Policy & Regulation
Goldfinch Bio Initiates Phase 1 Clinical Trial of GFB-024, a Novel Precision Medicine Product Candidate for Patients with Severe Insulin Resistant Diabetic Nephropathy
13 May 2021 - - US-based biotechnology company Goldfinch Bio has dosed the first subjects in its Phase 1 clinical trial of its product candidate, GFB-024, the company said.

GFB-024 is a peripherally restricted cannabinoid 1 inverse agonist monoclonal antibody intended to treat patients with severe insulin resistant diabetic nephropathy and CB1 pathway overactivation.

GFB-024 is Goldfinch Bio's second kidney precision medicine candidate to advance to clinical trials.

The company's most advanced precision medicine product candidate, GFB-887, a small molecule inhibitor of TRPC5, is currently in Phase 2 study (TRACTION-2 Clinical Trial) for the treatment of focal segmental glomerular disease and DN.

Patients with SIRD are characterized by obesity, marked insulin resistance, macroalbuminuria, and a fivefold greater risk of end stage kidney disease.

Preclinical data generated by Goldfinch Bio demonstrate that elevated levels of circulating endocannabinoids, such as those observed in patients with insulin resistance, activate the CB1 pathway in podocytes, tubular cells and peripheral metabolic organs.

This, in turn, can lead to mitochondrial dysfunction, resulting in podocyte cell death, proteinuria and tubular cellular injury, and ultimately serious harm to the kidneys.

In preclinical studies, Goldfinch Bio observed that treatment with GFB-024 resulted in inhibition of CB1 receptor signaling, protecting against both podocyte and tubular cell injury, with negligible exposure in the brain and no central nervous system physiological effect.

Currently, there are no approved peripherally restricted CB1 inhibitors.

The Phase 1 trial is a double-blind, randomized, placebo-controlled study designed to evaluate the safety, tolerability, and pharmacokinetics of single-ascending doses of GFB-024 in up to 56 overweight and obese healthy volunteers.

The trial will also evaluate repeat dosing of GFB-024 in one cohort of subjects with type 2 diabetes mellitus to assess immunogenicity, multiple dose PK and safety.

In addition, the trial will explore candidate predictive biomarkers to confirm peripheral CB1 target engagement to identify patients most likely to respond to GFB-024 and provide early central nervous system safety derisking.

Goldfinch expects to report initial data in 2022.

GFB-024 is a peripherally-restricted cannabinoid 1 inverse agonist monoclonal antibody intended to treat patients with severe insulin resistance diabetic nephropathy and CB1 pathway overactivation.

GFB-024 has the potential to provide disease-modifying effects in the kidney while treating the underlying metabolic dysfunction in obese patients without the CNS side-effects seen by prior small molecule CB1 antagonists.

GFB-024 has demonstrated podocyte protective effects in preclinical models, while clinical data from first generation CB1 antagonists have demonstrated metabolic benefits, such as reduced cholesterol, glucose, and HbA1c, in third-party clinical trials, which may also lead to additional beneficial kidney effects.


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