BioWorld International Correspondent

PARIS - ExonHit Therapeutics SA established a wholly owned subsidiary in the United States with its head office in Gaithersburg, Md.

Called ExonHit Therapeutics Inc., it is headed by Jim Lyddy with the job title of vice president business development worldwide. He is supported by Richard Einstein, vice president research U.S., who joined ExonHit from Gene Logic Inc.

Paris-based ExonHit is a functional genomics analysis company that is developing diagnostic and pharmacogenomics tools as well as ready-for-development compounds (RDCs) for cancers and neurodegenerative diseases. On completing a funding round in September in which it raised EUR13.9 million (US$12.3 million), CEO Bruno Tocqui said part of the proceeds would be used to set up a research center in the U.S.

Finding suitable premises took a few months longer than expected, he told BioWorld International, adding that the 10,000-square-foot laboratory ExonHit Therapeutics Inc. is currently setting up should be operational within a few weeks. Its first task will be to launch the company's flagship product, Safe-Hit, onto the American market.

Safe-Hit is a predictive toxicology macroarray based on RNA-splicing signatures that predicts the toxicity of potential therapeutic compounds before they reach the stage of traditional toxicology studies, ranking them by their potential to generate the toxic effects. The U.S. laboratory will include GLP suites for carrying out Safe-Hit analysis and additional diagnostic tests requiring the use of clinical biopsies.

The product has been on the market in Europe since October 1999, and is available both as a product under license or as a service supplied to local firms. Tocqui said the same policy would be applied in the U.S.

The other important mission ExonHit has assigned its U.S. subsidiary is to investigate the role of RNA splicing in the onset and development of cardiovascular diseases. This represents a significant broadening of the company's activities, which are focused at present on developing pharmacogenomics tools and RDCs for cancer and neurodegenerative diseases.

Its main pharmacogenomics product is Proof-Hit, a set of molecular cancer diagnostic tools for early identification of patients who will respond positively to conventional or novel chemotherapy regimens. It is currently undergoing Phase IIb clinical trials in Europe.

In addition, ExonHit has three RDCs in the pipeline. Its lead compound is EHT 201, a drug that is already on the market but which ExonHit is developing for the new indications of amyotrophic lateral sclerosis (ALS) and other CNS diseases. Tocqui said the company would be unveiling the results of preclinical studies in transgenic mice at the end of April. The other two RDCs are EHT 101, a smallmolecular-weight anticancer agent with what ExonHit claims to be a "unique and validated" mechanism, affecting the architecture of cancer cells without any effect on non-cancerous cells; and EHT 102, an anticancer agent targeting one of the major inhibitors of the development of ovarian, colon and breast cancer.

ExonHit's strategy will be to validate the efficacy of RDCs in Phase II trials and then to team up with a pharmaceutical partner for their continued development and commercialization. Tocqui said he is looking for a partner in the U.S. that would endorse ExonHit's core technology by entering into a strategic alliance with the company. It is also looking for a diagnostics company with which to conclude a collaboration agreement for the co-development of Proof-Hit and the subsequent industrial production and commercialization of the product.