Novartis iptacopan comes out successful in 2nd phase 3 trial rare blood disorder
Robert Way

Novartis (NYSE:NVS) said its oral drug iptacopan met the main goal of a phase 3 trial in certain patients with paroxysmal nocturnal hemoglobinuria (PNH) in a phase 3 trial.
The study, dubbed APPOINT-PNH, evaluated iptacopan as a standalone therapy (200-mg twice daily) in adults with PNH who had not received complement inhibitor therapy, including anti-C5 therapies â such as AstraZeneca's (NASDAQ:AZN) Soliris (eculizumab) and Ultomiris (ravulizumab).
PNH is a rare, acquired disorder in which defective red blood cells break apart prematurely because the complement system, a part of the immune system, attacks and destroys these red blood cells.
The Swiss pharma giant said that data showed a significant proportion of patients treated with iptacopan achieved clinically meaningful hemoglobin-level increases of 2 g/dL or more from baseline without the need for blood transfusions at 24 weeks.
The safety profile of iptacopan was consistent with previously reported data, the company added.
This was the second successful trial of iptacopan after the drug had shown superiority over AstraZeneca's Soliris and Ultomiris in a phase 3 trial called APPLY-PNH, results from which were reported in October.
"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, global head, Cardiovascular, Renal and Metabolism Development unit, Novartis.
Novartis added that detailed data will be presented at an upcoming medical meeting and included as part of global regulatory submissions in 2023.
The company noted that iptacopan is also being evaluated in phase 3 studies for the complement-mediated kidney diseases (CMKDs) C3 glomerulopathy (APPEAR-C3G), IgA nephropathy (APPLAUSE-IgAN), and atypical hemolytic uremic syndrome (APPELHUS), plus in certain other indications in phase 2.