Pharming Explores Further Uses For Ruconest
Executive Summary
Pre-eclampsia and nephropathy associated with the use of contrast media in cardiac patients are two new potential uses for Pharming’s marketed C1-esterase inhibitor product.
Leiden, the Netherlands-based Pharming Group NV expects to receive approval shortly to begin a clinical study of Ruconest (conestat alfa) in pre-eclampsia and plans to start an additional trial of the therapy to treat acute kidney injury in patients undergoing percutaneous coronary interventions accompanied by contrast-enhanced examinations in the second half of this year.
Such evaluation of potential new indications for its only marketed product, intravenous Ruconest for acute angio-edema attacks, is backed by Pharming’s continuing positive financial performance; the company reported a 19.5% rise in revenues in the first quarter of 2019, reaching €35.2m, and a 23% increase in net profits to €6.7m, both compared with the same quarter in 2018.
The favorable results continue the positive financials reported for 2018, Pharming’s first year of net earnings, when the company was profitable at the net level in every quarter and for the year as a whole making a total net profit of €25m, leading the company to exploit its technology further (see sidebar).
In the first quarter of 2019, most of Pharming’s net product sales, totaling €33.7m, came from the US with European and international sales only reaching €1.3m in the three months.
For the remainder of 2019, Pharming expects to report positive net earnings in future quarters, and to pursue further clinical trial programs for Ruconest for the acute and prophylactic treatment of hereditary angio-edema, including the development of new intramuscular, subcutaneous and intradermal versions of Ruconest, which are expected to be relatively painless to administer in comparison to competing HAI therapies, and representing a potential advantage in the marketplace.
Further back in development is the use of Ruconest for tissue damage due to hypoxic events, including delayed graft function, and hypovolemic shock after trauma. The company is also investigating at an early stage the use of transgenic rabbit-derived alpha-glucosidase for the treatment of Pompe disease and rabbit-derived alpha-galactosidase for Fabry disease.