Christopher Patriquin, MD, University of Toronto, discusses results of a clinical trial comparing combination C5 inhibitor therapy versus standard of care in patients with paroxysmal nocturnal hemoglobinuria (PNH).
PNH is a rare clonal hematopoietic stem cell disorder characterized by corpuscular hemolytic anemia, bone marrow failure and frequent thrombotic events.
The ACCESS-1 clinical trial is a phase 3, randomized, open-label study evaluating the safety and efficacy of pozelimab and cemdisiran (poze-cemdi) in patients with PNH. Poze-cemdi is a first-in-class combination therapy of an antibody and siRNA targeting C5. The combination therapy was compared to standard of care treatment ravulizumab, a complement C5 inhibitor.
The primary endpoint of cohort A in the study was percent change in lactate dehydrogenase (LDH) levels at 26 weeks. At the end of the study, 96% of patients treated with poze-cemdi achieved adequate LDH control compared to 80% with ravulizumab. 93% of patients achieved LDH normalization with poze-cemdi compared to 65% with ravulizumab. Patients treated with poze-cemdi experienced an 84% decrease in LDH from baseline at week 26 compared to 74% with ravulizumab. Additionally, the CH50 profile observed with poze-cemdi demonstrated complete and uninterrupted inhibition of terminal complement.
Additional data from the ACCESS-1 open-label extension following week 26 included a 95% achievement of LDH control in patients who switched from ravulizumab, compared to 68% that had adequate LDH control with ravulizumab. The safety profile of poze-cemdi was consistent with other approved C5 inhibitors.
For more information, click here.
To learn more about PNH and other rare hematologic disorders, visit https://checkrare.com/diseases/hematologic-disorders/