Novartis: Iptacopan Phase III Trial Meets Primary Goals In Adult PNH Patients With Residual Anemia

Swiss drug major Novartis AG (NVS) announced Monday positive results from Phase III APPLY-PNH trial of its investigational oral monotherapy iptacopan in adult paroxysmal nocturnal hemoglobinuria or PNH patients with residual anemia despite prior anti-C5 treatment.

PNH is a rare, chronic and serious complement-mediated blood disorder.

The trial met its two primary endpoints, showing iptacopan was superior to anti-C5 therapies (eculizumab or ravulizumab) in adults with PNH experiencing residual anemia despite prior treatment with anti-C5s.

The company will present detailed results at an upcoming medical meeting and will be included as part of global regulatory submissions in 2023.

Novartis said the topline results showed a statistically significant and clinically meaningful increase in the proportion of patients treated with iptacopan 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 primary endpoint of the study.

In addition, there was a statistically significant and clinically meaningful increase in the proportion of patients in the iptacopan arm achieving hemoglobin levels of 12 g/dL or more without the need for blood transfusions at 24 weeks, compared to anti-C5 therapies.

Iptacopan was well tolerated with a favorable safety profile consistent with previously reported data.

Novartis will continue to explore the potential of iptacopan as the first oral monotherapy option for PNH. Iptacopan is also being studied in complement-inhibitor-naïve patients with PNH in the ongoing Phase III APPOINT-PNH trial (NCT04820530), expected to read out in the coming months.

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