Target C3 nephropathy (C3G) the underlying cause! Novaral's powerful selective factor B inhibitor iptacopan significantly reduces proteinuria levels!

October 26, 2020 // -- Novartis recently announced positive results from the interim analysis of the interim analysis of the treatment of C3 renal ocococytic disease (C3G) open label (NCT03832114) by iptacopan (LNP023).
data from 12 C3G patients showed that after 12 weeks of treatment, iptacopan significantly reduced proteinuria by 49% compared to baseline values using a 24-hour urine protein/creatinine ratio (UPCR) assessment.
significantly inhibited the activity of complement replacement pathways and improved plasma C3 levels.
In addition, iptacopan stabilized renal function through eGFR (estimated cystic filtration rate) in week 12, which was maintained in a total of 7 patients who received treatment for a total of six months after being transferred to the Long-Term Extension Study (NCT03955445).
in this Phase II study, iptacopan also showed good safety and tolerance, no patient deaths, no suspected serious adverse events associated with iptacopan, and no adverse events that led to discontinuation of treatment. Lead investigator on the
study, Dr Edwin Wong, consultant nephrologist at newcastle University's National Kidney SupplementAl Therapy Centre and Newcastle-Anton-Tyne NHS Foundation Trust, said: "Proteinuria indicates inflammation in the kidneys and the results of this study suggest that iptacopan significantly reduces proteinuria in patients with C3G.
the data also highlight the underlying causes of iptacopan's ability to strongly and specifically inhibit complementary alternatives, target the disease, and potentially provide a much-needed treatment for C3G patients, who have significant unsuperial medical needs.
, Novarma's global head of drug development and chief medical officer, said: "Iptacopan is the most advanced asset in our nephrology pipeline.
data show that the drug has the potential to improve the lives of patients with C3G.
"iptacopan chemical structure (Photo source: medchemexpress.cn) iptacopan is a pioneering (first-in-class), oral, powerful, selective, small molecule, reversible B-factor inhibitor, B-factor is the key to the replacement pathway of the complement system serine protease.
iptacopan can target the underlying cause of C3 nephropathy (C3G).
recently, the European Medicines Agency (EMA) granted IPtacopan Priority Drug Qualification (PRIME).
C3G is an extremely rare and serious primary ngnephritis characterized by a rehydration disorder.
the annual incidence of the disease worldwide is 1-2 per million, about 10,000 in the United States, about 10,500 in Europe, about 3,200 in Japan and about 32,000 in China.
C3G is usually diagnosed in adolescents and young adults, and the prognosis is poor, with about 50% of patients developing end-stage kidney disease (ESRD) within 10 years and 50-70% relapsing after kidney transplantation.
addition to C3G, iptacopan is also being developed in parallel to treat a number of other kidney diseases that are suffering from adjunct systems and have significantly unsolvent needs, including IgA nephropathy (IgAN), atypical hemolytic uremia syndrome, and membrane nephropathy.
addition, Novaral is also studying the efficacy of iptacopan in treating hairy sleep-related hemoglobinuria (PNH).
Based on Phase II positive data presented at the European Society for Blood and Bone Marrow Transplant (EBMT) conference on August 10, Novarma plans to begin a randomized, positive drug-controlled, open-label Phase III trial in December 2020 to assess the efficacy and safety of iptacopan in patients with PNH (despite receiving anti-C5 antibody therapy).
in many supplement-driven diseases, iptacopan has the potential to be the first alternative pathway inhibitor to slow disease progression.
based on preliminary data from Phase 2 trials, iptacopan has been granted ODD status by the FDA and the European Union's EMA for the treatment of C3G orphan drugs.
() Original origin: Novartis presents offers interim Phase II data of potential first-in-class oral therapy iptacopan (LNP023) in rare renal disease C3 glomerulopathy (C3G)