BioWorld International Correspondent

InDex Pharmaceuticals AB signed its first product deal, out-licensing its antisense drug Kappaproct to Serono SA, of Geneva, for treatment of ulcerative colitis and other inflammatory conditions.

Stockholm, Sweden-based InDex stands to gain $35 million in up-front and milestone payments, plus royalties, should the compound gain regulatory approval in one indication.

Kappaproct inhibits production of the p65 subunit of the transcription factor nuclear factor kappa B (NF-kappa B), which activates the expression of multiple pro-inflammatory genes. It is undergoing a Phase II trial, which will involve 150 ulcerative colitis patients.

"We will be managing the Phase II trial," CEO Svante Rasmuson told BioWorld International. "Once we have the results from that, and once, hopefully, [Serono is] happy with the results, they will continue the development." He added that InDex expects to report on the study during the first half of next year.

"If this goes according to plan, this product could be launched on the market by the end of 2007," he said.

In an earlier study involving patients refractory to other treatments, the drug appeared to elicit a clinical response following a single dose. A number of participants who were about to undergo surgery have remained disease-free and have been weaned off steroid therapy, Rasmuson said. Why a single dose appears effective is not fully understood, but InDex Chief Scientific Officer Oliver von Stein said it might involve re-setting some sort of balance that has been upset by the inflammation process.

"It's an observation we have seen at numerous levels. To hazard a guess as to why, I'm not sure I could," he told BioWorld International.

Kappaproct is administered via an enema and exhibits low systemic uptake, a fact that has drawn a favorable response in discussions with regulatory authorities in Sweden, Denmark, Norway and two other countries, Rasmuson said. Early preclinical and clinical work on the compound was performed at the Karolinksa Institute and the HMQ Sophia Hospital in Stockholm.

InDex, which was founded in 2000, has so far raised SEK67 million (US$9.2 million) in funding. It is focusing on cancer as well as immunology, although its efforts in the latter field are more advanced. A second project, involving a diagnostic based on an array of genetic markers that distinguishes between ulcerative colitis and Crohn's disease, is at the early stages of the out-licensing process, Rasmuson said. Other drug development projects are further back in the pipeline.

"We focus not only on antisense but on sequence-specific RNA inhibitor compounds," he said.