Nov. 5, 2019 12:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Goldfinch Bio, a U.S.-based, clinical stage biotechnology company focused on discovering and developing precision medicines for the treatment of kidney diseases, today announced it will present one oral and two poster presentations at the American Society of Nephrology (ASN) Kidney Week 2019 Annual Meeting, taking place November 5-10, 2019, in Washington, D.C.

The oral presentation will address the ways in which Goldfinch Bios platform generates induced pluripotent stem cell (iPSC)-derived human podocytes and kidney organoids to enable target validation and preclinical assessment of prospective therapies for kidney disease. The poster presentations will include data on how GFB-887, a sub-type selective, small molecule transient receptor potential canonical 5 (TRPC5) ion channel inhibitor, was effective in reducing proteinuria in animal models of focal segmental glomerulosclerosis (FSGS) and other kidney diseases, as well as data on how Goldfinch Bios proprietary Kidney Genome AtlasTM can be leveraged to unravel molecular mechanisms of kidney diseases.

Details are as follows:

Abstract Title: GFB-887, a Small Molecule Inhibitor of TRPC5, Attenuates Proteinuria in Animal Models of FSGS, Minimal Change Disease, and Diabetic Nephropathy (poster presentation)Session Title: Glomerular Diseases: Podocyte Biology IPresenter: John F. Reilly, Ph.D., Goldfinch BioDate/Time: Thursday, November 7, 2019, from 10:00 a.m. to 12:00 p.m. ETLocation: Exhibit Hall, Walter E. Washington Convention CenterAbstract Number: TH-PO1063

Abstract Title: An iPSC platform for Human Preclinical Evaluation of Kidney Disease Targeting Compounds (oral presentation)Session Title: Glomerular Diseases: Technologies, Mechanisms, and TherapeuticsPresenter: Amy Duyen Westerling-Bui, Ph.D., Goldfinch BioDate/Time: Saturday, November 9, 2019, 5:30 p.m. to 5:42 p.m. ETLocation: 201, Walter E. Washington Convention CenterAbstract Number: SA-OR053

Abstract Title: Unraveling the Genetic Contributions to Kidney Disease with the Kidney Genome Atlas (poster presentation)Session Title: Genetic Diseases of the Kidney - IIIPresenter: Thomas Soare, Ph.D., Goldfinch BioDate/Time: Saturday, November 9, 2019, 10:00 a.m. to 12:00 p.m. ETLocation: Exhibit Hall, Walter E. Washington Convention CenterAbstract Number: SA-PO408

About the Kidney Genome AtlasTM

Goldfinch Bios Kidney Genome Atlas (KGA) is the most comprehensive patient registry to investigate the underlying mechanisms of kidney disease. Through the combination of genomic, transcriptomic and proteomic data with thousands of anonymized clinical patient profiles, Goldfinch Bio is able to conduct unprecedented analyses to elucidate pathways and novel targets for kidney disease.

In May 2019, Goldfinch Bio entered into a strategic collaboration with Gilead Sciences, Inc. to sequence 80,000 diabetic kidney disease (DKD) patients and diabetic controls. Goldfinch Bio received a $55 million upfront payment, including a $5 million equity investment, and a commitment of an additional $54 million to support the development of the KGA platform for DKD. Goldfinch Bio will validate targets and support discovery and development of products to which Gilead will have exclusive option rights in exchange for additional milestone payments.

About GFB-887

GFB-887 is a sub-type selective, small molecule transient receptor potential canonical 5 (TRPC5) ion channel inhibitor in clinical development for the treatment of focal segmental glomerulosclerosis (FSGS), treatment-resistant minimal change disease (TR-MCD) and diabetic nephropathy (DN). The ongoing Phase 1 study is evaluating the safety, tolerability, and pharmacokinetic profile of GFB-887 in healthy volunteers.

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, leading 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.

About FSGS, TR-MCD and DN

Focal segmental glomerulosclerosis (FSGS) 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 (DN) develops in approximately 30 to 40 percent 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.

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 AtlasTM is a proprietary biology platform that drives candidate discovery, patient selection and biomarker development. The Companys lead candidate, GFB-887, is a transient receptor potential canonical 5 (TRPC5) ion channel inhibitor being evaluated in a Phase 1 clinical trial for the treatment of kidney diseases. Goldfinch Bio is also developing GFB-024, a peripherally-restricted cannabinoid receptor 1 (CB1) monoclonal antibody, for the treatment of rare and metabolic kidney diseases, which it licensed from Takeda Pharmaceutical Company Limited in October 2019. The company expects to submit an IND for GFB-024 in 2H 2020. Goldfinch Bio, headquartered in Cambridge, Massachusetts, was launched in 2016 by Third Rock Ventures and has an established strategic collaboration with Gilead Sciences, Inc. For more information about Goldfinch Bio, visit http://www.goldfinchbio.com.

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Goldfinch Bio to Present Oral and Poster Presentations at the American Society of Nephrology Kidney Week 2019 Annual Meeting - BioSpace