Achillion Pharmaceuticals (NASDAQ:ACHN) is up 7% premarket on light volume in response to preliminary data from a Phase 2 clinical trial evaluating factor D inhibitor ACH-4471 in patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare inherited disorder characterized by the destruction of red blood cells resulting in anemia. The results were presented at The New Era of Aplastic Anemia and PNH Meeting in Naples, Italy.
Data from 11 patients at weeks 4 - 24 showed increases of mean hemoglobin of ~2.0 g/dL at week 4 (the mean rise was 2.6 g/dL in the four participants who reached week 24). Hemoglobin levels in PNH sufferers can be less than 6.0 g/dL compared to the normal range of 13.5 - 17.5 g/dL for men (12.0 - 15.5 g/dL for women).
Blood transfusions dropped from 34 totaling 58 units to one totaling two units during the treatment period.
CEO Joseph Truitt says, “Anemia is a persistent problem in the majority of patients with PNH treated with standard and even high doses of eculizumab [Alexion's (NASDAQ:ALXN) Soliris]. These interim data are encouraging and demonstrate that ACH-4471, when used in combination with a C5 inhibitor, such as eculizumab, has the potential to improve anemia, decrease transfusions and lead to improvement in important clinical parameters of hemolysis as well as quality of life measurements for patients with this devastating condition."
Subscribe for full text news in your inbox