Data from the open-label Phase II study (NCT03832114), showed that after 12 weeks, iptacopan significantly reduced proteinuria by 49% compared to baseline values, as measured by 24-hour urine protein/creatinine ratio (UPCR) assessment, in twelve patients with C3G (P=0.0005). Iptacopan strongly inhibited alternative complement pathway activity and improved plasma C3 levels. In addition, iptacopan stabilized renal function as assessed by eGFR (estimated glomerular filtration rate) at week 12 and this effect was maintained in the seven patients that were treated for a total of six months after rolling over into the long-term extension study (NCT03955445)7.
'Proteinuria indicates the presence of inflammation in the kidney. Results from this study demonstrate that iptacopan significantly reduces proteinuria in patients with C3G,' said the lead study investigator, Dr
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 the most advanced asset in our nephrology pipeline.' said
The
About C3 glomerulopathy (C3G)
C3G is an ultra-rare and severe form of primary glomerulonephritis, characterized by complement dysregulation.1,9 It has a worldwide annual incidence of 1-2 per million11 and an approximate prevalence of 10,000 in the US, 10,500 in
C3G is commonly diagnosed in adolescents and young adults. The disease has a poor prognosis; about 50% of patient progress to end-stage renal disease (ESRD) within 10 years, and 50-70% experience disease recurrence post kidney transplant.2
Currently, there are no approved therapies specifically designed to target the underlying complement dysregulation that occurs in people with C3G. Current standard of care is with non-specific immunosuppressants with limited clinical evidence. Although antihypertensive or immunosuppressive agents and terminal complement pathway blockers are helpful in some patients, no treatment is universally effective or curative. 2,3,9,10 Therefore, novel treatment options are needed to address disease symptoms and slow the progression of C3G.
About iptacopan
Iptacopan (LNP023) is a first-in-class oral, small-molecule, reversible inhibitor of factor B, a key serine protease of the alternative pathway of the complement cascade.4,5
In addition to C3G, iptacopan is in parallel development for a number of other renal conditions with complement system involvement where significant unmet needs exist, including IgA nephropathy, atypical hemolytic uremic syndrome and membranous nephropathy.
Novartis is also investigating iptacopan in paroxysmal nocturnal hemoglobinuria (PNH). Following positive Phase II data presented at the
Iptacopan has the potential to become the first alternative complement pathway inhibitor to slow disease progression in a number of complement driven diseases.
About the Study
The study (NCT03832114) is a Phase II, open-label, two cohort, non-randomized study evaluating the efficacy, safety and pharmacokinetics of iptacopan in patients with C3 glomerulopathy (C3G) (Cohort A) and patients who have undergone kidney transplant and have C3G recurrence (Cohort B).14 On completion of the study, patients can roll over into a long-term extension study (NCT03955445).
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About Novartis
Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world.
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