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.

Iptacopan's pivotal programme 
Paroxysmal nocturnal haemoglobinuria  
Ph3 Apply-PNH (vs Soliris/Ultomiris in patients with residual anaemia following Soliris/Ultomiris) Toplined positive October 2022
Ph3 Appoint-PNH (complement inhibitor-naive pts) Data due "in coming months"
C3 glomerulopathy  
Ph3 Appear-C3G (versus placebo) Data due 2023
IgA nephropathy  
Ph3 Applause-IgAN (versus placebo) Data due 2023
Atypical haemolytic uremic syndrome  
Ph3 Appelhus (single arm, in complement inhibitor-naive pts) Data due 2024+ 
Note: earlier trials in other settings ongoing. Source: Company statements, Evaluate Pharma. 

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