CLINICAL UPDATE | Novartis Builds Case for Iptacopan as Oral Rival to PNH Injectables
Novartis has a second positive phase 3 trial for its targeted factor B inhibitor iptacopan, seeking to become an oral alternative to injectable therapies for ultra-rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH).
On December 8, the Swiss pharma group announced that the top-line results of the APPOINT-PNH clinical trial showed that iptacopan provided “clinically meaningful” increases in haemoglobin levels in patients with PNH who had not previously been treated with injectable complement C5 inhibitors like AstraZeneca/Alexion’s Soliris (eculizumab) and Ultomiris (ravulizumab).
In the trial, a significant proportion of patients treated with iptacopan (200 mg twice daily) achieved clinically meaningful haemoglobin-level increases of 2 g/dL or more from baseline without the need for blood transfusions at 24 weeks.
The earlier APPLY-PNH study – reported in October – was carried patients who were still experiencing residual anaemia despite prior treatment with anti-C5 antibodies and showed that iptacopan was more effective than the C5 inhibitors at helping them to meet target haemoglobin levels.
Armed with both trial results, Novartis says it now intends to press ahead with plans to file for regulatory approval of iptacopan next year.
In PNH, the body’s complement system destroys red blood cells leading to anaemia, fatigued and in some cases needing blood transfusions. It is estimated that approximately 10-20 people per million worldwide live with PNH. It can develop at any age, though is often diagnosed in people between 30-40 years old.
PNH has a significant unmet need not addressed by anti-C5 therapies (eculizumab or ravulizumab): despite treatment with anti-C5s, a large proportion of people with PNH remain anemic, fatigued and dependent on blood transfusions2-5.
