Novartis (NYSE: NVS) reported that its oral drug iptacopan met the primary objective of the 3rd trial in some patients with paroxysmal nocturnal hemoglobinuria (PNH) in the 3rd trial.
The study, called APPOINT-PNH, evaluated iptacopan as a Standalone therapy (200-mg twice daily) in adults with PNH who have not received supportive care, including anti-C5 inhibitors – such as AstraZeneca’s.NASDAQ: AZN) Soliris (eculizumab) and Ultomiris (ravulizumab).
PNH is a rare disease in which the damaged red blood cells break down too quickly because the immune system, which is part of the immune system, attacks and destroys the red blood cells.
The Swiss pharma giant said that the data showed that a large number of patients treated with iptacopan benefited from an increase in the level of hemoglobin of 2 g / dL or more from the beginning without the need for blood transfusion at 24 weeks.
The safety profile of iptacopan was consistent with previously reported data, the company added.
It was the second successful trial of iptacopan after the drug beat Soliris and AstraZeneca’s Ultomiris in a phase III trial called APPLY-PNH, the results of which were reported in October.
“The second study of iptacopan for PNH confirms the potential of this treatment, prompting us to propose guidelines with the goal of making iptacopan the first oral treatment for PNH,” said David Soergel, global head, Cardiovascular, Renal and Metabolism. Development unit, Novartis.
Novartis added that more detailed information will be presented at an upcoming clinical meeting and included as part of the global guidelines in 2023.
The company noted that iptacopan is also being evaluated in phase III studies in advanced kidney diseases (CMKDs) C3 glomerulopathy (APPEAR-C3G), IgA nephropathy (APPLAUSE-IgAN), and atypical hemolytic uremic syndrome (APPELHUS), plus other indications in phase 2.