Novartis Experimental Drug Shows Meaningful Superiority Over Previous Treatment In Rare Blood Disorder

Novartis Experimental Drug Shows Meaningful Superiority Over Previous Treatment In Rare Blood Disorder
  • Novartis AG's NVS investigational oral monotherapy iptacopan was superior to anti-C5 therapies (eculizumab or ravulizumab) in adults with paroxysmal nocturnal hemoglobinuria (PNH). 
  • The Phase 3 APPLY-PNH trial analyzed the patients experiencing residual anemia despite prior treatment with anti-C5s1.
  • PNH is a rare blood disorder characterized by the destruction of red blood cells, blood clots, and impaired bone marrow function.
  • Topline results 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 needing blood transfusions at 24 weeks, compared to anti-C5 therapies.
  • Related: Novartis Unveils New Strategy Focused On US-First, Eight Blockbuster Brands.
  • Additionally, 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 needing blood transfusions at 24 weeks, compared to anti-C5 therapies.
  • Iptacopan was well tolerated, with a favorable safety profile consistent with previously reported data.
  • Iptacopan is also being studied in complement-inhibitor-naïve patients with PNH in the ongoing Phase 3 APPOINT-PNH trial, expected to read out in the coming months.
  • Price Action: NVS shares are up 0.32% at $77.35 during the premarket session on the last check Monday.

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