- Statistically significant and clinically important reductions in proteinuria were achieved for the primary endpoint for patients with C3G1
- Additionally, statistically significant reduction in C3 protein deposits were achieved in the same study in a cohort of patients whose C3G recurred following kidney transplantation1
- No current approved treatments exist for C3G – a rare and often progressive disease that often affects adolescents and young adults and frequently progresses to kidney failure2-4
- Novartis is rapidly advancing clinical development of iptacopan to potentially address several complement-driven renal diseases (CDRDs) with high unmet need, as part of our wider commitment to cardiovascular, renal and metabolic disease; pivotal Phase III APPEAR-C3G study is actively recruiting
Novartis moment blazoned that a Phase II study of investigational iptacopan (LNP023) – a first-in- class, oral, picky factor B asset – in cases with C3 glomerulopathy (C3G) met primary endpoints in both patient cohorts1. The data were presented at the American Society of Nephrology (ASN) 2021 Annual Meeting.
In the final analysis from the open- marker, two- cohort Phase II study (NCT03832114), cases were treated with 200 mg of iptacopan doubly daily for 12 weeks, in addition to background therapy1. Cases in cohort A (16 with C3G, but who haven’t had a order transplant ( native C3G)) showed a significant 45 reduction in proteinuria (protein in urine) compared to birth, as measured by 24-hour urinary protein to creatine rate (UPCR 24h; P = 0.0003) 1.
“ The data presented at ASN give a detailed picture of the eventuality of iptacopan for the treatment of cases with C3G and, for the first time, in cases whose C3G had returned following order transplantation,” said supereminent study investigator Edwin Wong, Adviser Nephrologist at the National Renal Complement Rectifiers Centre, Newcastle upon Tyne NHS Foundation Trust, Newcastle University, UK. “ These results are important for cases with C3G because proteinuria is a crucial threat predictor for order complaint progression, and deposits of C3 protein eventually beget inflammation and order damage.”
“ C3G is a ruinous complaint where people can end up facing life- altering and frequently exhausting order dialysis or transplantation at a time when they might else be concentrated on erecting their lives, careers, and families. With presently no approved treatments, there’s a major unmet need for curatives that can delay progression to order failure, “ said John Tsai, Head of Global Drug Development and Chief Medical Officer at Novartis. “ These data demonstrate the capability of iptacopan to explosively and specifically inhibit the crucial motorist for C3G – the indispensable complement pathway. The results also show the eventuality for iptacopan to give the first targeted treatment for people living with C3G, and we’re laboriously retaining for our vital Phase III APPEAR-C3G study.
About the study
NCT03832114 is a Phase II, open- marker, two cohort,non-randomized study assessing the efficacity, safety and pharmacokinetics of iptacopan in cases with C3 glomerulopathy (C3G) ( cohort A) and cases who have experienced order transplantation and have posterior C3G rush in the transplanted organ ( cohort B). The primary endpoint for cohort A was reduction in proteinuria (as measured by UPCR 24h) from birth to week. The primary endpoint for cohort B was change in C3 deposit score ( grounded on immunofluorescence microscopy) from order vivisection from birth to week. On completion of the study, all cases had the option to admit ongoing iptacopan in a long- term extension study (NCT03955445) 1.
Iptacopan is an investigational, first-in- class, orally administered factor B asset of the indispensable complement pathway, targeting one of the crucial motorists of CDRDs8-10. It’s the most advanced asset in the Novartis nephrology channel and has the implicit to come the first targeted remedy to delay progression to dialysis in C3G9. Discovered at the Novartis Institutes for BioMedical Research, iptacopan is presently in development for a number of CDRDs where significant unmet requirements live, including C3G, IgA nephropathy (IgAN), atypical hemolytic uremic pattern (aHUS), and idiopathic membranous nephropathy (iMN), as well as the blood complaint ferocious nightly hemoglobinuria (PNH).
Phase III studies in IgAN ( APPLAUSE-IgAN) and aHUS (APPELHUS), and a Phase II study in iMN, are laboriously retaining. Two Phase III studies in PNH ( APPLY-PNH and APPOINT-PNH) are also laboriously retaining. Grounded on complaint frequence and positive data from Phase II studies, iptacopan has entered EMA PRIME designation for C3G, orphan medicine designations from the FDA and EMA in C3G and PNH, EMA orphan medicine designation in IgAN, and FDA Advance Remedy Designation in PNH.
About C3 glomerulopathy (C3G) and complement- driven renal conditions (CDRDs)
In C3G, an exorbitantly-active indispensable complement pathway – part of the ingrain vulnerable system – causes deposits of C3 protein to make up in order glomeruli (a network of blood vessels that filter waste and remove redundant fluids from the blood)-13. This triggers inflammation and glomerular damage that results in proteinuria (protein in urine), hematuria ( blood in urine) and reduced order-13. Roughly 50 of C3G cases progress to order failure within 10 times of diagnosis2,. Among cases who have experienced order transplantation, complaint rush isn’t uncommon, with one study seeing an estimated 30 and 70 threat of transplant loss at 5 and 10 times,-16.
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