Needham initiated coverage on Ocular Therapeutix Inc OCUL, noting the company’s lead asset, Axpaxli, an investigational axitinib-based intravitreal implant for wet age-related macular degeneration (wet AMD).Axpaxli is the company’s key value driver and is under evaluation in two phase 3 trials. If positive, these trials could support approval with a product label that enables maintenance treatment with Q6M (every 6 months)-Q12M (every 12 months) dosing, which would represent a significant improvement over c ...

Core Insights - Adverum Biotechnologies has initiated the ARTEMIS Phase 3 study to evaluate the efficacy and safety of Ixo-vec for treating wet age-related macular degeneration (AMD) [1][4] - The trial aims to demonstrate that a single injection of Ixo-vec is non-inferior to the standard treatment with aflibercept, potentially transforming the treatment landscape for wet AMD patients [5][10] Company Overview - Adverum Biotechnologies is a clinical-stage company focused on gene therapy to treat prevalent ocular diseases, aiming to establish a new standard of care [11][12] - The company is developing Ixo-vec, a one-time gene therapy designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections for wet AMD patients [10][11] Study Design - The ARTEMIS trial is a randomized, double-masked, sham-controlled study involving approximately 284 patients, including both treatment-naïve and previously treated individuals [7][8] - The primary endpoint is the mean change in best corrected visual acuity (BCVA) from baseline at one year, with a non-inferiority margin of -4.5 letters [2][8] Treatment Context - Wet AMD affects around 20 million individuals globally and is a leading cause of blindness in those over 65 years of age [9] - Current treatment requires frequent injections, with up to 42% of patients discontinuing treatment after two years, leading to poor long-term vision outcomes [4][9] Ixo-vec Characteristics - Ixo-vec utilizes a proprietary vector for a one-time intravitreal injection, aiming to provide stable therapeutic levels of anti-VEGF and improve patient compliance [10][11] - The therapy has received Fast Track and Regenerative Medicine Advanced Therapy designations from the FDA, indicating its potential as a transformative treatment option [10]

Core Insights - Adverum Biotechnologies has initiated the ARTEMIS Phase 3 study to evaluate the efficacy and safety of Ixo-vec for treating wet age-related macular degeneration (AMD) [1][4] - The trial aims to demonstrate that a single injection of Ixo-vec is non-inferior to the standard treatment with aflibercept [2][5] Study Design - ARTEMIS is a US-based, randomized, double-masked, sham-controlled trial involving approximately 284 patients, including both treatment-naïve and previously treated individuals [2][7] - The primary endpoint is the mean change in best corrected visual acuity (BCVA) from baseline at one year, with a non-inferiority margin of -4.5 letters [2][8] - All participants will receive three loading doses of aflibercept before administration of Ixo-vec [2][8] Significance of Ixo-vec - Ixo-vec is positioned as a potential best-in-class gene therapy that could provide long-term efficacy and reduce the treatment burden associated with frequent anti-VEGF injections [5][10] - The therapy is designed for a one-time intravitreal injection, which is expected to optimize patient compliance and improve vision outcomes [10][11] Unmet Medical Need - There is a significant unmet need for longer-acting therapies for wet AMD, with data indicating that up to 42% of patients discontinue treatment after two years, leading to poor long-term vision outcomes [4][9] - The current standard of care requires lifelong repeated injections, highlighting the potential impact of Ixo-vec in reducing treatment frequency [9][10] Regulatory Designations - Ixo-vec has received Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, as well as PRIME designation from the EMA [10][11]