(RTTNews) - Swiss drug major Novartis AG (NVS) announced Monday positive Phase II interim analysis results for iptacopan (LNP023), an investigational oral treatment for rare renal disease C3 glomerulopathy or C3G.
C3G is an ultra-rare and severe form of primary glomerulonephritis, affecting young patients with a poor prognosis and significant unmet need. The company noted that Iptacopan is a potential first-in-class, oral, potent and selective factor B inhibitor of the complement system's alternative pathway, targeting the underlying cause of C3G.
The data presented at the virtually held American Society of Nephrology (ASN) 2020 Annual Meeting shows that investigational iptacopan effectively and safely reduced proteinuria in patients with C3G 7.
Novartis added that Iptacopan also had a favorable safety and tolerability profile in this Phase II study with no deaths, no serious adverse events suspected to be related to iptacopan and no adverse events leading to treatment discontinuation.
Iptacopan is in parallel development for a number of renal conditions, including C3G, IgA nephropathy or IgAN, atypical hemolytic uremic syndrome or aHUS, and membranous nephropathy or iMN as well as in paroxysmal nocturnal hemoglobinuria or PNH, a hematological disease.
The European Medicines Agency has granted iptacopan a priority medicines designation in C3G and an orphan drug designation in IgA nephropathy.
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