FRAMINGHAM, Mass.--(BUSINESS WIRE)--GTC Biotherapeutics, Inc. ("GTC", Nasdaq: GTCB) has obtained exclusive worldwide rights to the development and commercialization of recombinant human alpha-fetoprotein (rhAFP), including the recombinant, non-glycosylated version of rhAFP known as MM-093, for the treatment of autoimmune diseases from Merrimack Pharmaceuticals. GTC will receive an initial inventory of bulk drug substance suitable for use in clinical studies. GTC will also assume control of the transgenic goats that express rhAFP in their milk, which were originally developed by GTC for Merrimack and are cared for in GTC’s facilities. GTC intends to further develop rhAFP through commercial partnering.
“This product opportunity represents an exceptional strategic fit for GTC and we are excited to add a drug that has great potential as a treatment for multiple sclerosis and myasthenia gravis to our proprietary pipeline of recombinant human plasma proteins,” stated Geoffrey F. Cox, PhD, GTC’s Chairman and CEO. “The already established safety data and production capability, the published animal disease models, and access to sufficient drug substance make rhAFP an attractive candidate for partnering discussions to support further development and commercialization, potentially as a phase II product candidate.”
Published studies of rhAFP and AFP derived from cord blood indicate that the recombinant form could be as effective in treating multiple sclerosis as the native form that occurs during pregnancy. With a strong dosing and safety profile and supportive preclinical evaluations of efficacy, further studies of rhAFP in multiple sclerosis or myasthenia gravis may begin at the proof of concept phase II level of clinical evaluation. Preclinical models of myasthenia gravis support the use of rhAFP as a treatment for this unmet medical need.
Human AFP is an immunomodulatory blood protein normally produced at very high levels during pregnancy. Research on AFP suggests that it plays a role in modulating the mother’s immune system to protect the fetus. The presence of high levels of AFP in the mother’s blood has long been associated with remission of many autoimmune diseases. AFP is difficult to express in recombinant systems other than in transgenic mammals and is not available as a blood fractionation product due to very low levels except during pregnancy.
Multiple sclerosis is an autoimmune disease characterized by numbness, weakness, loss of muscle coordination, and problems with vision, speech, and bladder control. A number of products have been developed to treat multiple sclerosis, all of which have significant safety concerns. Multiple sclerosis affects approximately 400,000 people in the US and more than 2.5 million worldwide.
Myasthenia gravis is an autoimmune disease characterized by fatigue and exhaustion of the muscles. Multiple therapeutic approaches are used for the control of myasthenia gravis, often in combination therapy, including the use of immunomodulatory medications (corticosteroids, azathioprine, cyclosporine, and cyclophosphamide), AChE inhibitors, plasmapheresis, and thymectomy. Some of these treatments have a high incidence of significant side effects such as flu-like symptoms, susceptibility to infection, and an increased incidence of cancer. There is also a high incidence of relapse with these therapies. There are estimated to be approximately 40,000 patients with myasthenia gravis in the United States. RhAFP therapy has potential to significantly reduce the current heavy treatment burden for this chronic condition.
Merrimack Pharmaceuticals, Inc. is privately held and located in Cambridge, Massachusetts.
About GTC Biotherapeutics
GTC Biotherapeutics develops, supplies and commercializes therapeutic proteins produced through transgenic animal technology. ATryn®, GTC’s recombinant human antithrombin, has been approved for use in the United States and Europe. ATryn is the first and only product produced in transgenic animals to be approved for therapeutic use anywhere in the world. In addition to ATryn, GTC is developing a portfolio of recombinant human plasma proteins with known therapeutic properties. These proteins include recombinant forms of human coagulation factors VIIa and IX, which are being developed for the treatment of hemophilia. GTC is also developing a portfolio of follow-on biologic monoclonal antibodies and a CD20 antibody with enhanced ADCC (antibody-dependent cell-mediated cytotoxicity). GTC’s intellectual property includes a patent in the United States through 2021 for the production of any therapeutic protein in the milk of any transgenic mammal. GTC’s transgenic production platform is particularly well suited to enabling cost effective development of proteins that are difficult to express in traditional recombinant production systems as well as proteins that are required in large volumes. Additional information is available on the GTC web site, http://www.gtc-bio.com.
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including without limitation statements regarding the anticipated development of rhAFP, and plans for partnering the program. Such forward-looking statements are subject to a number of risks, uncertainties and other factors that could cause actual results to differ materially from future results expressed or implied by such statements. Factors that may cause such differences include, but are not limited to, the risks and uncertainties discussed in GTC's most recent Annual Report on Form 10-K and its other periodic reports filed with the Securities and Exchange Commission, including the risks and uncertainties associated with dependence upon the actions of collaboration partners. GTC cautions investors not to place undue reliance on the forward-looking statements contained in this release. These statements speak only as of the date of this document, and GTC undertakes no obligation to update or revise the statements, except as may be required by law.
GTC Biotherapeutics, Inc.
Thomas E. Newberry, 508-370-5374
Vice President, Corporate Communications and Government Relations