Cypros Progress With Sickle Cell Drug

Cypros Pharmaceuticals has reported positive data from its Phase IIstudy of CPC-111, a novel drug treatment for the red cell distortion which occurs in sickle cell anemia.

The trial looked at the activity of a single administration of one of three doses of CPC-111 (fructose-1,6-diphosphate), an intermediate of glycolysis, in reducing pain in patients undergoing a sickle cell crisis. At all three doses, CPC-111-treated patients had lower pain scores, and this result was significant for two of the doses.

CPC-111 acts in ischemic tissue by facilitating anaerobic respiration. It delays the inhibition of glycolysis caused by the lack of oxygen and reduces the production of lactate, which can cause damage by acidification of tissue. In sickle cell anemia, CPC-111 prevents and even reverses the red cell sickling which leads to blocking of blood vessels and ischemia, the primary cause of pain in a crisis. Aside from preventing sickling, CPC-111 may also preserve the function of ischemic tissues.