A rare disease treatment being developed by Novartis outperformed two rival drugs from AstraZeneca in a late-stage study that will help support regulatory approval applications.
The treatment, called iptacopan, met both of its primary goals in the study, which compared it to the drugs Soliris and Ultomiris in adults with paroxysmal nocturnal hemoglobinuria who are experiencing anemia despite prior therapy.
The results are a win for Novartis, which discovered iptacopan through its biomedical research laboratories and has marked as a future blockbuster. If approved in paroxysmal nocturnal hemoglobinuria, or PNH, it would offer an oral alternative to the AstraZeneca drugs Soliris and Ultomiris, which are widely used to treat the serious blood disorder.
No specific data were included in the Novartis statement Monday announcing the positive trial outcome. The company said it would disclose detailed results at an upcoming medical meeting.
The trial, which enrolled 97 PNH patients, was designed to test how well the three drugs helped increase levels of hemoglobin — a crucial oxygen-carrying protein — in the absence of blood transfusions. It also measured how many patients on each drug achieved “sustained” hemoglobin levels above a certain amount at six months.
Iptacopan, which targets a protein called factor B, was superior to Soliris and Ultomiris on both endpoints, Novartis said. Side effects were “well tolerated” and consistent with prior testing, according to the company.
Both Soliris and Ultomiris work a different way, blocking a crucial protein known as C5 that’s part of body’s complement system. Novartis’ study specifically enrolled patients whose disease was not fully controlled despite taking anti-C5 drugs. Another, ongoing trial with results due in the coming months is testing iptacopan in PNH patients who have never taken C5 inhibitors before.
Novartis describes factor B as “upstream” of C5 in the protein signaling cascade that gets overactivated in PNH, which involves the premature destruction of red blood cells and results in anemia, fatigue and blood clots. Treatment with iptacopan is meant to calm that overactivation and prevent red blood cell destruction.
While PNH is rare, the drugs for it are a big business. Alexion, which originally developed Soliris and Ultomiris, turned both into billion-dollar products, due in particular to setting high prices for each. Their commercial success was a major draw for AstraZeneca, which bought Alexion at the end of 2020 for $39 billion.
Novartis has forecast sales of iptacopan reaching beyond $2 billion a year, and is developing it for an array of other complement-mediated kidney diseases including C3 glomerulopathy, IgA nephropathy and atypical hemolytic uremic syndrome.
AstraZeneca is developing a successor drug to Soliris and Ultomiris, as well as a trio of oral compounds that target another protein called factor D.