Stockholm, Sweden – March 29, 2017. Pharmalink AB, a specialty pharma company, announces that positive results and analyses from the completed Phase 2b trial of its oral drug candidate Nefecon® in primary IgA nephropathy (IgAN) patients have been published online in The Lancet (Fellström et al, reference below). This is the first time the full results from this trial have been published in this format.
In the paper, clinical data are presented that demonstrate the clear potential of Nefecon, a novel, targeted-release formulation of the corticosteroid budesonide, as a new treatment for patients with primary IgAN (a progressive inflammatory kidney disease). More specifically, the trial (known as the NEFIGAN Trial) met its primary endpoint and concluded that the use of Nefecon, in addition to standardized rigorous blood pressure control (optimized RAS blockade), reduced proteinuria and stabilized estimated glomerular filtration rate (eGFR) in patients at risk of developing end-stage renal disease (ESRD). Both of these effects are indicative of a reduced risk of future progression to ESRD.
Pharmalink is preparing to start pivotal Phase 3 studies of Nefecon in this indication.
Johan Häggblad, CEO of Pharmalink, said: “We are delighted with the results from the NEFIGAN Trial that have been published in the prestigious Lancet journal. These results have encouraged us in our considerations and preparations for a pivotal Phase 3 registration trial of Nefecon in primary IgAN patients. We thank all the investigators and patients who contributed to this clinical trial and look forward to advancing our clinical development plans with this exciting drug candidate.”
“These clinical trial results strongly support the concept of targeting the gut immune system in primary IgAN patients with persistent proteinuria. Furthermore, they confirm the potential of Nefecon to become the first disease-specific treatment for primary IgAN, with a risk-benefit profile supportive of its use early in the course of disease,” commented Bengt Fellström, MD, PhD, Professor of Nephrology at Uppsala University Hospital and Principal Investigator of the NEFIGAN Trial. “IgA nephropathy is the most common inflammatory kidney disease and in real need of new treatment options that prevent or delay patients progressing to renal failure, which has a devastating impact of patients’ quality of life. A new medicine with the potential to stop or delay disease progression, and minimize any further loss of renal function, thereby reducing the requirement for dialysis or kidney transplantation, would be very welcome news to patients and clinicians.”
The primary outcome of the Phase 2b clinical trial was assessed on the full analysis set (n=149), defined as all randomized patients who took at least one dose of trial medication and had at least one post-dose efficacy measurement (modified intention-to-treat analysis). At nine months, mean urine protein creatinine ratio (UPCR) decreased by -26.4% with Nefecon [p=0.0066] (-29.3% with 16 mg/day [p=0.009], non-significant -23.7% with 8 mg/day [p=0.029]), vs. placebo. The effect was sustained throughout follow-up; mean UPCR decreased by -32% from baseline at 12 months for 16 mg/day vs. a 0.5% increase for placebo. Over nine months, eGFR was stable with Nefecon but decreased 9.8% with placebo (Nefecon vs. placebo: p=0.001). Nefecon was well tolerated and the total incidence of treatment-emergent adverse events was similar across all treatment groups.
ReferenceFellström, BC, et al. Targeted-release budesonide versus placebo in patients with IgA nephropathy (NEFIGAN): a double-blind, randomized, placebo-controlled phase 2b trial (2017) The Lancet, http://dx.doi.org/10.1016/S0140-6736(17)30550-0