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.

“The initiation of the ASSESS trial represents an important milestone for our investigational BAY 3401016 program,” said Andrea Haegebarth, Ph.D., Global Head of Research and Early Development for Cardiovascular, Renal, and Immunology at Bayer’s Pharmaceuticals Division. “We are collaborating closely with the patient organization community to gain a deeper understanding of the real challenges faced by people living with Alport Syndrome. We believe BAY 3401016 holds promise as a potential therapeutic approach, and we look forward to assessing its efficacy and safety profile as we advance this important program in our pipeline.”

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.¹ 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.^2,3 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).