The American Society of Nephrology held its annual Kidney Week from November 3-6, 2022. During this week, multiple stakeholders within the nephrology sphere met to discuss trends, clinical care practices, research, and more. One presentation during ASN Kidney Week came from Ionis Pharmaceuticals, Inc., a biotechnology company working to develop RNA-targeted therapeutics. Their presentation shared data from a Phase 2 study evaluating IONIS-FB-LRx for IgA nephropathy (IgAN). 

According to a news release on Pipeline Review, the study included 25 participants living with IgAN. The data presented at Kidney Week centered around ten patients within the study’s first cohort; these patients had received IONIS-FB-LRx treatment for a 29-week period. Researchers were evaluating kidney function, safety, tolerability, and urine protein excretion over a 24-hour period. 

Ultimately, the study found that this treatment option was relatively effective, safe, and well-tolerated. It contributed to a 44% mean reduction in urine protein excretion and helped strengthen kidney function. 

Ionis describes IONIS-FB-LRx as: 

a ligand-conjugated investigational antisense medicine designed to reduce the production of complement factor B, a key regulatory protein…produced predominantly in the liver and [circulated] at high levels throughout the vascular system, including in the kidney. Genetic association studies have shown that overaction of this cascade has been associated with the development of several complement-mediated diseases, including IgA nephropathy (IgAN).

Looking towards the future, Ionis will continue evaluating this therapy in Phase 3 studies. 

What is IgA Nephropathy (IgAN)? 

Also known as Berger’s disease, IgA nephropathy (IgAN) is a rare and chronic form of kidney disease characterized by the accumulation of an antibody called immunoglobulin A (IgA) in the kidneys. This buildup attacks the glomeruli, which are the parts of the kidney that filter waste and remove extra fluid from the blood. The exact cause of IgAN is not fully understood. However, the condition often follows an upper respiratory or gastrointestinal tract virus infection. 

IgAN is most common in late teens to early 30s. Men are 2x more likely to develop IgAN than women. Being Asian or Caucasian or having a family history of IgAN also increases the risk of developing it. In this condition, symptoms may not manifest until later stages. These symptoms can, but do not always, include:

• Swelling of the hands and feet
• Hematuria (blood in the urine)
• Loin and back pain
• Fatigue
• General itchiness 
• Dry skin
• Appetite loss
• Nausea and vomiting
• Difficulty concentrating
• High blood pressure 
• Drowsiness
• Proteinuria (excess protein in the urine)
• Kidney failure