Novartis on Thursday announced that its investigational oral monotherapy iptacopan has met its primary endpoint in the Phase III clinical trial in patients with paroxysmal nocturnal hemoglobinuria (PNH).
PNH is a rare, acquired, life-threatening disease of the blood. The disease is characterised by the destruction of red blood cells, blood clots, and impaired bone marrow function.
According to the company, during the trial a significant proportion of patients treated with iptacopan (200 mg twice daily) achieved clinically meaningful hemoglobin-level increases of 2 g/dL or more from baseline without the need for blood transfusions at 24 weeks.
In the study, the safety profile of iptacopan monotherapy was consistent with previously reported data. Detailed data will be presented at an upcoming medical meeting and included as part of global regulatory submissions in 2023, the company stated.
“We are very encouraged by the results of the complement-inhibitor-naïve data from the Phase III APPOINT-PNH trial. This second iptacopan readout for PNH underscores the robust potential for this therapy, enabling us to submit a broad regulatory package with the goal of iptacopan potentially becoming the first oral monotherapy for PNH,” said David Soergel, M.D., Global Head, Cardiovascular, Renal and Metabolism Development Unit, Novartis in a statement.
Moreover, iptacopan is also being investigated in Phase III studies for complement-mediated kidney diseases (CMKDs), IgA nephropathy, and atypical hemolytic uremic syndrome, as well as in a number of additional indications.