U.S. Orphan Drug Designation Follows Recent Orphan Drug Designation in the EU
Uppsala, Sweden – November 24, 2009 – TikoMed AB, a biotechnology company focused on development and commercialization of innovative treatments for immune diseases and cell therapies, today announced that IBsolvMIR® has been granted orphan drug designation for the prevention of graft rejection of pancreatic islet transplantation by the U.S. Food and Drug Administration (FDA). IBsolvMIR is a unique new treatment which could transform islet cell transplantation into a standard treatment for severe type 1 diabetes patients with unstable diabetic conditions. TikoMed’s product IBsolvMIR improves the survival of transplanted cells by inhibiting a destructive immunological reaction and through stimulation of growth factors.
The FDA’s orphan drug designation is reserved for new therapies being developed to treat diseases or conditions that affect fewer than 200,000 people in the U.S. The orphan drug designation provides for an accelerated review process, tax benefits, exemption from user fees and a seven-year period of market exclusivity in the U.S. after product approval. Hence, IBsolvMIR will not meet any generic competition during this period and can also without user fees receive FDA assistance in the design of clinical trials.
"Receiving U.S. orphan drug designation for IBsolvMIR is extremely gratifying as there is an acute need for more treatment options for patients with severe type 1 diabetes with unstable diabetic conditions,” said Adam Bruce, Chairman of TikoMed. "Aside from the opportunity to improve quality of life for these patients, the designation provides free assistance for the execution of clinical studies, so we are making significant progress towards commercialization on the U.S. market.
IBsolvMIR is in development to improve islet cell transplantation for patients with severe type 1 diabetes. An ongoing phase II clinical study has gained a $15 million grant from the National Institutes of Health (NIH) through Uppsala University, Sweden. IBsolvMIR has the ability to improve the transplantation outcome through avoiding destruction of the insulin producing islet cells by the immune system. Successful islet transplantation has the potential to make severe type 1 diabetics insulin independent and avoid hypoglycemic crises. In a previous phase I study, IBsolvMIR was well tolerated by healthy volunteers, with no dose-limiting adverse effects documented.
"The orphan drug designation strengthens TikoMed’s competitive position as exclusive marketing rights in the U.S. are very valuable," said Anders Waas, CEO of TikoMed. "This is a complement to our patent portfolio, adding value in our discussions for collaborations to develop and commercialize IBsolvMIR in the U.S."
Swedish-based TikoMed is an innovation driven biotechnology company focused on the development and commercialization of novel treatments for cell transplantation and immune diseases. Currently, TikoMed has three products in development:
• IBsolvMIR, which in addition to islet cell transplantation also has the potential to improve the outcome of other transplantations, such as liver and bone marrow transplantations.
• TM-110, is an oral product which can eliminate IP-10, a pro-inflammatory chemokine. It provides an opportunity for safe and effective treatment of severe autoimmune diseases, where there is a high unmet need. Ongoing studies are documenting the product utility for inflammatory bowel disease (ulcerative colitis and Crohn’s disease), a $3 billion market.
• TM-120, is an apheresis product for autoimmune and inflammatory diseases. It binds and down regulates IP-10, a pro-inflammatory chemokine. It will first be developed for rheumatoid arthritis. The market for treatment of rheumatism is larger than $8 billion and there is a significant unmet need for improved treatments.
For more information, please visit www.tikomed.com.
The National Institutes of Health (NIH) — The U.S. Medical Research Agency — includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. It is the primary federal agency for conducting and supporting basic, clinical and translational medical research, and it investigates the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, please visit www.nih.gov.
Anders Waas (CEO)
Adam Bruce (Chairman of the board)