Achillion rises after reporting proof-of-concept data for factor D inhibitor
Shares of Achillion (ACHN) are higher in pre-market trading after the company said that it "believes" it has achieved the goal of achieving proof-of-concept for ACH-4471 via factor D inhibition. DATA: Last night, Achillion announced data from a phase 2 three-month, dose-ranging trial with ACH-4471 for patients with untreated PNH, a rare, acquired, life-threatening disease characterized by destruction of red blood cells, blood clots, impaired bone marrow function, and a risk of developing leukemia.. To date, Achillion has data for four patients with PNH, two of whom have completed the three-month trial and have entered the long-term extension trial. One additional patient continues to receive dosing in the three-month trial and a fourth patient voluntarily withdrew from the trial on day 41 for reasons unrelated to safety. In summary, interim data from these ongoing trials demonstrated that ACH-4471 achieved clinically meaningful complement inhibition and demonstrated a favorable tolerability profile with no reports of clinically meaningful increases in liver enzymes. In this emerging data set, ACH-4471 has improved LDH, hemoglobin, fatigue score and other measures of response including PNH clone size. These interim results support the company's global expansion plans for the PNH clinical program. "Our focus in early clinical development with ACH-4471 has been on achieving proof-of-concept via factor D inhibition, and we are pleased to report that we believe we have achieved this goal. The emerging interim results from our phase 2 PNH trial have demonstrated a dose response to treatment with what we believe to be meaningful improvements in LDH, hemoglobin, fatigue score and other markers of response. To date, orally administered ACH-4471 has been well tolerated in this PNH clinical trial with four patients enrolled and treated with ACH-4471, two of whom have now received more than four months of dosing," said CEO Milind Deshpande. PRICE ACTION: In pre-market trading, Achillion shares are up 79c, or about 20%, to $4.80.