Novartis' iptacopan improves hemoglobin levels in rare blood disorder trial full data
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Novartis (NYSE:NVS) reported detailed results from a phase 3 trial of its oral drug iptacopan to treat certain patients with paroxysmal nocturnal hemoglobinuria (PNH).
The study, dubbed APPOINT-PNH, had met its main goal and shown clinically meaningful benefits in secondary objectives, according to the company.
The company noted that iptacopan improved hemoglobin to near-normal levels, leading to transfusion independence in all patients who had not previously received treatment.
The trial evaluated iptacopan as a standalone therapy in adults with PNH who had not received complement inhibitor therapy, including anti-C5 therapies â such as AstraZeneca's (NASDAQ:AZN) Soliris (eculizumab) and Ultomiris (ravulizumab).
PNH is a rare, acquired disorder in which defective red blood cells break apart prematurely because the complement system, part of the immune system, attacks destroys these red blood cells.
The Swiss pharma giant said that with iptacopan, an estimated 92.2% of patients achieved a 2 g/dL or more hemoglobin-level increase from baseline without the need for red blood cell transfusions after the 24-weeks of therapy.
The company added that secondary goals showed clinically meaningful benefits. An estimated 62.8% of patients achieved hemoglobin levels of 12 g/dL or more without the need for red blood cell transfusions.
In addition, about 97.6% of patients achieved red blood cell transfusion independence at 24 weeks (in contrast, 70% of patients received blood transfusions in the six months prior to treatment).
No clinical breakthrough hemolysis (BTH) events or major adverse vascular events (MAVEs) were seen, Novartis noted.
Lactate dehydrogenase (LDH) levels â a biomarker of intravascular destruction of red blood cells â decreased by 83.55% from baseline at 24 weeks, with reductions seen as early as day 7.
Novartis noted that at week 24, 95% of patients had LDH levels â¤1.5 times the upper limit of normal.
The most commonly reported adverse events were infections, mainly COVID-19 and upper respiratory tract infection. Iptacopan showed a tolerability and safety profile as seen with previous data.
This was the second successful trial of iptacopan after the medicine had shown superiority over AstraZeneca's Soliris and Ultomiris in a phase 3 study called APPLY-PNH.
Novartis plans to include data from the two studies for global regulatory submissions in H1 2023.