• Apecotrep (BI 764198) is a potential first-in-class, oral, selective TRPC6 inhibitor being investigated as a novel, targeted, non-immunosuppressive therapy in people with primary focal segmental glomerulosclerosis (FSGS).
• Primary FSGS is a rare kidney condition in which injury and loss of podocytes, the cells responsible for the kidney’s filtration system, lead to proteinuria and kidney failure over time.
• Phase II trial results, published in The Lancet, demonstrated a 40% reduction in proteinuria with apecotrep (20 mg dose group) compared to placebo after 12 weeks of treatment.
• The Phase III trial is open for recruiting adults and adolescents with primary FSGS. An additional Phase II trial in other proteinuric kidney diseases will start in the first quarter of this year.

Boehringer Ingelheim  announced results from a 12-week Phase II clinical trial evaluating apecotrep (BI 764198), an oral, potential first-in-class, non-immunosuppressive TRPC6 inhibitor for people with primary focal segmental glomerulosclerosis (FSGS). Apecotrep reduced proteinuria, a key indicator associated with kidney damage, by 40% in the 20 mg dose group compared to placebo.

The results were published in The Lancet and presented at the 2025 American Society of Nephrology (ASN)’s Kidney Week. The Phase III trial (NCT07220083) is open for recruiting adults and adolescents with primary FSGS. An additional Phase II trial (NCT07355296) evaluating the safety and efficacy of apecotrep in other proteinuric kidney diseases will start in the first quarter of this year.

Apecotrep demonstrates Boehringer’s commitment to addressing high unmet medical needs across a broad spectrum of kidney diseases. This includes primary kidney conditions where no approved disease-modifying therapies currently exist.

“The results underscore Boehringer’s scientific leadership in kidney health and deep commitment to people living with cardiovascular, renal, and metabolic diseases, including rare kidney conditions like FSGS,” said Paola Casarosa, Head of Innovation Unit, Board of Managing Directors, Boehringer Ingelheim. “With the Phase III trial now underway, we are advancing apecotrep driven by the potential to deliver the first disease-modifying treatment for primary FSGS and redefine the standard of care for patients.”

Primary FSGS is a rare, progressive kidney disease which can end in kidney failure.^2,3,4 Despite its severity and burden for patients, there are currently no approved targeted therapies.^5,6,7 There remains a significant unmet need for a targeted therapy that addresses the root cause of the disease.

“Primary FSGS is a serious glomerular disease and an important cause of kidney failure in both children and adults. By targeting the underlying mechanism of primary FSGS, apecotrep reduced proteinuria by 40% compared to placebo, with a favorable tolerability profile in adults,” said Howard Trachtman, Lead Investigator, University of Michigan. “These clinically relevant findings reinforce the importance of further investigation of its potential as a first-in-class targeted treatment for primary FSGS, where the unmet need remains high.”

In primary FSGS, the protein Transient Receptor Potential Channel 6 (TRPC6) is hypothesized to be overactivated on podocytes, cells responsible for the kidney’s filtration system.⁸ This allows excessive calcium to enter the cells, causing progressive podocyte injury and loss, and ultimately, proteinuria and kidney disease progression. Apecotrep intends to protect podocytes and slow down disease progression by decreasing proteinuria.⁹ 

Highlighting its potential as a new treatment option for primary FSGS, apecotrep was granted Breakthrough Therapy Designation by the Center for Drug Evaluation (CDE) of the National Medical Products Administration of China and Orphan Drug Designations by the European Medicines Agency (EMA) and the Japanese Ministry for Health, Labour and Welfare (MHLW).