Novartis’ investigational treatment iptacopan – also known as LNP023 – has met the primary endpoints in both patient cohorts in a Phase II C3 glomerulopathy (C3G) clinical trial.

In the final analysis from the Phase II study, patients received iptacopan 200mg twice daily for 12 weeks, plus background therapy.

Cohort A contained patients with C3G but who have not had a kidney transplant – patients in this cohort demonstrated a 45% reduction in proteinuria compared to baseline.

Meanwhile, patients in cohort B – which included those whose C3G had returned following a kidney transplant – showed significantly reduced C3 protein deposits compared to baseline as measured by C3 deposit score.

Novartis added that both cohorts in this study showed strong and sustained inhibition of alternative complement pathway activity, as well as normalisation of serum C3 levels over 12 weeks.

“C3G is a devastating disease where people can end up facing life-altering and often exhausting kidney dialysis or transplantation at a time when they might otherwise be focused on building their lives, careers, and families. With currently no approved treatments, there is a major unmet need for therapies that can delay progression to kidney failure,” said John Tsai, head of Global Drug Development and chief medical officer at Novartis.

“These data demonstrate the ability of iptacopan to strongly and specifically inhibit the key driver for C3G – the alternative complement pathway. The results also show the potential for iptacopan to provide the first targeted treatment for people living with C3G, and we are actively recruiting for our pivotal Phase III APPEAR-C3G study,” he added.

C3G is characterised by an overly-active alternative complement pathway, which in turn causes deposits of C3 protein to build up in kidney glomeruli.

This triggers inflammation and glomerular damage that results in proteinuria, haematuria (blood in urine) and reduced kidney function.

Around 50% of C3G patients progress to kidney failure within ten years of diagnosis, and disease recurrence is not uncommon among patients who have undergone kidney transplantation.