STOCKHOLM, January 13, 2014 /PRNewswire/ —
Pharmalink AB, a specialty pharma company focused on orphan and niche products, announces it has completed recruitment of patients into a Phase IIb NEFIGAN study of its orphan drug product, Nefecon® (http://www.nefigan.net). Pharmalink expects to announce top-line results in mid-2015.
The Phase IIb study is a multi-center, randomized, double-blinded placebo-controlled clinical trial designed to assess the efficacy and safety of two different doses of Nefecon in 90 IgA nephropathy patients at risk of end-stage renal failure, despite optimized standard-of-care therapy. The study is being conducted in 62 renal centers in ten European countries.
The primary efficacy objective of the study is to investigate whether patients on Nefecon achieve a larger mean reduction in proteinuria compared to patients on placebo. Randomized patients are treated for nine months following a six-month run-in phase in which standard-of-care therapy is optimized. The study will enable an optimal dose of Nefecon to be selected for a Phase III registration trial.
Alex Mercer, Clinical Development Director of Pharmalink, said: “The response to this study from renal disease specialists across Europe has been outstanding and to complete recruitment within one year in this rare disease is a sign of the great interest the study has generated. This enthusiasm bodes well for the future as we advance Nefecon towards marketing approval. If the highly promising results we have seen in earlier studies are confirmed in NEFIGAN, we trust that Nefecon can become an effective new treatment for IgA nephropathy patients at risk of renal failure.”
Bengt Fellström, MD, PhD, Professor of Medicine at Uppsala University Hospital, Sweden and Principal Investigator of the NEFIGAN study, added: “IgA nephropathy is a disease with a high unmet medical need. Progression to renal failure has a terrible impact on patients’ quality of life and is a high burden to health care systems. A new medicine for early treatment with the potential to stop disease progression and minimize any further loss of renal function would be very welcome news to patients and clinicians alike. I am delighted at the response we have had from the renal disease community to this study, which has led to its rapid recruitment, and now look forward to the results in due course.”
Nefecon is a potential disease-modifying treatment for patients with primary IgA nephropathy at risk of developing end-stage renal disease, an indication with a high unmet medical need and no approved treatment. Early treatment with Nefecon provides a therapy to slow or halt disease progression and further loss of renal function, preventing the need for dialysis and transplantation.
Nefecon is an enteric formulation of a locally-acting and potent corticosteroid 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. Nefecon has received orphan drug designation from the US Food and Drug Administration (FDA).
Pharmalink anticipates seeking a commercial partner for Nefecon in all territories.
About IgA Nephropathy
IgA nephropathy 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.
IgA nephropathy can 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% of patients with IgA nephropathy slowly progress to renal failure, often referred to as end-stage renal disease within 5-30 years following diagnosis. This patient population is estimated at approximately 200,000 in major markets.
Patients suffering renal failure require dialysis or kidney transplantation. IgA nephropathy 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.
Pharmalink is a Swedish specialty pharma company developing high-value products for niche indications. Pharmalink draws on its extensive experience of pharmaceutical development and marketing to identify and progress products that address significant unmet medical needs. With a successful history in pharmaceutical sales and marketing, and highly experienced, dynamic management team, Pharmalink is focused on the development and commercialization of valuable, de-risked projects. It has two late-stage clinical phase products under development, Nefecon® and Busulipo™. Pharmalink is actively seeking opportunities to acquire or in-license product opportunities in niche and hospital care indications. Visit http://www.pharmalink.se for further information.