BioWorld International Correspondent

PARIS - ExonHit Therapeutics SA delivered a small-molecule target to Allergan Inc. under the three-year drug discovery, development and commercialization agreement the companies signed in December 2002, and the U.S. company has initiated high-throughput screening.

ExonHit said the target showed efficacy in a previously unrecognized pathway it identified using its gene-profiling technology DATAS (Differential Analysis of Transcripts with Alternative Splicing), but declined to disclose the therapeutic field concerned. Its collaboration with Allergan, of Irvine, Calif., covers three areas: neurodegenerative diseases, pain and ophthalmology.

The collaboration calls for ExonHit to identify new molecular targets from various libraries, as well as tissue samples and model systems, and for the companies to jointly develop compounds and commercial products based on the targets. ExonHit will retain intellectual property rights to all compounds not developed, while the companies will co-own products taken into development. The French company also will retain co-development and co-commercialization options in therapeutic areas outside Allergan's specialties.

ExonHit is receiving research and development funding from Allergan, which also will pay milestones when drug candidates reach certain development stages and royalties on commercialized drugs. The delivery of the target did not trigger a milestone payment, ExonHit's business development manager, Aram Mangasarian, told BioWorld International.

The deal also gives Paris-based ExonHit access to Allergan's chemistry for the development of treatments for amyotrophic lateral sclerosis (ALS), one of the main pathologies targeted by the French company's in-house drug discovery programs. Its strategy is to discover and validate "ready-for-development compounds" for licensing out to third parties. Its lead compound, EHT 0201, a formulation of Rilutek (riluzole), of Strasbourg-based Aventis SA, is undergoing a Phase II trial in ALS. That trial, which involves 400 patients, is due to be completed by the fall of 2004.

ExonHit is about to take a second product into clinical development. A Phase I trial of EHT 0202, a small-molecule drug with potential in several neurodegenerative diseases as well as retinitis pigmentosa, is due to get under way in France in early December. Mangasarian said the trial would not be focused on a particular indication and that ExonHit has not decided in what indication a Phase II trial would take place.

The company has two other chemical entities in preclinical development for cancer and ocular revascularization disorders. In addition, it is developing a prostate cancer diagnostic test, based on alternatively spliced isoforms, as well as a blood-based diagnostic test for colon cancer, both of which it plans to outlicense in the near future.

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