Novartis on Monday announced that its investigational oral monotherapy iptacopan was superior to anti-C5 therapies (eculizumab or ravulizumab) in adults with paroxysmal nocturnal hemoglobinuria (PNH). The positive outcomes come from the pivotal Phase III APPLY-PNH trial which analysed the patients experiencing residual anemia despite prior treatment with anti-C5s1.
PNH is a rare, acquired, life-threatening disease of the blood and is characterized by the destruction of red blood cells, blood clots, and impaired bone marrow function.
“Topline results showed a statistically significant and clinically meaningful increase in the proportion of patients treated with iptacopan (200 mg twice daily) achieving hemoglobin-level increases of 2 g/dL or more from baseline without the need for blood transfusions at 24 weeks, compared to anti-C5 therapies, a primary endpoint of the study. Additionally, there was a statistically significant and clinically meaningful increase in the proportion of patients in the iptacopan arm achieving hemoglobin levels of 12 g/dL or more without the need for blood transfusions at 24 weeks, compared to anti-C5 therapies, also a primary endpoint of the study,” the pharma company stated on Monday.
The pharma major also claimed that Iptacopan was well tolerated with a favorable safety profile consistent with previously reported data.
“These positive topline Phase III results highlight the practice-changing potential of iptacopan for patients suffering from debilitating anemia and the burden of lifelong blood transfusions as a result of PNH. We look forward to discussing the data with regulators so we can bring this first-in-class alternative complement pathway inhibitor as the first oral monotherapy to people living with PNH,” said Shreeram Aradhye, M.D., President, Global Drug Development and Chief Medical Officer, Novartis in a statement.
According to the company, Iptacopan is also being studied in complement-inhibitor-naïve patients with PNH in the ongoing Phase III APPOINT-PNH trial (NCT04820530), expected to read out in the coming months6.
Additionally, iptacopan is being studied in Phase III trials for complement-mediated kidney diseases (CMKDs) C3 glomerulopathy (APPEAR-C3G [NCT04817618]), IgA nephropathy (APPLAUSE-IgAN [NCT04578834]), and atypical hemolytic uremic syndrome (APPELHUS [NCT04889430]), as well as in a number of additional indications in Phase II7-9, it stated.