Achillion reports positive interim Phase 2 data in paroxysmal nocturnal hemoglobinuria


Achillion Pharmaceuticals (NASDAQ:ACHN) reported interim data from its Phase 2 trial of ACH-4471, in combination with eculizumab, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

In PNH, the complement system destroys red blood cells in both the circulatory – or intravascular system – and extravascular systems such as the liver or spleen, leading to anemia. Complement D factor in particular, is believed to play a role in the destruction of extravascular red blood cells.

The Phase 2 trial is testing the safety and efficacy of ACH-4471, an orally-administered factor D inhibitor, in combination with intravenous eculizumab, a complement C5 inhibitor. Interim data demonstrated increases in mean hemoglobin of 2 grams per deciliter (g/dL) at week 4 in 11 patients; and 2.6 g/dL at week 24 in four patients. The number of blood transfusions was reduced from 34 to one during treatment.

“Anemia is a persistent problem in the majority of patients with PNH treated with standard and even high doses of eculizumab,” Joseph Truitt, Achillion’s CEO, said in a statement.

“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,” he added.

Abby Hardy