Boehringer Ingelheim has announced results from a 12-week phase II clinical trial evaluating apecotrep, an oral, 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 20mg dose group compared to placebo, results of the trial, published in The Lancet and presented at the 2025 American Society of Nephrology (ASN)’s Kidney Week (6–9 November, Houston, USA).
The phase III trial is open for recruiting adults and adolescents with primary FSGS. An additional phase II trial evaluating the safety and efficacy of apecotrep in other proteinuric kidney diseases will start in the first quarter of 2026.
Primary FSGS is a rare, progressive kidney disease which can end in kidney failure. Despite its severity and burden for patients, there are currently no approved targeted therapies.
“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 (Ann Arbor, USA). “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 hypothesised to be overactivated on podocytes, cells responsible for the kidney’s filtration system. 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.
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).
