Pharming Group of the Netherlands is looking at strategic alternatives, amid concerns about financing and a mistake that has delayed the completion of a Phase III trial in the USA of its hereditary angioedema drug Ruconest/Rhucin.
The Leiden-headquartered biotech says that as "the European capital markets continue to be challenging for life science companies", it has engaged Nomura Code alongside long-term advisor, Roth Capital Partners, to assist in a review of strategic options. These "could include a merger, equity investment or sale", though Pharming stresses that no decision has been made to enter into any specific transaction at this time.
The company noted that the review will be accompanied by additional cost containment measures "and ongoing discussions on platform technology collaborations". It expects to be able to provide more details in the third quarter.
It also appears that Pharming is having a few problems with putting together a data package on Ruconest (recombinant human C1 inhibitor) to satisfy the US Food and Drug Administration. In March 2011, the agency sent Pharming a 'refusal to file' letter indicating that its Biologics License Application was "not sufficiently complete to enable a critical medical review" for the HAE treatment, which is a human protein developed through Pharming's proprietary technology in the milk of transgenic rabbits.
The company said that recruitment for the US pivotal trial for Ruconest is ongoing (68 patients recruited to date) and it is expected that full enrollment (75) could be achieved within the next few weeks, in line with expectations. However, "an internal oversight has recently come to our attention that could potentially delay the unblinding of the top-line data by up to three months in order to complete the statistical package required by the FDA". This matter is currently being investigated.
Completion of the study and read-out of the top-line results will, if positive, trigger a $10 million milestone payment from Pharming's US partner Santarus. The drug is already approved in Europe for treatment of acute angioedema attacks in patients with HAE and is distributed by Swedish Orphan Biovitrum,