Recently, biopharmaceutical company Everest Medicines (“Everest”) announced positive topline data from the Phase 3 NefIgArd trial, which was run by its licensing partner Calliditas Therapeutics AB (“Calliditas”). Together, the companies seek to find treatments for patients with rare conditions in China and throughout Asia. The Phase 3 trial evaluated Nefecon as a potential treatment for patients with primary IgA nephropathy (IgAN).

NefIgArd Clinical Trial

Altogether, 199 patients enrolled in the clinical trial. During the trial, researchers analyzed the impact of Nefecon on patients with IgAN. Nefecon is an orally-administered budesonide formulation. When administered, Nefecon delivers the treatment directly to the Peyer’s patch, an area of the ileum. Once delivered, Nefecon mediates IgA production.

During the clinical trial, researchers found that Nefecon reduced proteinuria, or excess protein in the urine. Additionally, it improved and stabilized estimated glomerular filtration rate (eGFR). Overall, Nefecon showed significant improvement for a sustained time (1 year), and significant improvement compared to a placebo. The treatment was also safe and well-tolerated. While not yet approved for use, Nefecon received Orphan Drug Designation in the United States, and Orphan Medicinal Product designation in Europe.

Ultimately, Calliditas hopes to submit the drug application to the FDA and EMA for accelerated approval in 2021. Finally, Everest will enroll 160 patients for a Phase 3 clinical trial to continue evaluating the efficacy of Nefecon.

IgA Nephropathy (IgAN)

Also known as Berger’s disease, IgA nephropathy (IgAN) is a chronic and progressive kidney disease. It occurs when a protein called immunoglobulin A (IgA) accumulates in the kidneys. Normally, IgA plays a role in immune response and protects the body from “foreign invaders.” But when too much accumulates, it prompts inflammation and kidney harm. As a result, kidney function lowers as the kidneys struggle to filter out waste. Additionally, blood and protein begin leaking into the urine, causing further damage. Generally, IgAN progresses to become kidney failure. Researchers are not exactly sure what causes IgAN. There is some debate over whether it is caused by viral infections, genetics, or the immune system. Males are more likely to have IgAN than females.

While symptoms may not appear for years, the following are what patients might expect:

• High blood pressure
• Bloody or foamy urine
• Fatigue
• Drowsiness
• Proteinuria (excess protein in the urine)
• Pain, particularly in the groin, sides, and back
• Swelling of the hands and feet
• Flu-like symptoms

Learn more about IgAN here.