Interim analysis results for Novartis’ rare renal disease candidate iptacopan proved promising in a phase II study of the investigational drug.

The oral investigational candidate was evaluated as a treatment for C3 glomerulopathy (C3G) – a rare renal disease affecting young patients which carries a poor prognosis with a high unmet medical need.

The data, presented at the virtual American Society of Nephrology (ASN) 2020 annual meeting, from the phase II study of iptacopan showed that after 12 weeks, the drug significantly reduced proteinuria by 49% compared to baseline values in 12 patients with C3G.

“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 Edwin Wong, lead study investigator, consultant nephrologist at the National Renal Complement Therapeutics Centre, Newcastle upon Tyne NHS Foundation Trust, Newcastle University.

“This data also highlights iptacopan’s ability to strongly and specifically inhibit the alternative complement pathway, targeting the underlying cause of this disease and potentially providing a much needed treatment option for C3G patients who have significant unmet needs,” he added.

In addition, iptacopan stabilised renal function as assessed by estimated glomerular filtration rate (eGFR) 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 expansion study.

The European Medicines Agency (EMA) has already granted Novartis’ drug a PRIME designation in C3G. The EMA has also granted an orphan drug designation for iptacopan in IgA nephropathy (IgAN).

Alongside C3G, iptacopan is in parallel development for a number of other renal conditions with complement system involvement where significant unmet needs still exist.

This includes IgAN, atypical haemolytic uremic syndrome and membranous nephropathy, as well as paroxysmal nocturnal haemoglobinuria (PNH).