Academic and Industry Collaborative Study, including Goldfinch Bio, Demonstrates Benefit of Proteinuria Reduction on Kidney Survival in Patients with Focal Segmental Glomerulosclerosis (FSGS)

10.07.2020

— FSGS is a leading cause of end stage kidney disease in children and adults, with no approved therapies targeting underlying disease pathology —

 — Phase 2 study underway with Goldfinch Bio’s novel TRPC5 inhibitor, which utilizes a precision medicine approach designed to potentially reduce proteinuria and prolong kidney survival in patients —

CAMBRIDGE, Mass. – October 8, 2020 – Goldfinch Bio, a clinical stage biotechnology company focused on discovering and developing precision medicines for the treatment of kidney diseases, today announced the publication of data demonstrating the benefit of continuous proteinuria reduction on kidney survival in patients with focal segmental glomerulosclerosis (FSGS) and underscoring the utility of proteinuria as a clinically meaningful endpoint in FSGS. The new findings from an academic and industry collaborative study, including Goldfinch Bio, were published electronically in the American Journal of Kidney Diseases (AJKD), the official journal of the National Kidney Foundation (Troost et al., “Proteinuria Reduction and Kidney Survival in Focal Segmental Glomerulosclerosis”).

“We were pleased to participate with our academia and industry colleagues in this important study and are encouraged by the results, which show a clear relationship between reductions in proteinuria and preservation of kidney function, and suggest that even modest reductions in urinary protein levels can be associated with meaningful long-term clinical benefit,” said Liron Walsh, M.D., Vice President, Clinical and Translational Nephrology of Goldfinch Bio and an author on the AJKD publication.

Dr. Walsh continued, “In preclinical studies, treatment with our lead product candidate, GFB-887, significantly reduced proteinuria in models of both FSGS and diabetic nephropathy (DN). We look forward to seeing how these benefits translate in our ongoing Phase 2 clinical trial of GFB-887 and believe GFB-887 could potentially be the first therapeutic to effectively halt disease progression and prevent the onset of kidney failure.”

The AJKD publication is a retrospective analysis of data from a National Institutes of Health-funded, multicenter, randomized clinical trial of 138 children and adults with steroid-resistant primary FSGS, who were treated with cyclosporine or the combination of oral dexamethasone and mycophenolate mofetil for 26 weeks, as well as a maximally tolerated dose of an angiotensin converting enzyme inhibitor (lisinopril) or an angiotensin receptor blocker (losartan).

The analysis revealed that a greater reduction in proteinuria was associated with both a slower rate of decline in estimated glomerular filtration rate (eGFR), a key measure of kidney function, and a decreased likelihood of progression to end-stage kidney disease (ESKD) or death. Moreover, the study findings demonstrated that incremental proteinuria reductions can translate to clinically meaningful differences in eGFR slope (a metric for the severity of disease progression). This suggests that lowering proteinuria levels – even if not to complete remission – may offer patients years of preserved native kidney function, delaying the onset of ESKD with attendant morbidity and mortality and the need for renal replacement therapy.

Goldfinch Bio is currently advancing GFB-887, a first-in-class highly potent and selective inhibitor of Transient Receptor Potential Canonical Channel 5 (TRPC5). Overactivation of the TRPC5-Rac1 pathway leads to injury of podocytes, which are cells lining the kidney that, in their normal state, prevent essential protein loss (proteinuria). Injury of podocytes causes podocyte loss, proteinuria and, eventually, kidney failure. TRPC5-Rac1 pathway overactivation is the key cause of disease in a substantial proportion of FSGS and DN patients. Currently, there are no approved drugs that specifically target the TRPC5-Rac1 pathway in these diseases.

GFB-887 is currently being evaluated in TRACTION-2, a Phase 2 clinical trial in patients with FSGS or DN. The primary endpoint of the study is to assess the clinical activity of multiple doses of GFB-887 over 12 weeks, as measured by the percentage change from baseline in proteinuria. Goldfinch expects to report initial clinical data at the end of 2020 or early in 2021, and final 12-week data in mid-2021, subject to the impact of COVID-19.

About Goldfinch Bio

Goldfinch Bio, Inc. is a clinical stage biotechnology company that leverages a genomics-based, precision medicine approach to discovering and developing kidney disease treatments. Its Kidney Genome Atlas (KGA™) is a proprietary biology platform that drives candidate discovery, biomarker development and patient selection. The Company’s lead candidate, GFB-887, is a TRPC5 ion channel inhibitor, in Phase 2 development for the treatment of kidney diseases. Goldfinch Bio is also developing GFB-024, a peripherally-restricted cannabinoid receptor 1 (CB1) inverse agonist monoclonal antibody, for the treatment of rare and metabolic kidney diseases and expects to submit an investigational new drug (IND) application in 2021. Goldfinch Bio, headquartered in Cambridge, Massachusetts, was launched in 2016 by Third Rock Ventures and named in 2020 as one of FierceBiotech’s “Fierce 15.” Goldfinch Bio has an established strategic collaboration with Gilead Sciences, Inc. For more information about Goldfinch Bio, visit www.goldfinchbio.com.

Goldfinch Bio Contacts:

Investors: Hannah Deresiewicz
Stern Investor Relations, Inc.
212-362-1200
hannah.deresiewicz@sternir.com

Media: Liz Melone
lmelone@goldfinchbio.com