iBio, Inc. (NYSE AMEX:IBIO) today announced it has acquired Orphan Drug Designation for plant-produced human alpha galactosidase A (“α-Gal A”) and related property rights from an affiliate of Kentucky Bioprocessing LLC (“KBP”) and has initiated a program, based on its iBioLaunch™ platform, to develop an improved version of the enzyme for therapy of Fabry disease. iBio will work with its regular research and development collaborator, Fraunhofer USA Center for Molecular Biotechnology, for product development, and with KBP for manufacture of clinical and commercial quantities of the new product for iBio or its licensees. This Fabry disease therapeutic program will be part of iBio’s broader program to bring forward approximately ten more candidate proteins for commercialization as “biosimilar” or “biobetter” therapeutic products. iBio intends to advance these candidates to a point sufficient to demonstrate the advantages of making them using the iBioLaunch platform technology and then license them to industry partners. iBio already has demonstrated the applicability of its platform technology to most therapeutic protein classes, ranging from cytokines and growth factors to enzymes and antibodies. Under its current program, iBio is selecting commercial targets that exemplify advantages of the iBioLaunch technology and meet apparent market needs. Selection of the Fabry disease candidate was influenced by: the current worldwide shortage of α-Gal A for enzyme replacement therapy for Fabry disease; the likelihood that the disease is significantly under-diagnosed; the opportunity to acquire Orphan Drug Designation for plant-produced α-Gal A (which can provide significant market protection and tax advantages); and iBio’s belief this will further demonstrate some of the advantages of our technology such as scalability for Orphan Drug applications, more rapid product development, and improved efficacy compared to other systems. “We believe the unusual scalability, speed and efficiency of iBio’s plant-based protein expression platform technology can make important contributions to rapid development and availability of therapies against orphan diseases such as Fabry Disease,” said Robert Erwin, President of iBio.
Current estimates indicate a patient population affected by Fabry Disease of approximately 8,000 to 10,000. Current therapies are estimated to cost more than $200,000 per patient year. Symptoms usually begin in childhood. Unless continuously treated, Fabry Disease results in many severe health problems such as kidney failure, heart damage and cerebrovascular problems.About Fabry Disease Fabry disease is caused by the inherited deficiency of the enzyme, alpha galactosidase A, whose function is to break down a fatty substance called globotriaosylceramide. The defective gene is carried on the X chromosome, and men with the defect tend to experience worse symptoms than women who carry one copy of the defective gene. However, many women who are heterozygous for the defect are symptomatic and are frequently under-treated. Symptoms of the disease typically begin in childhood and include pain in the hands and feet, angiokeratomas, and changes in the cornea. The disease is progressive and symptoms of kidney, heart and neurological damage may occur in young adulthood. Enzyme replacement therapy with recombinant human alpha-galactosidase A has been proven clinically beneficial in the reduction of disease symptoms. About iBio, Inc. iBio, Inc. is a biotechnology company offering its proprietary, transformative iBioLaunch technology platform for the production of biologics including therapeutic proteins and vaccines. The iBioLaunch platform uses transient gene expression in green plants for superior efficiency in protein production. Advantages include significantly lower capital and process costs, and the technology is ideally suited for complex proteins and for applications where speed, scalability, and surge capacity are important. The iBioLaunch technology was developed for iBio by the not-for-profit Fraunhofer USA Center for Molecular Biotechnology (FCMB) during the past eight years to overcome the inadequacies of existing technologies. iBio owns the intellectual property and technology developed at FCMB, and continues to sponsor development and application of the technology for biological applications in human health. Further information is available at www.ibioinc.com. About Fraunhofer USA Center for Molecular Biotechnology Fraunhofer USA CMB, a division of Fraunhofer USA, Inc., is a not-for-profit research organization whose mission is to develop safe and effective vaccines targeting infectious diseases and autoimmune disorders. The technology CMB developed for iBio, Inc. provides a safe, rapid and economical alternative for both vaccine and therapeutic protein production. The Center conducts research in the area of plant biotechnology, utilizing new, cutting edge technologies applicable to the diagnosis, prevention and treatment of human and animal diseases. The Center houses individuals with expertise and excellence in plant virology, pathology, molecular biology, immunology, vaccinology, protein engineering, and biochemistry. Further information is available at www.fraunhofer-cmb.org. Forward-Looking Statements 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.