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Abstract: INFO22

Safety and Efficacy Evaluation of GFB-887, a TRPC5 Channel Inhibitor, in Patients with DN, FSGS, or TR-MCD: Phase 2 Study Design (TRACTION Program)

Session Information

Category: Glomerular Diseases

  • No subcategory defined


  • Walsh, Liron, Goldfinch Bio Inc, Cambridge, Massachusetts, United States
  • Reilly, John F., Goldfinch Bio Inc, Cambridge, Massachusetts, United States
  • Cornwall, Caitlin, Goldfinch Bio Inc, Cambridge, Massachusetts, United States
  • Gaich, Gregory A., Goldfinch Bio Inc, Cambridge, Massachusetts, United States
  • Ge, Tingting, Goldfinch Bio Inc, Cambridge, Massachusetts, United States
  • Gipson, Debbie S., University of Michigan, Ann Arbor, Michigan, United States
  • L Heerspink, Hiddo Jan, University Medical Center Groningen, Groningen, Netherlands
  • Johnson, Leslie, Goldfinch Bio Inc, Cambridge, Massachusetts, United States
  • Trachtman, Howard, NYU Langone Health, New York, New York, United States
  • Tuttle, Katherine R., Providence Washington, Seattle, Washington, United States
  • Czerwiec, Frank S., Goldfinch Bio Inc, Cambridge, Massachusetts, United States

Introduction: Activation of the TRPC5-Rac1 pathway in podocytes is a key driver of podocytopathies such as focal segmental glomerulosclerosis (FSGS), minimal change disease (MCD), and diabetic nephropathy (DN). Inhibition of the TRPC5 ion channel may be a potential therapeutic target for these podocytopathies. GFB-887 is a small molecule TRPC5 ion channel inhibitor that has been shown in preclinical models to prevent podocyte damage mediated by Rac1 signaling. In a first-in-human study, single doses of GFB-887 were well-tolerated with a favorable PK profile in healthy participants. GFB-887 exhibited dose-dependent reductions in urinary Rac1, indicating that GFB-887 engages TRPC5 in the podocytes and inhibits the TRPC5-Rac1 pathway.
Study Design: This is a phase 2, multicenter, double-blind, placebo-controlled, randomized, multiple ascending dose study designed to characterize the safety, efficacy, PK, and PD of GFB-887 in patients with DN, FSGS, or treatment resistant (TR)-MCD (NCT04387448). The study population will include patients on stable RAAS blockade and/or immunosuppression with residual proteinuria, defined as UPCR ≥ 1.0g/g (FSGS and TR-MCD) or UACR between 150mg/g and 3500mg/g (DN), who remain at high risk of disease progression. Participants in all dose levels will undergo a 4-week screening period, a run-in period of up to 2 weeks, a 12-week treatment period, followed by an 8-week off drug washout period. The first cohort will consist of 8 participants and subsequent cohorts will include a total of 48 participants with DN and 30 participants with either FSGS or TR-MCD. The primary objective is to evaluate the effect of increasing doses of GFB-887 on proteinuria, with the secondary objectives of exploring the safety and PK of GFB-887 after 12 weeks of therapy. Urinary Rac1 will be measured during the study to explore the potential association between urinary Rac1 and GFB-887 treatment responsiveness.
Discussion: Results from this study will provide evidence that blockade of TRPC5-Rac1 pathway with GFB-887 is an effective strategy to treat patients with podocytopathies and guide the next phase of clinical development of GFB-887.


  • Goldfinch Bio