Bayer announced initiation of a Phase IIa clinical trial with BAY 3401016, an investigational monoclonal antibody with potential to block a protein called Semaphorin 3A (Sema3A), which is thought to be involved in the progression of kidney damage in Alport Syndrome (AS), a rare genetic disorder. The first-in-patient study, ASSESS, is a randomized, double-blind, placebo-controlled, group-comparison trial (NCT07211685), with an extension phase, which will investigate the efficacy and safety of BAY 3401016 in adult participants with Alport Syndrome. Alport Syndrome is caused by genetic mutations that affect the type IV collagen found in the kidneys, ears, and eyes.
It leads to progressive severe proteinuria, loss of kidney function, and early onset of end-stage renal disease. Diagnosis typically occurs in childhood or adulthood (depending on mutation type) via urine tests, kidney biopsy or genetic testing. Both men and women are affected by Alport Syndrome.
Many women may initially have milder symptoms and experience a later onset of disease progression. Currently, there is no specific treatment approved for Alport Syndrome and, despite guideline-recommended therapy, patients still experience progressive decline of kidney function resulting in end-stage kidney disease around their fourth decade of life or even earlier. The main goal of the ongoing study is to learn how well BAY 3401016 works in slowing down the loss in kidney function in adults with rapidly progressing AS.
Investigational BAY 3401016 is derived from Bayer's strategic research collaboration with Evotec. The program has received Fast Track Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). Alport Syndrome (AS) is a rare genetic condition that causes kidney disease, hearing loss, and eye abnormalities that occur due to changes in specific genes (COL4A3, COL4A4, and COL4A5) that affect the protein type IV collagen.
Type IV collagens are an important component of basement membranes in the kidney, but also other organs, and mutations in these genes result in functional impairment. People with AS have a high risk of developing chronic kidney disease (CKD), a condition in which there is progressive loss in kidney function over time resulting in end-stage kidney Disease. As a consequence, kidneys have lost their ability to remove waste products from the body properly, leading to the need for kidney replacement therapy, which involves either dialysis or a kidney transplant.
A common sign of impaired kidney function is the presence of excess of the protein albumin in the urine that is not usually found with healthy kidney diseases. This condition is known as albuminuria. The study drug BAY 3401016 is a monoclonal antibody that blocks a protein called Semaphor in 3A (Sema3 A), which is thought to be involve in the progression of kidney damage In Alport Syndrome (AS).
By blocking the action of the kidney, which is thought to be involved with the kidney damage in Alport Syndrome. By blocking the action of the protein albumininuria, and eye abnormalities, which is thought to be associated in the progression of kidney damage.
