Pharmalink AB, a specialty pharma company focussed on orphan and niche products, announces that clinical results from the initial run-in phase of its phase IIb clinical study with Nefecon will be presented in a poster during Kidney Week 2014, the annual meeting of the American Society of Nephrology (ASN).

The abstract of the presentation is published on the ASN website. ASN Kidney Week, the world's largest nephrology meeting, will take place 11-16 November in Philadelphia, PA.

Nefecon is a new oral modified-release capsule formulation of the  corticosteroid,budesonide, in development by Pharmalink for the treatment of patients with IgA nephropathy (IgAN) at risk of developing end-stage renal disease (ESRD)despite optimised RAS blockade.

The safety and efficacy of Nefecon is currently under evaluation in a prospective,randomised, placebo-controlled study (NEFIGAN Trial). Patients entering the study undertake a six-month Run-in Phase during which standard-of-care therapy[angiotensin-converting enzyme inhibitor (ACEI) and/or angiotensin II receptor I blocker (ARB)]is optimised according to KDIGO guidelines. Run-in phase data from patientscompleting the Run-in Phase will be presented.

Alex Mercer, clinical development director of Pharmalink, said, “The data from the Run-in Phase of the NEFIGAN Trial show that IgAN patients remain at risk of progression to ESRD despite optimised standard-of-care therapy, and highlight the need for new treatment options.

This need is recognised in the renal disease community and we have been delighted with the positive and enthusiastic engagement from key opinion leaders in the area around the NEFIGAN Trial. This support has enabled Pharmalink to complete enrolment into the study and remain on track to report headline results in Q3 2015 as planned.”

The NEFIGAN Trial: A randomised, Placebo-Controlled Study to evaluate the efficacy and safety of Nefecon in IgA Nephropathy Patients at risk of developing ESRD: Preliminary data from the Run-in phase.

Nefecon is a potential disease-modifying treatment for patients with primary IgA nephropathy (IgAN) at risk of developing end-stage renal disease (ESRD).Nefecon has shown positive results in an open-labelled phase II trial evaluating safety and efficacy and is now being tested in a placebo-
controlled randomised phase IIb study..

The study has been designed to enable an optimal dose of Nefecon to be selected for a phase III registration trial. Headline data is anticipated in Q3 2015.

Nefecon is an oral formulation of a locally-acting and 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. Early treatment with Nefecon of IgAN patients at risk of developing ESRD may slow or halt disease progression and further loss of renal function, and provide an alternative to dialysis and transplantation. Nefecon has received orphan drug designation by the US Food and Drug Administration (US FDA).

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

IgANcan occur at any age, but the clinical onset is commonly during the second or third decades of life. It has been estimated that 20-40 per cent of patients with IgAN progress to renal failure, often referred to as end -stage renal disease 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 kidneytransplantation.  IgAN accounts for 10 per cent of renal transplants among patients with primary glomerulonephritis in the US and between 7-20 per cent of patients in Europe and Australia in long-term dialysis and renal transplantation programmes.

The Nefigan trial is a multicentre, randomised, double-blind, placebo-controlled study conducted at more than 60 centers in ten European countries. The objective of the study is to evaluate efficacy and safety of two different doses of Nefecon in the treatment of IgAN patients at risk of developing ESRD, under standardised rigorous blood pressure control with an angiotensin-converting enzyme inhibitor (ACEI) and/or angiotensin II receptor I blocker(ARB).