Research & Development
Goldfinch Bio Initiates Phase 1 Clinical Trial of GFB-887 for Kidney Disease
10 June 2019 - - US-based biotechnology company Goldfinch Bio, Inc has initiated dosing in a Phase 1 clinical trial evaluating GFB-887, its subtype-selective, small molecule TRPC5 inhibitor, as a potential treatment for kidney diseases associated with proteinuria and progressive renal dysfunction including focal segmental glomerulosclerosis, treatment-resistant minimal change disease (TR-MCD) and diabetic nephropathy, the company said.
The Phase 1 study will evaluate the safety, tolerability, and pharmacokinetic profile of GFB-887 in healthy volunteers and in patients with chronic kidney disease. Topline safety data from healthy volunteers are expected by year-end 2019.
The Phase 1 study will be conducted in two parts.
Part 1 comprises a single-ascending dose escalation component and a food effect component in healthy volunteers, and Part 2 comprises a multiple ascending dose component in healthy volunteers.
The Phase 1 programme will also include a study in renally-impaired patients. Primary study objectives include evaluating the safety, tolerability and pharmacokinetic profile of single and multiple doses of GFB-887.
Secondary objectives include evaluating the safety, tolerability and PK of GFB-887 in patients with renal impairment. For more information about the clinical trial design visit: www.clinicaltrials.gov (NCT03970122).
The GFB-887 clinical programme is supported by preclinical data in multiple models of progressive kidney disease, including data from human stem-cell derived organoids and podocytes, recently presented at the 2019 ISN World Congress of Nephrology meeting in April 2019. 
GFB-887 is a sub-type selective, small molecule TRPC5 ion channel inhibitor in clinical development for the treatment of FSGS, TR-MCD and DN. 
TRPC5 is a calcium-permeable ion channel implicated in the pathogenesis of kidney disease. Recent evidence has demonstrated that TRPC5 and Rac1, a critical regulator of cellular motility, form a vicious cycle that drives pathogenic remodeling of the actin cytoskeleton in podocytes.
This causes podocyte loss and breach of the filtration barrier, which leads to proteinuria, the hallmark of progressive kidney diseases such as FSGS, TR-MCD and DN. Inhibition of TRPC5 offers a potential point of therapeutic intervention to restore podocyte integrity and halt progression of these diseases.
FSGS (focal segmental glomerulosclerosis) is a rare kidney disorder and histopathologic diagnosis characterized by scarring of the kidney's filtering units, or glomeruli, leading to proteinuria, an excess of essential proteins spilling into the urine. FSGS is associated with loss of podocytes, terminally differentiated cells of the kidney glomeruli essential for filtration and proper kidney function.
Recent research into the genetics of kidney disease has identified over 50 genes associated with FSGS and implicates the podocyte as a central player in the pathogenesis of FSGS. There are currently no FDA approved treatments available for patients with FSGS.
Similar to FSGS, treatment-resistant minimal change disease (TR-MCD), is a rare kidney disorder characterized by podocyte injury and is an important cause of nephrotic syndrome in children as well as adults.
Clinical hallmarks of MCD include rapid onset of edema and weight gain. While MCD may be managed with corticosteroids, a subset of patients fail to respond and are considered treatment-resistant.
There are currently no FDA approved treatments available for patients with TR-MCD.
Diabetic nephropathy develops in approximately 30-40 % of patients who have diabetes and is a leading cause of end-stage kidney disease, cardiovascular disease and early mortality worldwide.
Despite current therapies, the number of people with DN continues to increase, highlighting the need for additional treatments that preserve kidney function.
Goldfinch Bio is a biotechnology company that is singularly focused on discovering and developing precision therapies for patients with kidney diseases.
Just as the goldfinch has long been a symbol of healing and renewal and was a prominent figure of the Renaissance, Goldfinch Bio is leading a new age of therapeutic discovery to transform the treatment paradigm for patients with kidney diseases. Goldfinch was launched in 2016 by Third Rock Ventures, and is headquartered in Cambridge, Mass.
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