Patient dosing is underway in Achillion Pharmaceuticals' (NASDAQ:ACHN) open-label proof-of-concept Phase 2 clinical trial assessing small molecule D inhibitor ACH-4471 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare life-threatening blood disease characterized by the destruction of red blood cells by the complement system.
The primary endpoint is the the change from baseline in an enzyme called serum lactate dehydrogenase (LDH) during 28 days of dosing.
ACH-4471 controls hemolysis (destruction of red blood cells) by blocking the amplification loop of the complement alternative pathway.
According to ClinicalTrials.gov, the enrollment target is four patients. The estimated study completion date is August.