Bayer Launches Phase IIa Clinical Trial of Novel Therapy for Alport Syndrome
Bayer has announced the initiation of a Phase IIa clinical trial evaluating BAY 3401016, an investigational monoclonal antibody designed to inhibit Semaphorin 3A (Sema3A)—a protein increasingly implicated in the progression of kidney damage in Alport Syndrome (AS). This rare genetic disorder, which currently lacks an approved disease-modifying therapy, causes progressive kidney dysfunction and often leads to end-stage renal disease (ESRD) by early adulthood. The trial, dubbed ASSESS (NCT07211685), marks a significant step forward in the search for effective treatments for this debilitating condition.
The ASSESS trial is a first-in-patient, randomized, double-blind, placebo-controlled, group-comparison study with an extension phase. It is designed to evaluate both the safety and efficacy of BAY 3401016 in adult patients diagnosed with Alport Syndrome who are experiencing rapid disease progression. By targeting Sema3A—a protein shown in preclinical models to contribute to podocyte injury and glomerular scarring—the therapy aims to slow or halt the decline in kidney function that characterizes advanced stages of AS.
Andrea Haegebarth, Ph.D., Global Head of Research and Early Development for Cardiovascular, Renal, and Immunology at Bayer’s Pharmaceuticals Division, emphasized the significance of this development: “The initiation of the ASSESS trial represents an important milestone for our investigational BAY 3401016 program. 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 arises from mutations in genes encoding type IV collagen, a critical structural component of basement membranes in the kidneys, ears, and eyes. These mutations disrupt normal collagen networks, leading to progressive damage in the glomeruli—the kidney’s filtering units. The disease typically manifests with Bayer symptoms such as hematuria (blood in urine), proteinuria (excess protein in urine), and progressive loss of kidney function. While males often experience more severe symptoms and earlier onset of ESRD, females can also be significantly affected, though their disease course may be milder or delayed.
Diagnosis is usually confirmed through a combination of clinical evaluation, urine and blood tests, kidney biopsy, and increasingly, genetic testing. Despite current standard-of-care approaches—such as angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs)—that help manage proteinuria and blood pressure, many patients still progress to ESRD, often before the age of 40. This underscores the urgent unmet medical need for therapies that directly target the underlying disease mechanisms.
BAY 3401016 emerged from a strategic research collaboration between Bayer and Evotec, leveraging cutting-edge insights into the molecular pathways driving Alport-related kidney injury. The U.S. Food and Drug Administration (FDA) has recognized the potential of this therapeutic candidate by granting it both Fast Track and Orphan Drug designations—statuses that facilitate accelerated development and regulatory review for drugs targeting rare and serious conditions.
The ASSESS trial will enroll adult participants with genetically confirmed Alport Syndrome who exhibit signs of rapid kidney function decline. Participants will be randomized to receive either BAY 3401016 or a placebo, with outcomes closely monitored over the study period and during the extension phase. Key endpoints will include changes in estimated glomerular filtration rate (eGFR), proteinuria levels, and safety parameters.
If successful, BAY 3401016 could become the first disease-modifying therapy specifically approved for Alport Syndrome, offering new hope to patients and families affected by this challenging disorder. Bayer’s commitment to advancing innovative renal therapies reflects a broader industry shift toward precision medicine in rare kidney diseases.
As the trial progresses, continued collaboration with patient advocacy groups, clinicians, and regulatory bodies will be essential to ensure that the development pathway remains patient-centered and responsive to real-world needs. The Bayer ASSESS study not only represents a scientific milestone but also embodies a growing recognition that even the rarest diseases deserve robust, compassionate investigation and therapeutic innovation.
About Alport Syndrome
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 kidneys. This condition is known as albuminuria.4
About BAY 3401016
The study drug BAY 3401016 is a monoclonal antibody that blocks a protein called Semaphorin 3A (Sema3A), which is thought to be involved in the progression of kidney damage in Alport Syndrome (AS). By blocking the action of the Sema3A protein, BAY 3401016 may reduce proteinuria and slow down the loss in kidney function due to AS.
About the ASSESS study
The Alport Syndrome Sema3A Efficacy & Safety Study (ASSESS) is a Phase IIa clinical trial with an investigational drug (BAY 3401016) for patients with Alport Syndrome. The main goal of the ASSESS study is to learn more about how well BAY 3401016 works in slowing down the progression of kidney damage in patients with Alport Syndrome with increased albuminuria. The study will also evaluate the safety of the BAY 3401016 and record any side effects experienced by participants. Further details of the trial can be found at www.clinicaltrials.gov (NCT07211685).
About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses.
At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to www.bayer.com.
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