The News

Exonhit Therapeutics awaits final efficacy results as amyotrophic lateral sclerosis trial moves forward

26/01/2004

Data Safety Monitoring Board Recommends Continuation of the large European Clinical Trial For Ikomio

Paris, France… ExonHit Therapeutics, a privately-held drug discovery company, announced it has received recommendation by an independent Data Safety Monitoring Board (DSMB) to continue its clinical trial for Ikomio, a potential treatment for Amyotrophic Lateral Sclerosis (ALS), a neurodegenerative disease commonly known as Charcot’s disease or Lou Gehrig’s disease.  

The DSMB concluded that interim data raised no safety concerns and the trial is cleared to proceed to its planned conclusion in 2004.  Survival is the primary endpoint in this 400 patients double-blind, randomized, placebo-controlled study. Ikomio (or placebo) is administered in addition to Rilutek® (riluzole), currently the sole approved drug for ALS. Ikomio appears well tolerated in patients suffering from ALS and does not alter the safety profile of Rilutek®.
 “As clinical investigators, our primary goal is to avoid any aggravation of the clinical status of these fragile patients. One of the concerns, which is today behind us, was to know how this drug would be tolerated in these patients as compared to the general population” commented Vincent Meininger, Professor of Neurology, Hôpital Pitié-Salpétrière, Chairman of the Study Steering Committee.
“We are pleased by Ikomio’s safety profile” commented Philippe Guillet, M.D., VP Clinical Research, ExonHit Therapeutics.  Dr. Guillet, a veteran of trials in ALS, further noted: “This trial is being run very efficiently. It is one of the largest trials conducted in ALS and the 400 patients were enrolled in just four months. In parallel to the conduct of the study, we are developing a formulation suitable for patients with swallowing difficulties. We are working diligently to provide new therapies for ALS, which is a devastating, fatal disease. ”  
Ikomio was chosen from a library of marketed compounds after scientists at ExonHit discovered a tissue-specific RNA isoform produced as a result of alternative splicing that occurs during the early phases of the disease in animal models of ALS.  Ikomio, the company’s first compound in clinical trials, helps to prevent neuronal death and acts on the regulation of respiratory compensatory mechanisms involved in the progression of ALS.  
The process of finding and understanding alternative RNA splicing enables ExonHit  to find new potential targets and to gain insight into the molecular causes of disease. The company has further uncovered a large number of potential targets which are being pursued.     
“ExonHit Therapeutics intends to share the first safety results with the Regulatory Authorities and seek their advice for bringing this drug to the patient community in a timely manner. Our commitment to developing new products for the treatment of ALS stands firm” declared Bruno Tocqué, CEO of ExonHit Therapeutics.

About Amyotrophic Lateral Sclerosis (Motor Neuron Disease, Charcot’s or Lou Gehrig’s disease)
Amyotrophic Lateral Sclerosis (ALS) is a progressive and fatal neurological disease characterized by a degeneration of motor neurons in the spinal cord, brain stem and cerebral cortex, leading to muscle wasting and weakness. It affects 100,000 to 120,000 people worldwide with 5,000 and 7,500 new cases occurring each year in the US and in Europe, respectively. There is no cure for ALS, nor is there a proven therapy that can prevent or reverse the course of the disorder. ALS affects all muscles under voluntary control resulting in sufferers losing the strength and the ability to move their arms, legs, and body. When respiratory muscles are affected, patients lose the ability to breathe without ventilatory support. 80% of ALS patients die from respiratory failure.

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.  

ExonHit’s proprietary gene profiling technology, DATASTM(Differential Analysis of Transcripts with Alternative Splicing), systematically identifies RNA splicing alterations on a genome-wide scan.  RNA splicing is altered during the onset and progression of disease, therefore, by distinguishing discrete splicing events the company is able to identify landmarks of disease.

DATASTM is central to internal drug discovery programs.  The company has two drugs currently in clinical trials. In addition, the profiling technology is utilized in broader applications by several external therapeutic and diagnostic collaborators, and 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 68 employees worldwide and has raised a total of €47 million.

Contacts: 
ExonHit Therapeutics :
Bruno Tocqué, CEOPhilippe Guillet, MD, Vice-President Clinical Research
Sheila Khatri, External Communications (USA) France:+33 (0)1 58 05 47 00USA: +1 240 683 7050
Media Relations Sylvie Roquefeuil-Dedieu, De Facto Communications Tel  +44 (0)207 496 33 00