Pharming Group N.V. has presented its (unaudited) financial report for the full year ended 31 December 2016. Sijmen de Vries, CEO and Chairman of the Board of Management, comments:
2016 was a major year for Pharming. During the year we achieved a number of positive milestones that culminated in December in the game-changing re-acquisition of commercialization rights for Ruconest in North America from subsidiaries of Valeant Pharmaceuticals International, Inc.
Early in the year, we expanded our collaboration with Cytobioteck S.A.S. for the exclusive distribution of Ruconest in Latin America by the addition of four countries. Subsequently, we amended our agreement with Swedish Orphan Biovitrum AB, resulting in the return of the commercialization rights for Ruconest in certain Western European, North African and Middle Eastern markets. This accelerated our goal towards becoming a fully integrated specialty pharma company.
In May, the European Medicines Agency (EMA) confirmed that pre-exposure testing was no longer necessary for Ruconest. Later in the year a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) was obtained recommending permission for home treatment with Ruconest, with a custom-designed self-administration kit, which was confirmed by the EMA with the appropriate label adjustment early in 2017. This EU approval of self-administration is further to the US approval received in 2014.
In July, positive clinical and statistically significant results were achieved in our randomized double- blind Phase II clinical trial for Ruconest in prophylaxis of HAE, meeting all primary endpoints. The study showed that Ruconest, used once-weekly, results in a very similar reduction of HAE attack frequency to that obtained with twice-weekly dosing of the only currently approved product for the prophylaxis of HAE (i.e. approximately 50% reduction in attack frequency in approximately 50% of patients). Ruconest dosed twice-weekly achieved an unprecedented response rate (reduction of attack frequency of at least 50%) of 97% and average reduction of attack frequency of 73%. These results demonstrate, yet again, that the appropriate dosing of our C1 inhibitor leads to results that patients can rely on.
In order to continue to improve the convenience of Ruconest administration, our R&D scientists have formulated a highly-concentrated vial of Ruconest, so that we are now looking to enter clinical trials with intra-muscular and/or sub-cutaneous administration of smaller injections of Ruconest within the next twelve months.
Following a preliminary announcement of the conditional deal in August, in December we announced the definitive acquisition of the North American commercialization rights for Ruconest from Valeant. The transition of the sales force that we acquired as part of the deal was smoothly executed, with the team selling Ruconest one day for Valeant and selling Ruconest the next day for Pharming. Immediately after the close of the deal, we initiated our plans to increase awareness and sales of Ruconest in the US market. We have now hired additional experienced HAE/rare disease sales force members, medical science liaison professionals and a very seasoned management team with expertise in marketing, sales, commercial activity and patient support.
As a result of these EU and US transitions, we now operate with an optimal commercial presence in both Western Europe and the US and can focus fully on delivering on our commitment to become an operationally profitable company during 2017.