Novartis: Phase III APPOINT-PNH Study Of Oral Monotherapy Iptacopan Meets Primary Endpoint

Swiss drug maker Novartis AG (NVS) announced Thursday that Phase III APPOINT-PNH study (NCT04820530) of investigational oral monotherapy iptacopan in complement-inhibitor-naïve (including anti-C5 therapies) adults with PNH met its primary endpoint.

The primary endpoint was to assess the proportion of participants achieving an increase in hemoglobin levels from baseline.

The topline results showed a significant proportion of patients treated with iptacopan achieved clinically meaningful hemoglobin-level increases of 2 g/dL or more from baseline without the need for blood transfusions at 24 weeks.

The company noted that the safety profile of iptacopan monotherapy in the trial was consistent with previously reported data.

Novartis will present detailed data at an upcoming medical meeting and will include as part of global regulatory submissions in 2023.

David Soergel, Global Head, Cardiovascular, Renal and Metabolism Development Unit, Novartis, said, “This second iptacopan readout for PNH underscores the robust potential for this therapy, enabling us to submit a broad regulatory package with the goal of iptacopan potentially becoming the first oral monotherapy for PNH.”

The company recently announced topline results for the pivotal Phase III APPLY-PNH study that met its two primary endpoints, with iptacopan demonstrating superiority over anti-C5 therapies in adults with PNH experiencing residual anemia despite prior anti-C5 treatment.

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