Phase 3 ARTEMIS Clinical Trial: Ixoberogene Soroparvovec (Ixo-vec) in Neovascular (Wet) Age-Related Macular Degeneration

What is a clinical trial?

A clinical trial is a research study that tests whether an investigational product is safe and effective for use in humans. All medications must be successfully tested in clinical research studies before they can be approved by the FDA and prescribed to patients.

What is Adverum’s ARTEMIS Phase 3 trial in wet AMD?

This Phase 3 clinical trial is being conducted to determine if the investigational treatment Ixo-vec is as safe and effective as the currently approved standard treatment in participants with wet AMD. Potential participants must have active wet AMD and demonstrate a meaningful therapeutic response to anti-VEGF therapy to be eligible for evaluation for potential inclusion in the clinical trial. This trial is open for enrollment.

What is Ixo-vec?

Ixoberogene soroparvovec (Ixo-vec; ADVM-022; AAV.7m8-aflibercept) is an investigational gene therapy product being developed for wet AMD. Ixo-vec uses a modified virus, known as an adeno-associated virus (AAV), which has been engineered so that it cannot replicate or cause infection. AAV is not known to cause disease in humans and has been previously used in gene therapy to effectively deliver genes into specific cells.

In this case, Ixo-vec delivers a therapeutic gene to cells in the back of the eye. This gene enables the cells to produce aflibercept, a protein that reduces the growth of abnormal blood vessels and helps maintain vision. Aflibercept is the active ingredient in the approved anti-VEGF therapy, which is typically injected into the eye every 4 to 8 weeks to manage wet AMD. By enabling the eye to produce its own aflibercept, Ixo-vec may reduce the need for ongoing aflibercept injections.

How is Ixo-vec Delivered?

Ixo-vec is delivered through a procedure called intravitreal injection. This involves injecting the treatment directly into the vitreous, the gel-like substance inside the eye. The intravitreal injection allows the therapeutic genes carried by the AAV to reach the cells in the retina, where they can produce the aflibercept protein. The procedure is performed by an eye care professional using a fine needle and is typically done under local anesthesia to minimize discomfort.

Who can participate in Adverum’s Phase 3 trial of Ixo-vec in wet AMD?

You may be eligible to participate if you:

• Are at least 50 years of age
• Have been diagnosed with wet AMD and have active disease (i.e., presence of fluid on a non-invasive eye imaging test)
• Have a visual acuity score between 35 and 78 ETDRS letters (20/200 to 20/32) in the study eye
• Demonstrate a meaningful response to anti-VEGF therapy during the study screening period
• Are able to reliably use eye drops as required by the study protocol

You might not be eligible to participate if you:

• Have any medical condition that contraindicates the use of Ixo-vec or compromises the participant’s ability to comply with planned study activities
• Received any prior gene therapy
• Experienced any active eye infections recently
• Suffered a heart attack or stroke in the past six months
• Have uncontrolled high blood pressure
• Evidence of poorly controlled diabetes
• Diagnosed with other serious eye conditions
• Underwent major eye surgery in the last three months

Additional criteria apply, and the study doctor is the only one who can determine eligibility to participate in the study.  Please see ClinicalTrials.gov for more information.

Why participate in this study?

There is no guarantee that you will benefit from taking part in this study or that you will receive the study product. If you are randomized (a way of putting people into different groups at random) into the study product arm of the study, it is possible:

• The study drug may decrease the need for regular aflibercept injections
• There may be an improvement in your visual acuity or a slowing of progression of your visual loss from wet AMD
• That the results may not help you individually but the information we get from the study will help us improve treatment for participants with the same disease as yours in the future

What are the potential risks of participating in this trial?

As with all research studies, the study product and study procedures may involve unknown risks. Any medication can have temporary and permanent side effects and can cause unforeseen adverse reactions, though not everyone gets them. The potential risks of participating in this clinical trial will be explained to you before you decide to participate.

Are there any potential payments related to the study?

There may be travel support available to get you to and from the office of the study doctor for your study visits. There may also be payments to you if you meet a pre-defined level of compliance with procedures and study visits. Any payments will be discussed with you during the Informed Consent Process.