Novartis has announced positive data from a Phase III trial investigating iptacopan in patients with paroxysmal nocturnal haemoglobinuria (PNH). The study met its primary endpoint and demonstrated clinically meaningful benefits across secondary endpoints.

Iptacopan is a targeted factor B inhibitor that prevents intravascular and extravascular haemolysis in PNH by acting upstream of the C5 terminal pathway.

PNH is a rare blood disorder in which red blood cells break apart prematurely. Despite anti-C5s treatments, a large proportion of PNH patients remain anaemic, fatigued, and dependent on blood transfusions.

The data analysis showed that approximately 92.2% of complement-inhibitor-naïve patients with PNH treated with iptacopan achieved a 2 g/dL or more haemoglobin-level increase compared to baseline, meeting the primary endpoint. Those patients also did not require a red blood cell transfusion after 24 weeks of core treatment.

As for secondary endpoints, 62.8% of patients treated with iptacopan achieved haemoglobin levels of 12 g/dL or more without the need for red blood cell transfusions. Approximately 97.6% of patients achieved red blood cell transfusion independence at 24 weeks.

Patients also reported clinically meaningful improvements in fatigue, with an adjusted average 10.75-point increase from baseline. This endpoint was measured in the Functional Assessment of Chronic Illness Therapy – Fatigue score and it reached similar levels to those reported in the general population.

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Trial principal co-investigator Dr Antonio Risitano said: “In addition to improvement of haemolysis and fatigue seen on currently available treatments, haemolytic PNH patients treated with iptacopan achieve improvement of anaemia never seen before with anti-C5s; these data underscore the potential of iptacopan to be a practice-changing oral medicine for this devastating disease.”

The open-label, single-arm trial (NCT04820530) enrolled 40 patients who received twice-daily, oral iptacopan monotherapy.

In December 2022, Novartis announced Phase III data investigating iptacopan in an active-comparator controlled trial (NCT04558918). The 97-patient trial achieved primary and secondary endpoints showing iptacopan superiority over anti-C5 therapy.

Data from both Phase III trials will be included in global regulatory submissions. Novartis is planning to submit approval applications in the EU and US in H1 2023 and seek regulatory approval in Japan in H2 2023.

The positive trial results were announced days after Novartis revealed that the company is cutting its clinical pipeline by 10%. In a Q1 financial earnings meeting, the company said that the decision was made to narrow down the R&D focus and prioritise programmes with commercial potential and competitive landscape and around 20 early-stage projects have been discontinued.