An Inside Look: Vertex’s Study in APOL1-FSGS July 29, 2020 by Kylie Winkler We recently sat down with Dr. Ogo Egbuna, Vertex Pharmaceutical’s clinical development lead for their clinical trial in APOL1-mediated focal segmental glomerulosclerosis (FSGS) to learn a bit more about their program and why it’s exciting for the disease community. Dr. Egbuna is a board-certified nephrologist with a deep interest in FSGS and he provided some insight into the importance of the trial and what potential participants should know if they’re interested. Dr. Ogo Egbuna The purpose of the study discussed below is to evaluate the safety, tolerability and effectiveness of an investigational medication in individuals with APOL1-mediated FSGS. The word “investigational” means this medication is not approved for use by the Food and Drug Administration (FDA) in the United States or other regulatory agencies in the UK, Europe or elsewhere. How is this drug different than other treatments currently in use for FSGS? As many of you may know, there are many different causes of FSGS. At Vertex, we’re focused on FSGS mediated by variants of the APOL1 gene. With our investigational small molecule treatment, we are aiming to target the underlying cause of disease by inhibiting the APOL1 pathway. Why is a drug for APOL1-mediated FSGS needed? A key part of our strategy at Vertex is to work on diseases where there is a high unmet need for treatment. The current treatments do not address the underlying cause of disease. Let’s assume that you are able to successfully recruit participants in this study and the drug proves to be effective. What is an estimated or typical timeline for when we could see this drug available on the market? Drug discovery and development is a long process, but we’re working as quickly as possible to do the things needed to determine whether this potential therapy has an acceptable risk/benefit profile for patients and if so, we’ll work with regulators to bring it to people who need it as soon as possible. At Vertex, we focus on serious diseases where we can have a transformative impact for patients, not just an incremental benefit. Rather than looking for problems we can solve with only the tools we’ve used before, we figure out the problems that need to be solved for the diseases we’re going after and invent the tools to potentially fix them. Who is this study for? We are excited to work with and grow our relationships with the FSGS community. This study is for adults of African or Caribbean descent (ages 18-60) with two APOL1 gene variants and biopsy confirmed FSGS. For more information on this study, please visit FSGSResearchStudy.com Do I have to live near a study site to participate? We are committed to designing our trial to be as easy for participants as possible, and we have taken the participant considerations into account each step of the way. With that in mind, we’re excited to incorporate telemedicine and participant choice into our clinical trial process. You do not need to live near a study site to participate in the Vertex APOL1-mediated FSGS study. Only your first screening visit needs to be in person; after that, all visits can be done from home, at the study site or a combination of the two. It is your choice. For home visits, a home health company will provide a nurse to visit your home to collect information and perform required tests. You’ll be able to complete a telemedicine phone call or video visit with the study doctor, and the study drug can be shipped right to your home. For visits at the study site, Vertex provides travel assistance that will support costs and arrangements. How will I know if I have an APOL1 gene variant? As part of the Vertex APOL1-mediated FSGS study, you will be tested for APOL1 gene variants. What does it mean if I test positive for the APOL1 gene variant? This means you have a genetic variant that increases your risk of developing kidney disease and accelerates the progression of kidney disease. Can I be in remission from proteinuria and still participate in the study? Patients who are in remission are not eligible for this study. Can I be on dialysis? Patients currently on dialysis are not eligible for this study. If I need a biopsy to participate in this study, will my insurance cover it? Would Vertex cover it? The study is enrolling participants with a previously confirmed FSGS diagnosis. This article was developed in partnership with Vertex Pharmaceuticals.