On the presentation of the Pharming Group N.V. financial report for the six months ended 30 June 2018 CEO Sijmen de Vries said:
“We are delighted with the further progress we have made expanding the reach of Ruconest, allowing more patients to access the clinical benefits of our product. We have continued net profitability in the second quarter of the year, which gives us the confidence and the financial resources to move forward with our new programs. With five studies underway or expected to initiate over the next six months, we anticipate significant strengthening of our pipeline.”
From the CEO’s comments:
During the first half of the year, we continued to invest in the development of our commercial infrastructure in North America and Europe to drive the growth of new patients using our lead product Ruconest for the treatment of HAE, as well as to manage the increased demand for the product.
The positive sales momentum in the USA continued in Q2, following higher than expected sales in Q1 as a result of the shortage of a competitor product. As the clearest measure of the success of Ruconest, the number of patients using the product regularly in the USA has been increasing steadily since we reacquired the commercial rights.
In January, we announced that the U.S. Food and Drug Administration (FDA) had accepted for review Pharming’s supplemental Biologics License Application (sBLA) for Ruconest for routine prophylaxis to prevent attacks in adult and adolescent patients with HAE. The FDA indicated that the sBLA was sufficiently complete to permit a substantive review and has set an action date of 21 September 2018.
The highlights of these announcements were:
- An ongoing investigator-sponsored study of Ruconest in Basel, Switzerland a double-blind, placebo-controlled trial of contrast-induced nephropathy which was initiated last year and is expected to report top-line data in Q3 2018.
- An ongoing investigator-sponsored head-to-head study of Ruconest in an open label clinical trial testing therapy failure rates (i.e. the need for re-dosing under either therapy) to treat an attack of HAE, which is expected to be fully recruited in Q3, with top-line data expected in Q4 2018.
- Pharming has developed new versions of small vial liquid and fast-dissolving dosage forms for Ruconest, for use in subcutaneous and intramuscular versions and in an entirely new, intradermal version expected to be painless, starting with subcutaneous studies later in 2018/ early 2019.
Based on the continued momentum in sales volumes, underpinned by improving trends in identifying and diagnosing HAE patients, combined with better patient care and management practices, a focus on specialty pharmacy customers and subject to an FDA approval for prophylaxis of HAE, we expect to continue to increase sales of Ruconest further. If that approval is granted, we expect the efficacy of Ruconest to be appealing to healthcare professionals and the patients they manage for complete management of their HAE condition. It will also be the only approved product for both prophylaxis and treatment of breakthrough HAE attacks.