BioWorld International Correspondent

Shares in Pharming Group NV rose more than 27 percent Monday on news that it stopped a placebo-controlled clinical trial of Rhucin in hereditary angioedema (HAE) on ethical grounds, following advice from an independent data monitoring committee. An interim analysis of the data indicated that none of the patients treated during the study experienced a relapse, and no adverse events were reported.

The stock closed at €2.83 (US$3.82), up on its previous close of €2.22. It shed some of those gains Tuesday, however. By midafternoon, the stock was changing hands at €2.71 on the Euronext exchange in Amsterdam.

Rhucin is a recombinant version of the human C1 inhibitor, which is produced in the milk of transgenic rabbits. It is designed to correct the inherited C1 inhibitor deficiency associated with HAE, which results in painful and sometimes life-threatening swelling of the hands, feet, limbs, face, gut or airways.

Pharming, based in Leiden, the Netherlands, already filed a marketing authorization application for the protein with the European Medicines Agency, but said the new data - details of which will be presented to analysts and media in London Aug. 30 - has been added to the dossier. The latter originally was filed more than a year ago, and a decision from the EMEA is expected before year-end. (See BioWorld International, Aug. 2, 2006.)

"Maybe it might help them to make a decision," Christophe Van Vaeck, analyst at KBC Securities NV, in Brussels, told BioWorld International. "I don't expect it will change much about the timing, which has been a disappointment," he said. When he first started following the stock, in January 2005, approval for Rhucin was anticipated before the end of 2005, he said. "The lead time Pharming had two years ago over the competition has evaporated."

Nevertheless, Van Vaeck still sees upside potential in the stock, and he retains an "accumulate" recommendation, although he has reduced his target price from €3.80 to €3.50, to take account of the EMEA's acceptance for review Aug. 15 of a rival product, the peptidomimetic bradykinin B2 receptor antagonist Icatibant, which Berlin-based Jerini AG is developing.

He remains cautious on the stock, however, and any further delays in gaining approval for Rhucin could lead to problems. "The cash position is something that has to be kept an eye on because it's going down rapidly," he said.

The company reported a net loss of €11.3 million for the first half of 2007, and it had cash and equivalents totaling €19.1 million on its balance sheet as of June 30. However, it was due to make payments of $2 million and $10 million in July 2007 and July 2008, respectively, to the New York-based Paul Royalty Fund, as part of a royalty agreement it entered early last year. Pharming is due to collect $10 million in milestones once Rhucin gains approval in the U.S., although the company has not yet said when it will file for FDA approval.