Novartis has recently released interim data on iptacopan, according to a press release. The data on the C3 glomerulopathy treatment was released at the American Society of Nephrology 2020 Annual Meeting, which was held virtually due to the pandemic.
About C3 Glomerulopathy
C3 glomerulopathy is a group of conditions that result in kidney malfunctions. There are two major forms, which are C3 glomerulonephritis and dense deposit disease. Both forms share common symptoms, which include low protein levels in the blood, proteinuria, lower amounts of urine, and blood in the urine. Symptoms are progressive as well, and affected individuals typically enter end-stage renal disease ten years after being diagnosed. A number of various genes can mutate and result in C3 glomerulopathy, with a few examples being the C3 and CFH genes. Mutations are typically sporadic, but at times there are multiple affected family members. In terms of treatment, it is symptomatic. Doctors will prescribe medication as necessary, and they may recommend kidney transplants later in progression.
Iptacopan is an oral, investigative, small molecule reversible inhibitor of factor B. It has been indicated for a number of other renal conditions, such as IgA neuropathy and membranous neuropathy. Novartis is planning to evaluate this medication as a treatment for paroxysmal nocturnal hemoglobinuria as well.
Data on Iptacopan
The data released is in regards to an open-label Phase II study of iptacopan, in which researchers found that it was able to reduce proteinuria by 49% when compared to a placebo. Further results from this study include:
- Improved plasma C3 levels
- Stabilized renal function
- Maintained results over a six month period
- Favorable safety and tolerability profile
If results continue to remain positive, iptacopan could be a viable treatment option for those with C3 glomerulopathy.