BioWorld International Correspondent
TEL AVIV, Israel - Ester Neuroscience Ltd. announced preliminary results showing the first successful oral delivery of the company's proprietary antisense drug, EN101, to patients with myasthenia gravis (MG).
"The endpoints for measuring MG are very simple - either the patient can overcome neuromuscular weakness and fatigue characteristic of MG, or not," Ester CEO Eli Hazum said. "The well-established and widely used quantitative MG clinical severity scoring system is utilized to monitor the effects of EN101 administration on MG patients. This scoring system contains several clinical criteria [ocular muscles, vision, bulbar control, proximal muscle strength, vital capacity] as defined by the Myasthenia Gravis Foundation of America.
"We have been giving the drug for several days in clinical trials being conducted in the UK and Israel, and have found significant improvement in all parameters."
This is not the first time that oral delivery of an antisense drug has been tried, but it is the first time that an orally delivered antisense drug has reached clinical trials and shown to be effective. In March, Portland, Ore.-based biotechnology firm AVI BioPharma Inc. said it was bringing several antisense drugs to human clinical trials, including compounds to treat heart disease and cancer. In June, AVI and DepoMed Inc. announced their collaboration to develop oral formulations of one or more of AVI's antisense agents.
"Oral delivery is a critical attribute that has been long sought after by antisense drug developers," said Hazum, who also is chief scientific officer of Medica Venture Partners, a U.S./Israel health care investment fund and main backer of Ester Neurosciences. "In MG patients it will eliminate the need for daily or more frequent injections and is anticipated to greatly improve patient compliance. We have every reason to believe that the oral route will be equally effective for a wide range of peripheral neurological disorders that we are working on, including multiple sclerosis and Sjogren's syndrome."
EN101 is the lead compound in Ester's disease-modifying platform technology for the pre-expression control of the AChE protein. The technology is based on modulating the company's novel target, a stress-response variant of acetylcholinesterase (AChE), the enzyme degrading the neurotransmitter acetylcholine and thus balancing cholinergic transmission.