billion in sales worldwide in 20194.
ADVM-022 gene therapy candidate utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. ADVM-022 is administered as a one-time intravitreal injection, designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance and improve vision outcomes for patients with wet AMD. The OPTIC Phase 1 clinical trial of ADVM-022 in treatment-experienced patients with wet AMD is ongoing.