Novartis iptacopan beats AstraZeneca's Soliris/Ultomiris to treat rare blood disorder in trial
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Novartis (NYSE:NVS) said its oral drug iptacopan met the two main goal of a phase 3 trial by showing superiority over AstraZeneca's (NASDAQ:AZN) Soliris (eculizumab) and Ultomiris (ravulizumab) to treat certain adults with paroxysmal nocturnal hemoglobinuria (PNH).
PNH is a rare, acquired disorder in which red blood cells break apart prematurely.
The phase 3 trial called APPLY-PNH enrolled 97 patients and evaluated oral iptacopan monotherapy (200 mg) to treat adults with PNH who were experiencing residual anemia despite a stable regimen of anti-C5 treatment in the last six months prior to randomization.
The data showed a statistically significant and clinically meaningful increase in the proportion of patients treated with iptacopan (200 mg twice daily) achieving hemoglobin-level increases of 2 g/dL or more from baseline without the need for blood transfusions at 24 weeks, compared to anti-C5 therapies, a main goal of the study, the company said in a press release on Monday.
In addition, there was a statistically significant and clinically meaningful increase in the proportion of patients in the iptacopan group achieving hemoglobin levels of 12 g/dL or more without the need for blood transfusions at 24 weeks, compared to anti-C5 therapies, which was another main goal of the trial, according to Novartis.
"We look forward to discussing the data with regulators so we can bring this first-in-class alternative complement pathway inhibitor as the first oral monotherapy to people living with PNH," said Shreeram Aradhye, president, Global Drug Development and chief medical officer, Novartis.
The company noted that iptacopan was well tolerated with a favorable safety profile consistent with previously reported data.
Iptacopan is a targeted factor B inhibitor of the alternative complement pathway and acts upstream of the C5 terminal pathway, preventing intravascular and extravascular hemolysis in PNH, according to Novartis.