Calliditas announces primary endpoint successfully met in Phase 3 NefIgArd trial evaluating Nefecon® in IgA nephropathy

STOCKHOLM, March 13, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (NASDAQ: CALT) (NASDAQ Stockholm: CALTX) ("Calliditas") today announced positive topline results from the global, randomized, double-blind, placebo-controlled Phase 3 clinical trial NefIgArd, which investigated the effect of Nefecon (TARPEYO®/Kinpeygo® (budesonide) delayed release capsules) versus placebo in patients with primary IgA nephropathy (IgAN).

• The trial met its primary endpoint with Nefecon demonstrating a highly statistically significant benefit over placebo (p value < 0.0001) in estimated glomerular filtration rate (eGFR) over the two-year period of 9-months of treatment with Nefecon or placebo and 15-months of follow-up off drug.
• Supportive 2-year total slope analyses were statistically significant and clinically meaningful reflecting a sustained treatment benefit.
• The eGFR benefit was observed across the entire study population, irrespective of urine protein-to-creatinine ratio (UPCR) baseline, which the company believes supports a regulatory filing for full approval in the study population.
• UPCR reductions observed were durable, reflecting a long lasting treatment effect during the 15-month follow-up period off treatment.

"This is truly a great outcome for IgAN patients. This reflects sustained impact on kidney function across the entire study population with a treatment which was specifically designed to treat IgAN by downregulating pathogenic IgA1 antibodies at their presumed source and we believe this dataset supports regulatory filing for full approval based on the Phase 3 study population," said CEO Renée Aguiar-Lucander.

"These data show the kidney function protection delivered by Nefecon and demonstrate that the approach offers patients a truly disease modifying treatment with sustained reductions in proteinuria over two years and continued eGFR benefit. Importantly Nefecon was well tolerated and together with the proteinuria and eGFR data mean that Nefecon has cemented its place as a key treatment option for patients with IgA nephropathy at risk of progressive kidney function loss," said Dr Jonathan Barratt, Mayer Professor of Renal Medicine at Leicester University.

"These data establish that there is an option for patients with IgA nephropathy to specifically target their illness and to safely slow and delay progression of their kidney disease. The sustained effects on proteinuria and on eGFR are impressive and clinically meaningful," said Richard Lafayette, Professor of Medicine (Nephrology) at Stanford University.

This data readout from Part B provides longer term data from the Phase 3 NefIgArd trial, which read out topline data on Part A in November 2020. An additional 29 Chinese patients, required for local Chinese regulatory purposes only, are expected to complete Part B in Q3, 2023. Based on the Part A data, Calliditas received accelerated approval from the U.S Food and Drug Administration (FDA) in December 2021 and conditional marketing authorization from the European Commission (EC) in July 2022, marking the first time a drug was approved for the treatment of IgAN in the US and the European Economic Area (EEA). Nefecon is being marketed by Calliditas in the US under the brand name TARPEYO®, and by STADA Arzneimittel AG in the EEA, Switzerland and the UK under the brand name Kinpeygo®.

"I am delighted with the positive outcome of the NefIgArd trial. This important milestone is the culmination of many years of hard work and dedication from so many people involved in the study. I would like to extend my thanks in particular to the investigators and site staff involved in the study, as well as of course the participating patients," said Calliditas' CMO, Dr. Richard Philipson.

On the basis of this data, Calliditas plans to file for full approval from the FDA, and support filing for full approval with EC and UK MHRA during 2023 for patients with primary IgAN based on the Phase 3 study population.

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