Tryn® - RECOMBINANT HUMAN ANTITHROMBIN
Opinion Approved
Development/LEO
Phase III Study
EXTERNAL PROGRAM PORTFOLIO
GTC follows a partnership strategy in its portfolio of external programs, where both a unique intellectual property position and molecular biology expertise are used in the development of a transgenically produced version of the external partner’s protein. The advantages to external partners of using our production platform include enabling the development of proteins that are difficult to produce in traditional recombinant production systems, requiring significantly lower capital investment, assuring lower cost of goods, and providing for flexibility in capacity expansion.
The most advanced program in GTC’s external portfolio is with Merrimack Pharmaceuticals. The Merrimack program is for MM-093, a recombinant form of human alpha-fetoprotein, or rhAFP. Alpha-fetoprotein is a human blood protein normally produced during pregnancy and, therefore, is not commercially available from fractionation of the human blood supply. MM-093 has been difficult to express in traditional recombinant systems. GTC has developed goats for Merrimack that express this protein in their milk. In 2002, GTC agreed to expand this relationship to include production and purification of MM-093. Merrimack completed a Phase I study of MM-093 in 2003 and began Phase II evaluations in 2004, and GTC continues to deliver purified MM-093 for use in further human clinical studies by Merrimack. Potential indications for MM-093 include autoimmune diseases such as rheumatoid arthritis, multiple sclerosis and myasthenia gravis (a chronic autoimmune neuromuscular disease).
GTC has two monoclonal antibody programs with Centocor. In the first, for a transgenically produced form of Remicade®, GTC has developed founder animals which are being maintained. In the second, for an undisclosed monoclonal antibody, GTC has supplied product for preclinical evaluation by Centocor. Historically, GTC has had external partnerships with companies such as Bristol-Myers Squibb, Abbott, Elan, Progenics and Abgenix. These programs have either been successfully concluded, have entered maintenance of founder animals or are awaiting further direction.
Malaria
In collaboration with the National Institutes of Allergy and Infectious Diseases, GTC is developing a novel transgenic protein which may have medical value as a vaccine for malaria. To date, high levels of this protein have been expressed in the milk of transgenic mice. GTC has filed patent applications for technologies related to the expression of the malaria protein which would be utilized to produce a vaccine.
CD137 Antibody
GTC has entered into agreements with Mayo Clinic to begin preclinical development of an agonistic antibody to CD137 as a potential therapeutic for solid tumors. The antibody was identified and underwent earlier stage research at Mayo Clinic. The agreements define a collaborative relationship with Mayo Clinic to support GTC's development program. GTC's initial development work will be substantially funded by a recently awarded Phase I grant from the National Cancer Institute of the National Institutes of Health under the Flexible System to Advance Innovative Research For Cancer Drug Discovery by Small Business of the Small Business Innovative Research program (FLAIR-SBIR). GTC will produce and purify the CD137 antibody and Mayo Clinic will collaborate on the preclinical testing. CD137, also known as 4-1BB, is a member of the tumor necrosis factor/nerve growth factor family of receptors and is a surface glycoprotein found on certain cells of the immune system. The agonistic antibody binds to and stimulates CD137 resulting in the strengthening of the otherwise weak immune response to tumors.
The collaboration between GTC and Mayo Clinic will provide GTC with rights to any patents that may issue under the patent applications that cover the CD137 antibody. Under the agreement, GTC has two years to exercise an option for an exclusive license to these patents.