Adverum IND application activated for AMD treatment
Adverum Biotechnologies announced its investigational new drug application is now active for its planned phase 1 study of ADVM-022, a novel gene therapy candidate for the treatment of wet age-related macular degeneration.
The gene therapy candidate ADVM-022 uses a propriety vector capsid (AAV.7m8) to deliver, in one intravitreal injection, potentially sustained therapeutic levels of aflibercept, which could minimize the burden of frequent anti-VEGF injections, according to a company press release.
The multicenter, open-label, dose escalation trial is designed to assess the tolerability and safety of the therapy. The trial is expected to enroll 18 patients with wet age-related macular degeneration and evaluate three doses of ADVM-022.
Safety and tolerability at 24 weeks after a single intravitreal injection will be evaluated as the primary endpoint, with change in best corrected visual acuity at 24 weeks being evaluated as the secondary endpoint. Central retinal thickness and the number of rescue aflibercept injections will also be evaluated, and patients will be followed for 2 years.
A preclinical study of the therapy showed ADVM-022 was well tolerated and resulted in no serious adverse events. Additionally, the therapy induced sustained intraocular expression of aflibercept for up to 16 months, according to the release.