On the development side, what are some of the additional indications you are working on for Ruconest?
A pediatric HAE study is ongoing, which we expect to report on early next year; we’ve already announced some very favorable interim results this year. Valeant is our 50-50 partner for developing an HAE prophylaxis indication. We are currently recruiting patients for this study from which we expect to have results by the second quarter of 2016. This study is a three arm, double blind, placebo-controlled, crossover design where we have groups receiving Ruconest and placebos both once and twice per week. Twice a week is the standard for prophylaxis, and the current gold standard product offers a 50 percent reduction in attack frequency; this is the standard we must improve upon, with either weekly or bi-weekly treatments.
We are also exploring a phase II study for treatment of acute pancreatitis, and are interested in a study for delayed graft function, an acute kidney failure problem, where there is also good evidence that a C1 inhibitor may provide benefits. There are other possibilities too.
How are you moving forward with projects not related to Ruconest?
Pharming is working on lead optimization for alpha-glucosidase for treatment of Pompe disease, and alpha-galactosidase for the treatment of Fabry disease. These two projects are being moved forward by our new French research site, which we developed around assets acquired from TRM, a small contract research organisation that had worked extensively with rabbit models. Now we are using this site to generate the new lines for new products, and they are currently working on new vectors and constructs for these two indications, with the goal of developing a generation of founder rabbits for each product. Developing a founder generation can be challenging and sometimes a bit unpredictable, just as with cell-line development, and you really need good experience working with genetic vectors and constructs to be able to get it right in a timely fashion; this is one of the reasons we were very happy and proud to be able to develop this research site in France with the experienced rabbit developers from TRM.
Looking beyond your current initiatives and pipeline, what sort of other projects do you see as being particularly well suited to this rabbit platform?
The next direction we will be able to take will be to prove that the rabbit platform is suitable to compete strongly against CHO cell-lines in other areas, such as antibody production. We are not sure that the difference in concentration compared to cell-line bioreactors will be as great as we see with the C1 inhibitor, which achieves up to 50 times higher concentration, but we do expect to see significantly higher concentrations as well as probable advantages in terms of lower immunogenicity and improved glycosylation in antibodies if we choose the right sort of project.
Now with our French research site we can create new leads with relative ease and frequency, so it is our goal to demonstrate that our platform can successfully produce other therapeutic molecules, and then begin development ourselves. Depending on the project, we would either proceed ourselves or partner with other companies to develop any candidates further. For example, we expect that we will be able to develop orphan products, such as those for Pompe and Fabry diseases, under our own steam, and we have the ambition to commercialize these ourselves eventually as well.