Novartis’s Soliris challenger clears the first hurdle

There is a long list of developers coming after Astrazeneca’s blockbuster anti-C5 franchise, Soliris/Ultomiris, and Novartis’s iptacopan is considered one of the most worthy challengers. That image was burnished today with top-line results from the Apply-PNH trial, which found iptacopan to be superior to anti-C5 treatment on two co-primary endpoints. The study was in adults with paroxysmal nocturnal haemoglobinuria (PNH) who were experiencing anaemia despite prior Soliris/Ultomiris therapy – a treatment-naive study remains an important test. As do full details from Apply-PNH, which remain under wraps, with safety a big focus given the different mechanisms here. Iptacopan is a factor B inhibitor, said to work upstream of the C5 agents in the complement cascade. Novartis is also due to unveil results in other settings next year, which will also be important for shoring up some of the more bullish sales expectations out there. The Swiss pharma giant slapped a $3bn peak sales estimate on the project last month, a number that many in the sellside community believe is do-able, pending these readouts. Consensus for 2028 currently sits around $600m, making iptacopan Novartis's most valuable pipeline project, according to Evaluate Pharma.