Goldfinch Bio Announces Publication in Science Advances on the Validation of Human Derived Organoids as a Predictable and Translatable Preclinical Model in Kidney Disease for the Evaluation of Therapeutic Drug Candidates

-- Company shares proprietary and scalable approach using human derived organoids for preclinical pharmacodynamic (PD) assessment of drugs targeting podocyte cells --

-- Recent preliminary data from Phase 2 study of GFB-887 in focal segmental glomerular disease (FSGS) further validate feasibility and value of using transplanted human organoids in preclinical PD studies --

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Goldfinch Bio, Inc., a clinical stage biotechnology company focused on discovering and developing precision medicines for the treatment of kidney diseases, today announced the publication of data validating transplanted, perfused human kidney organoids as a novel, predictive and translatable preclinical PD model for use in kidney disease drug development. This breakthrough approach was initially validated with the company’s lead drug candidate, GFB-887, a selective Transient Receptor Potential Canonical Channel 5 (TRPC5) inhibitor, which, since this original organoid work, demonstrated clinical proof of concept in a Phase 2 study in patients with FSGS. The manuscript, titled “Transplanted organoids empower human preclinical assessment of drug candidate for the clinic,” is available online in the international peer-reviewed journal Science Advances at: https://www.science.org/doi/10.1126/sciadv.abj5633.

“Since the start of Goldfinch Bio, we have been committed to using human organoids to improve the preclinical drug development process in kidney disease, which has lacked adequate and reliable tools to date. Our belief in the use of organoids was initially validated using GFB-887 in a human organoid model of podocyte injury, and the recent preliminary data from our ongoing Phase 2 trial of GFB-887 for FSGS demonstrates the successful clinical translation of our approach,” said Anthony Johnson, M.D., President and Chief Executive Officer at Goldfinch Bio. “We believe our pioneering approach is applicable across an array of tissues and organs, and we look forward to sharing our findings with the broader drug development community in hopes of ushering in a new era of more translatable preclinical models and, ultimately, more efficient drug discovery.”

A key challenge in modern drug development is the limited predictive power and translatability of PD studies in animals to relevant patients, which makes it difficult to accurately assess the potential efficacy of novel therapeutic drug candidates preclinically. To address this shortcoming, Goldfinch Bio scientists developed a novel, scalable approach for in vivo PD studies that leverages human tissue in the form of induced pluripotent stem cell (iPSC)-derived kidney organoids.

The Science Advances manuscript outlines new insights gleaned from Goldfinch Bio’s preclinical study of GFB-887, including when best to transplant organoids to optimize vascularization, perfusion and differentiation, and the preferred method for organoid profiling and quality control.

As described in the Science Advances publication, and as previously presented at the 13^th International Podocyte Conference, Goldfinch Bio used human-derived organoid systems to establish preclinical efficacy for GFB-887, the Company’s highly potent and selective inhibitor of TRPC5. In February 2022, Goldfinch Bio announced treatment with GFB-887 in an ongoing Phase 2 clinical trial demonstrated a statistically significant, clinically meaningful 32% placebo-adjusted mean reduction in urine protein creatine ratio (UPCR) in patients with FSGS.

About Goldfinch Bio

Goldfinch Bio, Inc. is a clinical stage biotechnology company focused on delivering disease-modifying precision medicines that bring hope and renewed quality of life to people living with kidney diseases. We aspire to save kidneys and end dialysis. We have a robust pipeline of novel, precision medicine product candidates targeting kidney diseases with significant unmet need, including two clinical-stage assets. Visit us at www.goldfinchbio.com to learn more.