Calliditas Therapeutics has enrolled the first patient in a pivotal Phase III NEFIGARD clinical trial to evaluate nefecon for the treatment of patients with IgA nephropathy (IgAN).
The double-blind, placebo-controlled trial intends to investigate the efficacy, safety, and tolerability of oral nefecon in comparison with placebo.
Calliditas aims to enrol a total of 450 patients across around 150 clinical sites in 19 countries.
Patients will be randomised to receive once-daily oral treatment of either nefecon or placebo for nine months.
The trial’s primary endpoints are a change in proteinuria, measured as urine protein to creatinine ratio (UPCR), and events based on renal function as measured by estimated glomerular filtration rate (eGFR).
Secondary endpoints include events based on renal function measured as estimated glomerular filtration rate (eGFR) and renal function.
Top-line data from the NEFIGARD trial is expected to be available in 2020.
Calliditas Therapeutics chief medical officer Jens Kristensen said: “We have previously shown good efficacy and safety of nefecon in the comprehensive NEFIGAN study, which as of today is the only successful, placebo-controlled Phase IIb study in this indication.
“We expect to see a similar study result of reducing proteinuria and stabilising the kidney function of patients coupled with the benign safety profile we have seen so far.”
Calliditas plans to file for market approval of nefecon with regulatory agencies depending on positive results from the first 200 dosed patients in the NEFIGARD trial.
Nefecon is an oral formulation of budesonide developed by Calliditas as a potential treatment for patients with IgAN who are at risk of developing end-stage renal disease (ESRD).