iBio, Inc. Receives FDA Orphan Drug Designation for Fibrosis Product Candidate
NEW YORK, NY -- (Marketwired) -- 07/05/16 -- iBio, Inc. (NYSE MKT: IBIO), a leading provider of plant-based biotechnology for developing and manufacturing biological products, announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to iBio's investigational biotherapeutic product, iBio-CFB03, for the treatment of systemic sclerosis.
Systemic sclerosis is a fibrotic disorder that affects connective tissue of skin and internal organs as well as the walls of blood vessels. Early diagnosis and individualized therapy can be helpful, but treatment of systemic sclerosis is limited to symptom management. No approved drug currently available has been shown to arrest the underlying process or processes that drive progression of the disease.
"This is an important step toward our goal for a family of iBio proprietary products against fibrotic diseases like systemic sclerosis and idiopathic pulmonary fibrosis," said Robert B. Kay, iBio's Executive Chairman. "We expect to apply for additional Orphan Drug Designations for products against additional fibrotic diseases. Viewed together and across the major geographic markets, effective therapies against fibrotic diseases are among the most significant unmet medical needs in the world."
The FDA Office of Orphan Products Development (OOPD) grants Orphan status to drugs "intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S." Designation of an investigational drug as an orphan product indicates that the drug qualifies for the benefits specified in the Orphan Drug Act during development for the designated indication, including tax credits for qualified clinical testing, orphan drug exclusivity, and an exemption from marketing application user fees. As per FDA guidelines, orphan drug designation, "does not alter the standard regulatory requirements and process for obtaining marketing approval," such as establishing the safety and efficacy of a drug through adequate and well-controlled studies.
iBio-CFB03 is a recombinant, plant-made protein designed by iBio based on discoveries and inventions made by iBio's research partner, Dr. Carol Feghali-Bostwick. iBio licensed worldwide exclusive rights to her intellectual property covering iBio-CFB03 and related products and established a research agreement with the Medical University of South Caroline where Dr. Feghali-Bostwick is now the Kitty Trask Holt and SmartState® SC Centers of Economic Excellence Endowed Professor. The company is scaling up production of iBio-CFB03 for clinical trials at its subsidiary company, iBio-CMO LLC in College Station, Texas based on the use of proprietary iBio gene expression technology.
Development of iBio-CFB03 as an anti-fibrotic agent is based on the involvement of endostatin in fibrotic disease, identification of endostatin's anti-fibrotic activity residing in the C-terminal region on which the design of iBio-CFB03 is based, and the efficacy of endostatin, its C-terminal peptides, and iBio-CFB03 in experimental models of fibrosis. iBio-CFB03 was developed by iBio using iBio CMO's capabilities to improve the solubility, stability, and purification of pharmaceutical-grade product versus the predecessor molecules.
About iBio, Inc.
iBio is developing proprietary products for the treatment of a range of fibrotic diseases including idiopathic pulmonary fibrosis, systemic sclerosis, and scleroderma. IBIO-CFB03, produced using the Company's proprietary gene expression technology, is the first product candidate from this program being advanced for IND development. The Company also offers proprietary products and product licenses to others based on its proprietary technologies, providing collaborators full support for turn-key implementation of its technology for protein therapeutics and vaccines.
iBio CMO LLC is a 70 percent subsidiary of iBio jointly owned with affiliates of Eastern Capital Limited for development and large-scale manufacture of plant-made pharmaceuticals. The iBio CMO multiproduct facility includes laboratory and pilot-scale operations as well as large-scale automated hydroponic systems capable of growing over 4 million plants as "in process inventory" and producing over 300 kilograms of finished therapeutic protein per year. This translates into more than a half million doses per year of a typical therapeutic antibody and approximately 50 million vaccine doses every three weeks. Facility capacity can be doubled by adding additional plant growth equipment in a space already reserved for that purpose. iBio CMO's lease includes the right to develop another facility on the balance of the leased property that would have the effect of quadrupling capacity from the current level. iBio CMO offers a range of pharmaceutical product and process development, analytical, and manufacturing services.
In Brazil, iBio has formed a subsidiary company, iBio do Brasil Biofarmaceutical Ltda., and has been collaborating with the Oswaldo Cruz Foundation (Fiocruz) to develop a recombinant yellow fever vaccine based on iBio technology. Further information is available at: www.ibioinc.com.
STATEMENTS INCLUDED IN THIS NEWS RELEASE RELATED TO IBIO, INC. MAY CONSTITUTE FORWARD-LOOKING STATEMENTS WITHIN THE MEANING OF THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995. SUCH STATEMENTS INVOLVE A NUMBER OF RISKS AND UNCERTAINTIES SUCH AS COMPETITIVE FACTORS, TECHNOLOGICAL DEVELOPMENT, MARKET DEMAND, AND THE COMPANY'S ABILITY TO OBTAIN NEW CONTRACTS AND ACCURATELY ESTIMATE NET REVENUES DUE TO VARIABILITY IN SIZE, SCOPE AND DURATION OF PROJECTS. FURTHER INFORMATION ON POTENTIAL RISK FACTORS THAT COULD AFFECT THE COMPANY'S FINANCIAL RESULTS CAN BE FOUND IN THE COMPANY'S REPORTS FILED WITH THE SECURITIES AND EXCHANGE COMMISSION.
Source: iBio, Inc.