Rare Blood Disorder Treatment Breakthrough: Novartis' Candidate Improves Hemoglobin To Near-Normal Levels

Novartis AG NVS released detailed results from the Phase 3 APPOINT-PNH trial of investigational oral monotherapy iptacopan in complement-inhibitor-naïve (including anti-C5 therapies) adults with paroxysmal nocturnal hemoglobinuria (PNH). 

PNH is a rare blood disorder that leads to premature death and impaired production of blood cells.

The trial met its primary endpoint and demonstrated clinically meaningful benefits across secondary endpoints. The topline data was released in December 2022.

With iptacopan treatment, 92.2% of patients achieved a 2 g/dL or more hemoglobin-level increase from baseline without needing red blood cell transfusions after the 24-week core treatment period.

The APPOINT-PNH trial also showed clinically meaningful benefits for secondary endpoints. 62.8% of patients achieved 12 g/dL or more hemoglobin levels without red blood cell transfusions.

97.6% of patients achieved red blood cell transfusion independence at 24 weeks (in contrast, 70% received blood transfusions six months before treatment).

No clinical breakthrough hemolysis events or major adverse vascular events were observed during the 24-week trial period1.

Lactate dehydrogenase levels decreased by 83.55% from baseline at 24 weeks, with reductions seen as early as day seven. Patients also reported clinically meaningful improvements in fatigue, and iptacopan demonstrated a tolerability and safety profile.

Data from APPOINT-PNH and APPLY-PNH studies are to be included in global regulatory submissions in H1 2023.

Price Action: NVS shares are down 1.15% at $102.84 on the last check Wednesday.