Biotech firm TxCell announced that it has received orphan drug designation from the European Commission for its investigational immunotherapy Col-Treg intended for eye disease autoimmune uveitis.
Col-Treg is the company's personalized T-cell immunotherapy based on the properties of autologous collagen-II specific regulatory T lymphocytes. The cells are drawn toward areas of inflammation, where they are activated by a certain antigen. To fight this inflammation, the Col-Treg cells then release immune suppressive factors, cell-cell contacts, and cytotoxic activity. TxCell began developing Col-Treg for autoimmune uveitis in January. The immunotherapy has received Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA) earlier this year.
“The achievement of these designations for Col-Treg, our second product candidate, is a result of the strength of TxCell’s ASTrIA platform to generate innovative personalized T cell immunotherapy treatments for severe autoimmune diseases. After moving Col-Treg into a first clinical study in the first half of 2015, TxCell will then look to pursue a streamlined development program towards the market, given the very significant need for new treatments for this severe disease,” said Damian Marron, CEO of TxCell.
Autoimmune uveitis is a severe inflammatory eye condition that often causes permanent damage to vision. Though rare, uveitis is one of the major causes of blindness in the developed world. It affects around 35 to 50 per 100,000, of which autoimmune uveitis comprises 80 to 90 percent of all cases. There are currently no approved treatments for patients with autoimmune uveitis, which is resistant to treatment with corticosteroid compounds. The disease causes 30,000 new cases of blindness every year in the U.S. alone and afflicts approximately 168,000 in the EU.
Miguel Forte, SVP of clinical development and regulatory affairs at TxCell, said, “Obtaining orphan drug designation for Col-Treg in the European Union is a recognition of the therapeutic potential of Col-Treg in autoimmune uveitis… TxCell estimates around 30,000 autoimmune uveitis patients per year in the U.S. and EU alone, for whom existing treatments do not work, could benefit significantly from the development of Col-Treg.” If approved for its orphan indication, Col-Treg would secure 10 years of marketing exclusivity in the EU. The designation also confers a range of incentives on TxCell, such as discounts in regulatory fees and protocol assistance from the European Commission.
The company said it will launch a placebo-controlled, dose-ranging proof of principle study in autoimmune uveitis for Col-Treg in the first half of 2015, the results of which are anticipated by mid-2016.