Positive Phase 2b Trial of Pharmalink's Nefecon in Primary IgA Nephropathy Presented at Leading Kidney Disease Conference

STOCKHOLM, June 4, 2017 /PRNewswire/ --

Pharmalink AB, a specialty pharma company, is pleased to announce that its recent publication in The Lancet on the positive Phase 2b trial of Nefecon® in primary IgA nephropathy (IgAN) patients, was presented at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) conference (Madrid, Spain).

The Phase 2b trial (known as the NEFIGAN trial), was presented in a special session co-hosted by The Lancet by lead author Bengt Fellström, MD, PhD, Professor of Nephrology at Uppsala University Hospital and Principal Investigator of the NEFIGAN Trial.

In The Lancet paper, published March 28, 2017, clinical data from the NEFIGAN trial are presented that demonstrate the clear potential of Nefecon, a novel, oral, targeted-release formulation of the corticosteroid budesonide, as a new treatment for patients with primary IgAN (a progressive inflammatory kidney disease and orphan indication with a high unmet need). The 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.

Renee Aguiar-Lucander, CEO of Pharmalink, said: "We are proud that the results of our positive Phase 2b trial have been selected for presentation in this prestigious forum. The study provides strong confirmation of the potential of Nefecon to become the first disease-specific treatment for primary IgAN. Our data suggest that Nefecon could delay disease progression, thereby minimizing any further loss of renal function and reducing the requirement for dialysis or kidney transplantation. We look forward to further evaluating the therapeutic potential of Nefecon in this orphan indication in the Phase 3 trial that we are currently planning."

The full reference of the publication in The Lancet is:

Fellstrӧm, BC, et al. Targeted-release budesonide versus placebo in patients with IgA nephropathy (NEFIGAN): a double-blind, randomized, placebo-controlled phase 2b trial (2017) TheLancet, Vol. 389 (No. 10084) p2117-2127http://dx.doi.org/10.1016/S0140-6736(17)30550-0

Prof. Fellström will give a further presentation of results from the NEFIGAN trial at the conference entitled "Proteinuria reduction in IgA nephropathy by Nefecon, a targeted release formulation of budesonide - results from the NEFIGAN trial," in the communication session "Glomerulonephritis" on June 6, 2017 between 8.00-9.30 CET in the Sala Neptuno.

Notes to Editors 

About Pharmalink AB 

Pharmalink is a specialty pharmaceutical company developing high value products for patients with significant unmet medical needs. With a highly experienced, dynamic management team, Pharmalink draws on its extensive experience of pharmaceutical development and marketing to efficiently identify and progress valuable and de-risked products. Visit www.pharmalink.se for further information.

About Nefecon^® 

Nefecon^® is an investigational treatment for patients with primary IgA nephropathy (primary IgAN) at risk of developing ESRD. Nefecon^®  has successfully completed a randomized, placebo-controlled Phase 2b study in 149 primary IgAN patients (full analysis set) at risk of developing ESRD, under standardized rigorous blood pressure control with an angiotensin-converting enzyme inhibitor (ACEI) and/or angiotensin II receptor blocker (ARB). A Phase 3 registration trial is being planned.

Nefecon^®  is an oral, targeted-release and locally acting formulation of the potent corticosteroid, budesonide, that down-regulates the disease process in the kidney through suppression of the gastrointestinal immune system thus exploiting the pivotal role the gastrointestinal tract plays in the overall immune response. Promising results indicate that treatment with Nefecon^® may provide clinical benefits to primary IgAN patients at risk of progressing to ESRD, and provide an alternative to dialysis and transplantation. Nefecon^® has received orphan drug designation in primary IgAN by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

About the NEFIGAN trial 

The NEFIGAN trial was randomized, double-blinded, and placebo-controlled in male and female patients (aged ≥18 years) with primary IgAN and overt proteinuria considered at risk of progressing to end-stage renal disease (ESRD). The trial was conducted at 62 sites across 10 European countries between November 2012 and June 2015. Data from 149 patients constituted the final analysis set, from a total of 249 patients screened.

Following a 6-month run-in phase (to optimize RAS blockade treatment), patients underwent a 9-month treatment phase in which they were randomized in a 1:1:1 ratio to receive Nefecon at 16 mg/day, 8 mg/day or placebo.

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.

About IgA Nephropathy 

IgA nephropathy (IgAN) is the most common form of glomerulonephritis (inflammation of the kidney glomeruli). The disease is characterized by deposits, predominantly containing polymeric IgA antibody, in the kidney that cause inflammation and renal damage.

IgAN can occur at any age, but the clinical onset is commonly during the second or third decades of life. It has been estimated that up to 40% of patients with primary IgAN progress to renal failure, often referred to as ESRD within 5-30 years following diagnosis. This patient population is estimated to at least 200,000 in major markets.

Patients suffering renal failure require dialysis or kidney transplantation. Primary IgAN accounts for 10% of renal transplants among patients with primary glomerulonephritis in the US and between 7-20% of patients in Europe and Australia in long-term dialysis and renal transplantation programs.

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