In a news release from commercial-stage biopharmaceutical company Calliditas Therapeutics AB CALT (“Calliditas”), the company shared positive topline results from the Phase 3 NefIgArd study. Within the study, researchers examined Nefecon (TARPEYO/Kinpeygo) as a therapeutic option for individuals living with IgA nephropathy (IgAN). Nefecon is a novel oral formulation of budesonide. Administered via 4mg delayed-release capsules, this corticosteroid is designed to reduce proteinuria (excess protein in the urine) in IgAN. Nefecon is the first treatment approved directly for IgAN, providing a more targeted approach than the current standards-of-care.

The trial was split into two separate portions: Part A and B. Data from Part A allowed for Nefecon to be considered under Accelerated Approval and Conditional Marketing Authorization in Europe. Part B, alternately, focused on follow-up.

Altogether, researchers analyzed data from 364 participants with IgAN. Participants received either Nefecon or a placebo for an initial 9-month period. Next, researchers followed participants – while not on the treatment regimen – for an additional 15 months. Findings from the study show that:

• The primary trial endpoint was met. Nefecon significantly improved estimated glomerular filtration rate (eGFR), a measure of kidney function.
• Used in conjunction with RAS inhibitors, Nefecon reduced proteinuria by 31% compared to just RAS inhibitor use alone. This treatment also showed durable and sustained responses over the follow-up period.
• Nefecon was found to be safe and well-tolerated. However, some side effects did occur. These included muscle spasms, acne, unintentional weight gain, dermatitis (skin inflammation), high blood pressure, facial and peripheral edema, indigestion, shortness of breath, fatigue, and excess hair growth.

What is IgA Nephropathy (IgAN)?

Also known as Berger’s disease, IgA nephropathy (IgAN) is a rare and chronic kidney disease that occurs when immunoglobulin A (IgA), an antibody, accumulates in the kidneys. This causes kidney damage and may, down the line, progress to become kidney failure. IgAN often follows an upper respiratory or gastrointestinal tract viral infection. Some hypothesize that IgAN is an autoimmune disease, while others suspect that heredity may play a bigger role than expected. IgAN is more common in males, those of Asian or Caucasian background, or those with a family history of this condition. It also manifests most frequently between teenage years and early 30s.

Many patients are often asymptomatic in early stages. When symptoms appear, they may include:

• Hematuria (blood in the urine)
• Foamy urine from protein leakage (proteinuria)
• Drowsiness / fatigue
• Swelling in the hands and feet
• Pain in the loin and below the ribs

Treatment options have included blood pressure medications, anti-inflammatory medications, diuretics, dialysis, or kidney transplantation. As a result, Nefecon has the potential to significantly change the treatment landscape.