Nov 10 1998
CAMBRIDGE, Mass. - PRNewswire - Vertex Pharmaceuticals Incorporated announced today that it has begun the first-ever clinical trial investigating the potential of a neurophilin compound to slow or reverse neural dysfunction in humans. The Company has started a Phase II clinical trial with timcodar dimesylate (VX-853), an orally delivered neurophilin ligand, in patients with diabetic neuropathy. Approximately 70 patients will be enrolled in the trial, which will be conducted in 8 centers in the United States.
Neurophilin ligands are small molecules that have the potential to promote neurite outgrowth and accelerate recovery following nerve injury. They may play a future role in the treatment of a variety of acute and chronic neurological conditions. Neurophilin compounds designed by Vertex are orally deliverable, and have been shown to accelerate functional recovery and promote nerve growth in several different animal models of central and peripheral nervous system injury.
"Peripheral neuropathies represent an important starting point for clinical development, because of both the lack of existing therapies for these patients and the possibility of affirming the broader potential of neurophilin ligands in a variety of neurological indications," said Dr. Vicki Sato, Senior Vice President of Research and Development and Chief Scientific Officer of Vertex.
"The safety data we have collected to date gives us confidence that we can move timcodar forward into Phase II testing and begin to understand its potential for producing clinical efficacy.
Results of several laboratory and preclinical studies, which we expect to present publicly in the coming months, have demonstrated timcodar to have potent activity in promoting nerve growth and restoring nerve function."
Clinical safety and pharmacokinetic data supporting timcodar's potential as a therapeutic compound are available from a single-dose study of four different doses in healthy volunteers, which was completed earlier in 1998. In vivo laboratory experiments indicate that timcodar can prevent neural dysfunction in a model of diabetic polyneuropathy and in vitro results have shown timcodar's ability to promote neurite outgrowth. Results presented at the Society for Neuroscience 27th Annual Meeting in October 1997 showed that an oral neurophilin compound designed by Vertex that is related to timcodar could enhance functional recovery through neuronal regeneration in animal models of peripheral nerve injury, Parkinson's disease, and spinal cord injury.
The double-blind, placebo controlled Phase II clinical trial announced today will enroll approximately 70 patients with diabetic neuropathy in 8 clinical centers in the United States, under an investigational new drug application (IND) that has been reviewed by the U.S. Food and Drug Administration (FDA).
Primary objectives will be to evaluate the safety and tolerability of six different dose regimens of timcodar administered orally over a 28-day period. Nerve function will also be monitored.
"Neuronal regeneration represents a major market opportunity, and we are positioned at the forefront of clinical research in this area," said Dr. Joshua Boger, Chairman, President, and CEO of Vertex. "Vertex's information- intensive approach to drug discovery and development has enabled us to make rapid progress with timcodar, and allows us to continue building our innovative clinical pipeline."
In August 1998, Schering AG, Germany and Vertex formed a collaboration to research, develop and commercialize novel, orally active neurophilin compounds to promote nerve growth and repair. Under terms of the agreement, Schering AG, Germany agreed to provide $28 million in upfront and research support payments over five years. An additional $60 million in milestone payments is possible through the development of specific compounds arising from the collaboration. Under the agreement, Schering AG has an option to co-develop timcodar with Vertex.
Neuropathy is a frequent long-term complication of diabetes that can involve persistent painful sensation or loss of sensation, most commonly in the hands, feet and legs. Diabetic neuropathy affects an estimated 1.3 million patients in the United States, and if severe, can lead to amputation of an affected limb. According to the American Diabetes Association, approximately 56,000 amputations in the United States each year are due to diabetic neuropathy.
Timcodar dimesylate began development at Vertex under the name VX-853, as an oral agent for the reversal of cancer multidrug resistance (MDR). A Phase I/II trial examining high doses of VX-853 in patients with advanced cancer is ongoing.
The dose range under investigation for neurological diseases is substantially lower than that believed to be optimal for MDR.
Vertex holds an issued United States patent directed to compound classes that include timcodar. Vertex also holds a United States patent application under a Notice of Allowance claiming the use of these compound classes for stimulating nerve growth and for the treatment of various neurological diseases.
Vertex Pharmaceuticals Incorporated is engaged in the discovery, development and commercialization of novel, small molecule pharmaceuticals for the treatment of diseases for which there are currently limited or no effective treatments. The Company is a leader in the use of structure-based drug design, an approach to drug discovery that integrates advanced biology, biophysics and chemistry. The Company is concentrating on the discovery and development of drugs for the treatment of viral diseases, multidrug resistance in cancer, autoimmune and inflammatory diseases, and neurodegenerative diseases.
There can be no assurance that clinical trials will continue, that initial results will be predictive of any future results, that drugs under development by Vertex or its partners will receive marketing approval from the U.S. Food and Drug Administration or other regulatory authorities, or that drugs, if any, which receive such approval will be marketed successfully. There can be no assurance that Schering will exercise its option to participate in the development and commercialization of timcodar dimesylate. Investors are also directed to consider other risks and uncertainties discussed in Vertex documents filed with the Securities and Exchange Commission.
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