Design of stable and efficient formulation of proteins for their use as drugs has been the major focus of several biotechnology and pharmaceutical companies. In the Phase I, we developed a stable, focus of several biotechnology and pharmaceutical companies. In the Phase I, we developed a stable, active formulation of lipase from Candida rugosa, TheraCLEC-lipase, for use in the treatment of pancreatic insufficiency in cystic fibrosis. TheraCLEC-lipase exhibited characteristics much superior to commercially available pancreatic lipase products exhibiting very high activity against triglycerides and stability under low pH and towards various proteases present in the intestine. Moreover, in preliminary investigations, the TheraCLEC-lipase did not show any toxicity when animals were fed at a concentration of 5,000mg/kg-body weight. Based on these results, in Phase II, we will prepare a drug product prototype for the use in the treatment of pancreatic insufficiency, and steatorrhea in cystic fibrosis patients. As a first step, we will crystallize and crosslink the amylase for use in a final formulation. This final formulation of TheraCLEC lipase will be tested for efficacy in digestion of fat in dogs with ligated pancreatic ducts. Using radiolabeled TheraCLEC-lipase, we will follow the lack of absorption or absorption into systemic circulation. In addition, we will test the subacute, subchronic and long-term effects of feeding TheraCLEC-lipase in two species. If successful, TheraCLEC-lipase prototype will be ready to enter clinical trials, and will provide a novel potent, efficient drug for the treatment of pancreatic insufficiency in cystic fibrosis.
Currently, there are 25,000 CF patients in the US and additional 20,000 in the ret of the world. In addition, the number of alcohol-induced pancreatic insufficiency ranges from 10,000-13,000 in the US to 20,000 worldwide. The prototype TheraCLEC-lipase has enormous commercial potential over the currently available pancreatic lipase products. The worldwide market is currently $400 million with a $200 million portion being derived from US sales.
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