Adverum launches phase 3 trial for wet AMD gene therapy

REDWOOD CITY, Calif. - Adverum Biotechnologies, Inc. (NASDAQ:ADVM), a clinical-stage gene therapy company with a market capitalization of approximately $87 million, has initiated the ARTEMIS Phase 3 study to evaluate the efficacy and safety of its gene therapy candidate, Ixo-vec, for patients with wet age-related macular degeneration (AMD (NASDAQ:AMD)). According to InvestingPro data, the company maintains a strong liquidity position with more cash than debt on its balance sheet, though it’s currently unprofitable as it focuses on clinical development. This pivotal trial marks the first registrational intravitreal gene therapy trial for wet AMD, targeting both treatment-naïve and previously treated patients.

The ARTEMIS trial will involve approximately 284 U.S.-based patients, comparing a single administration of Ixo-vec with the standard of care, aflibercept, administered every eight weeks. The primary goal is to determine if Ixo-vec is non-inferior to aflibercept in improving best corrected visual acuity (BCVA) after one year, with a non-inferiority margin of -4.5 letters. With a current ratio of 5.82, InvestingPro analysis shows the company has sufficient liquid assets to fund its clinical programs, though investors should note the company is burning through cash rapidly as it advances its pipeline.

Patients enrolled in the study will receive three initial doses of aflibercept before the Ixo-vec injection, with the option for additional aflibercept treatments if necessary, along with prophylactic steroid eye drops. The trial’s secondary endpoint will measure the reduction in the number of injections required, a significant factor given the high treatment burden associated with current wet AMD therapies.

Wet AMD is a leading cause of blindness in individuals over 65, with an estimated 20 million people affected worldwide. The current standard treatment requires frequent injections to manage the condition, which can lead to inconsistent patient compliance and suboptimal long-term vision outcomes.

Ixo-vec is designed to be a one-time intravitreal injection that could potentially offer lifelong benefits by reducing the need for frequent anti-VEGF injections. The therapy has received Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, as well as PRIME designation from the EMA and the Innovation Passport from the UK’s Medicines and Healthcare Products Regulatory Agency for the treatment of wet AMD.

Adverum’s commitment to advancing gene therapy as a potential new standard of care for ocular diseases is underscored by this trial’s initiation. The company aims to transform the treatment landscape for wet AMD patients by offering a more convenient and potentially more effective therapeutic option.

The initiation of the ARTEMIS trial follows the positive outcomes observed in the earlier OPTIC and LUNA trials for Ixo-vec. Adverum anticipates that the ARTEMIS study will generate the necessary data to seek regulatory approval and establish Ixo-vec as a transformative treatment for a broad spectrum of wet AMD patients. Recent InvestingPro data shows the stock has gained nearly 23% in the past week, though it remains significantly below its 52-week high. While five analysts have revised their earnings expectations downward, comprehensive analysis available in the Pro Research Report suggests the stock may be undervalued at current levels, presenting a potential opportunity for investors interested in the biotechnology sector.

This information is based on a press release statement from Adverum Biotechnologies, Inc.

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