IBIO-CFB03, an investigational fibrotic therapy, was selected as iBio’s lead candidate for further development, the company announced in a press release. The product may be a new treatment option for idiopathic pulmonary fibrosis (IPF) and scleroderma.
Fibrosis is characterized by inflammation and buildup of collagen and other proteins outside the cells. This process results in scarring of affected tissues. The underlying mechanisms of fibrosis are similar in different organs, which supports the development of medications with a common mechanism of action, although organ-specific targeting may also be pursued.
IBIO-CFB03 is a peptide derived from endostatin, a type of plant protein similar to collagen. The product slows down disease progression by preventing the formation of new blood vessels, or angiogenesis, which stops the growth, maturation, and multiplication of cells that lead to fibrosis.
IBIO-CFB03 is being developed in collaboration with a research team led by Carol Feghali-Bostwick, PhD, at the Medical University of South Carolina (MUSC).
Initial work at MUSC involved identifying peptides based on a particular region of endostatin. iBio then acquired an exclusive license to the patents and further developed these peptides.
Work in a human skin model and in mouse models of lung fibrosis showed that an endostatin-derived peptide named E4 can block and reverse skin fibrosis. Because E4 required modification for human use, iBio developed a portfolio of related treatment candidates, including IBIO-CFB03, which is an E4-based fusion protein.
Unlike most other peptides that are genetically engineered or cloned in animal models, IBIO-CFB03 will be grown in plants.
Experimental data showed that both intravenous and oral administration of IBIO-CFB03 relieves pulmonary fibrosis in rodent models. The company is now determining the minimal effective dosing by both routes, and comparing the product’s effectiveness with fibrosis medications already approved by the U.S. Food and Drug Administration (FDA).
iBio will collect a variety of data to support an application for human safety testing, including body and lung weight, pathology, and numbers of inflammatory cells. The company is also performing biomarker studies.
The next step will be manufacturing a product that meets FDA standards for quality to support an investigational new drug application to the FDA and a Phase 1 clinical study.
iBio’s priority will be scleroderma, or systemic sclerosis, for which the FDA granted IBIO-CFB03 orphan drug status, a designation for therapies intended to treat disorders affecting fewer than 200,000 people in the U.S. Development of IBIO-CFB03 for other fibrotic disease indications, including IPF, is expected to follow.