Global life sciences company Bayer has launched the phase IIa clinical trial for its experimental Alport Syndrome therapy.

The clinical trial is of BAY 3401016, an investigational monoclonal antibody targeting Semaphorin 3A (Sema3A), a protein implicated in kidney damage in Alport Syndrome (AS), a rare genetic disorder.

The first-in-patient study, dubbed ASSESS, is a randomized, double-blind, placebo-controlled trial with an extension phase designed to evaluate the safety and efficacy of BAY 3401016 in adult AS patients.

“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 affecting type IV collagen in the kidneys, ears, and eyes, leading to proteinuria, declining kidney function, and early onset of end-stage renal disease.

Diagnosis typically occurs through urine tests, kidney biopsy, or genetic testing. Both men and women are affected, though women often experience a slower progression of the disease. Currently, there is no approved treatment, and patients frequently develop end-stage kidney disease by their 40s, despite guideline-recommended therapies.

BAY 3401016, developed through Bayer’s strategic collaboration with Evotec, has received Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration. The investigational therapy works by blocking Sema3A, potentially reducing proteinuria and slowing kidney damage in AS patients.

The ASSESS study will primarily investigate whether BAY 3401016 can slow the loss of kidney function in adults with rapidly progressing Alport Syndrome while closely monitoring safety and side effects.