Nefecon Phase 3 Trial Data at 2024 ISN Nephrology Congress

On April 18, 2024, Calliditas Therapeutics, a biopharmaceutical company, unveiled further data analyses from the NeflgArd trial at the ISN World Congress of Nephrology. The NeflgArd trial is a comprehensive study that assessed the efficacy of Nefecon (TARPEYO®/budesonide delayed-release capsules/Kinpeygo®) in treating IgA nephropathy (IgAN), a chronic autoimmune kidney disease.

The study, which spanned two years, included a diverse patient population and provided compelling evidence of Nefecon's effectiveness. Richard Philipson, Calliditas' Chief Medical Officer, stated that the data underscore Nefecon's positive impact on all participants, regardless of their baseline urine protein-to-creatinine ratio (UPCR) levels or ethnic backgrounds.

The trial's findings were presented through various posters, highlighting several key aspects:

Kidney Function Improvement: Nefecon demonstrated a consistent enhancement in the estimated glomerular filtration rate (eGFR) across the entire study cohort when compared to a placebo. Notably, patients with a baseline UPCR below 0.8 g/g showed sustained eGFR improvement that lasted up to 18 months post-treatment initiation.

UPCR Reduction and Kidney Function Preservation: Over the 9-month treatment duration, Nefecon led to a 30% reduction in UPCR, an effect that was maintained for two years. The study also indicated a significantly reduced risk of kidney function decline in patients treated with Nefecon.

Quality of Life: The trial assessed the impact of Nefecon on the quality of life using the 36-Item Short Form Survey (SF-36). No significant differences were observed between the Nefecon and placebo groups after nine months of treatment, and these results were consistent during the subsequent off-drug follow-up period.

Ethnicity and Efficacy: The Nefecon treatment response was evaluated in both Asian and White patient groups, showing favorable eGFR changes and significant reductions in UPCR, with delays in kidney function decline events, irrespective of race and ethnicity.

TARPEYO, the drug under investigation, is specifically formulated to target Peyer's patches in the ileum, which are responsible for producing the galactose-deficient IgA1 antibodies that cause IgAN.

The NeflgArd trial's primary endpoint was the time-weighted average of eGFR over two years, which showed a statistically significant benefit for TARPEYO over placebo. The drug's positive effect on eGFR was evident by the third month and persisted throughout the study.

However, it is important to consider TARPEYO's contraindications and warnings. It is not suitable for patients with hypersensitivity to budesonide and may lead to systemic effects such as hypercorticism and adrenal suppression. The drug also carries risks of immunosuppression and can cause adverse reactions in certain patient populations.

In conclusion, the NeflgArd trial provides valuable insights into the potential of Nefecon in managing IgAN, showing promising results in preserving kidney function and improving patient outcomes across diverse populations. The study's comprehensive approach and detailed findings contribute significantly to the understanding and treatment of IgAN.