The News

Allergan and Exonhit designate target for high throughput screening

19/11/2003

ExonHit Therapeutics identifies novel target
Paris, France… ExonHit Therapeutics announced that Allergan, Inc. (AGN), a global specialty pharmaceutical company, has initiated high throughput screening on a target generated pursuant to a 10 month old strategic alliance between the companies.  ExonHit is a privately held company that discovers neurodegenerative and cancer therapeutics and diagnostics based on alternative RNA splicing.

ExonHit has delivered to Allergan a small molecule target that shows efficacy in a previously unrecognized pathway identified utilizing DATASTM, ExonHit’s proprietary gene profiling technology.  This target will begin high throughput screening.

The strategic alliance was formed in December 2002 with the purpose of developing new drugs in the field of neurodegenerative diseases, pain and ophthalmology.  The dual goals of the agreement are to identify new molecular targets based on ExonHit Therapeutics’ gene profiling DATAS™ technology and to work collaboratively toward developing and commercializing unique compounds and commercial products based on these targets.     Les Kaplan Executive Vice President and President of R&D at Allergan stated, “I am pleased both with the productive working relationship and with the progress made to date”.

“Rapidly delivering a designated target and expecting to deliver one or two more within the next five months exemplifies the power of DATAS™ and of our ability to analyze the role of alternative RNA splicing events in disease,” commented Fabien Schweighoffer, Executive VP, Research, ExonHit Therapeutics, “I view the ExonHit approach as an engine for drug discovery and I’m pleased this approach is moving the alliance with Allergan in a promising direction and with impressive speed-- it’s a process that has worked well for our diagnostic collaborations and internal therapeutic programs.”

ExonHit’s therapeutic pipeline includes a compound for Amyotrophic Lateral Sclerosis (commonly known as Charcot or Lou Gehrig's disease) which is currently in a large Phase II clinical trial (400 patients) for evaluation of its safety and its efficacy on survival. This study is expected to be completed in the Fall 2004.  A Phase I trial is planned for a small molecule therapeutic with indications in several neurodegenerative diseases and retinitis pigmentosa.  Furthermore, two new chemical entities with anti-angiogenesis properties are in pre-clinical stages for the treatment of cancer and ocular revascularization disorders (the leading cause of blindness). 

In addition to its therapeutic programs, ExonHit has ongoing diagnostic collaborations with Roche Applied Sciences and bioMerieux.  Internal diagnostic projects include a prostate cancer diagnostic test, based on alternatively spliced isoforms of PSA and KLK2, and a blood-based diagnostic test for colon cancer.   ExonHit intends to out-license both tests in the near future.  

Forward-Looking Statements

This press release contains "forward-looking statements", such as the statements of Dr. Kaplan, and Dr. Schweighoffer, and the parties’ goals of developing drug compounds and commercial products.  These statements are based on current expectations of future events.  If underlying assumptions prove inaccurate or unknown risks or uncertainties materialize, actual results could vary materially from the parties’ expectations and projections. Risks and uncertainties include general industry and pharmaceutical market conditions; general domestic and international economic conditions; technological advances and patents attained by competitors; challenges inherent in new product marketing such as the unpredictability of market acceptance for a new pharmaceutical product or approved indication; potential difficulties in manufacturing a new product formulation; domestic and foreign health care reforms; and trends toward managed care and health care cost containment; and governmental laws and regulations affecting domestic and foreign operations.

About ExonHit Therapeutics

ExonHit Therapeutics is a privately-held company whose mission is to develop novel therapeutics and diagnostics for neurodegenerative diseases and cancer.  The company is a leader in alternative RNA splicing holding a premier scientific and intellectual property position in the field.  

Utilizing DATASTM, the company’s proprietary gene profiling technology, allows the detection of disease-relevant changes in mRNA populations resulting from alternative RNA splicing events, providing key insight into biological causes of pathology. DATASTM stands for: Differential Analysis of Transcripts with Alternative Splicing. 

DATASTM is central to internal drug discovery programs but the profiling technology also has broad applications.  ExonHit has several external therapeutic and diagnostic collaborators, and has used the technology to capitalize on the growing needs for pharmacogenomic tools, and toxicity prediction assays. 

ExonHit Therapeutics, founded in 1997, is headquartered in Paris, France and has a US facility in Gaithersburg, Maryland. The company has 70 employees worldwide and has raised a total of €47 million.

About Allergan, Inc.

Allergan, Inc., with headquarters in Irvine, California, is a technology-driven, global health care company providing eye ca