Slingshot Insights

Clinical Data ALXN1210, a Long-Acting C5 Inhibitor, Results in Rapid and Sustained Reduction of LDH with a Monthly Dosing Interval in Patients with PNH: Preliminary Data from a Dose-Escalation Study (Abstract LB2247)2In a poster session, interim results were presented from a Phase 1/2, open-label, 24-week dose-escalating study of ALXN1210 in patients with PNH. The primary efficacy endpoint was the percent change in LDH levels from baseline; other efficacy endpoints included change in blood transfusion requirements and change in hematologic parameters from baseline. Patients with PNH (aged 18 and older; n=13) with mean LDH levels ≥3 times the upper limit of normal and who were complement inhibitor-naïve were separated into two study cohorts. Patients in Cohort 1 (n=6) received either 400 mg or 600 mg induction doses of ALXN1210, followed by a 900 mg maintenance dose once-monthly. Patients in Cohort 2 (n=7) received 600 mg and 900 mg induction doses of ALXN1210, followed by an 1,800 mg maintenance dose once-monthly.All patients showed rapid reductions in mean LDH levels at Day 8 (the first evaluable time point of the study), which were sustained for up to five once-monthly dosing intervals. At the most recent evaluable time point, the mean percentage reduction in LDH levels from baseline was 85.4 percent in Cohort 1 (Day 148) and 86.0 percent in Cohort 2 (Day 85). Among five patients with one or more transfusions in the year prior to the study, only one patient, from Cohort 1, required a transfusion during treatment with ALXN1210. This patient received two units of packed red blood cells (RBC) while receiving ALXN1210, compared to 12 units of RBC in the six months prior to ALXN1210. In addition, mean levels of hemoglobin, another direct marker of intravascular hemolysis, were improved or stable in both cohorts."PNH is a devastating, ultra-rare blood disorder caused by uncontrolled activation of complement, putting patients at risk for severe and life-threatening consequences," said lead author Jong-Wook Lee, M.D., of The Catholic University of Korea, Seoul St. Mary's Hospital, Seoul, Korea. "The interim data presented at EHA suggest that treatment with ALXN1210 results in effective blockade of complement-mediated hemolysis and reduces transfusion requirements in patients with PNH. All patients achieved rapid decreases in LDH levels that were sustained through extended, once-monthly dosing intervals, consistent with the longer half-life of ALXN1210."No serious adverse events or study withdrawals were observed in either patient cohort. The most common treatment-related adverse events were headache and upper respiratory tract infection (each occurring in 3 patients), which resolved during ongoing treatment with ALXN1210.

Catalyst

Do you think this event is important to the companies below? How will it affect their stock price?

Additional Information

Slingshot Insights Explained