Novartis' oral therapy iptacopan shows promise in PNH

Novartis has revealed new positive data for its investigational treatment iptacopan as a monotherapy treatment for patients with the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH).

The data, presented at the Annual Congress of the European Hematology Association (EHA), showed treatment with 12 weeks of iptacopan monotherapy was generally well tolerated and resulted in ‘rapid and durable’ transfusion-free improvement of haemoglobin levels in most patients.

Novartis added that all treatment-naïve patients completing at least 12 weeks of iptacopan treatment achieved at least a 60% reduction in their lactate dehydrogenase (LDH) levels, a biomarker of intravascular haemolysis.

With the exception of one patient receiving a single red blood cell (RBC) transfusion, all patients remained transfusion-free through 12 weeks of study.

In addition, patients showed improvements across other biomarkers of haemolysis and a market increase in the proportion of PNH-type RBCs, reflecting an overall control of both intra- and extravascular haemolysis.

“PNH is a rare and life-threatening blood disorder with often debilitating symptoms,” said John Tsai, head global drug development and chief medical officer, Novartis.

“New treatment options are needed, and these positive results further strengthen the profile of iptacopan as a promising oral monotherapy. We are excited to continue to explore the potential of iptacopan as new standard-of-care treatment for PNH in the ongoing Phase III study,” he added.