Press Release - Potential Treatment for Huntington's Disease

Regeneron and Medtronic to collaborate on potential treatment for Huntington�s Disease; Medtronic invests $10 million, agrees to support research on currently untreatable progressive neurological disease

TARRYTOWN, N.Y. and MINNEAPOLIS--(BUSINESS WIRE)--June 27, 1996--Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) and Medtronic Inc. (NYSE:MDT) announced today that they have entered into a worldwide exclusive joint development agreement to collaborate on research and development of a family of therapeutics for central nervous system diseases and disorders using experimental Regeneron compounds and Medtronic delivery systems.

The initial target of the collaboration will be the development of Regeneron's AXOKINEtm for the potential treatment of Huntington's disease, using Medtronic's implantable pump to infuse AXOKINE into the brain.

Under the collaboration, Regeneron will direct the preclinical development of the drug and retains all rights to manufacture and commercialize the drug, and Medtronic retains all rights to manufacture and commercialize medical devices to deliver drugs to the central nervous system. Medtronic will make payments to Regeneron upon the achievement of certain clinical milestones and will receive royalties on sales of any drug developed under the collaboration.

Medtronic has also purchased 460,500 shares of Regeneron Common Stock for $10 million and received five-year warrants to purchase an additional 107,400 Regeneron shares at an exercise price of $21.72 per share.

Huntington's disease is a slowly progressive inherited disorder of the brain. Its symptoms include uncontrolled movements, emotional disturbances, and loss of intellectual faculties. These symptoms typically begin in early to mid-adulthood, after the patient's childbearing years, though the disease can also begin at earlier or later ages. Death typically ensues 15 to 20 years after onset of symptoms. Approximately 80,000 people in the United States carry the gene for Huntington's disease. Each child of a person with Huntington's disease stands a 50% chance of developing the disease. About 30,000 of these individuals currently manifest symptoms of the disease, and the remainder will inevitably become ill.

Although Huntington's disease is best known for the abnormal, jerky, uncoordinated movements called "chorea," which is the most obvious symptom, Huntington's disease is a much more pervasive disorder that insidiously robs those it strikes of abilities most people take for granted. People with the disorder lose the ability to work, plan, and think clearly, and often suffer severe depression, obsessive-compulsive behavior, and sometimes even hallucinations and delusions, but they almost always recognize their families and are aware of their situation. As the disease progresses, the incessant, uncontrollable movements of chorea grow more exaggerated, other movement abnormalities emerge, the capacity to speak is lost (although the ability to understand is maintained), choking becomes a constant hazard, people become bedridden and totally dependent, and they eventually die, usually of aspiration pneumonia.

Huntington's disease results primarily from degeneration of nerve cells deep in the center of the brain, in a region called the basal ganglia (also known as the striatum or caudate-putamen). These cells are referred to as GABAergic neurons as they use the chemical messenger gamma-amino butyric acid (GABA). The basal ganglia are responsible for coordinating voluntary movement and for planning and sequencing muscle activity, and may also be involved in other complex behaviors. There are no effective treatments for the terrible degeneration of motor function and behavior of Huntington's disease.

In 1993, scientists identified the gene that is abnormal in all Huntington's sufferers. This gene is responsible for the production of a protein which is found in all cells, but when this protein functions abnormally, it confers a unique vulnerability on those cells of the basal ganglia which degenerate in Huntington's disease. Since it is not known precisely how the genetic abnormality translates into damage to nerve cells, scientists rely on animal models, in which they can induce changes in the brain similar to those found in Huntington's disease, to search for drugs that might delay disease onset, alleviate symptoms, or slow disease progression.

The most commonly studied animal model of Huntington's disease involves the injection of a chemical, quinolinic acid, into the brains of animals, thereby causing a pattern of cellular damage which remarkably resembles that seen in people with Huntington's disease. In preliminary results presented at the 4th International Congress of Movement Disorders held in Vienna, Austria in June 1996, Regeneron scientists showed that direct administration of AXOKINE, its proprietary second-generation neurotrophic factor, into the brains of rats protected nerve cells from quinolinic acid-induced degeneration. A full account of this work will soon be published in a peer-reviewed scientific journal.

Ronald M. Lindsay, Ph.D., Regeneron's vice president, neurobiology, commented, "Our promising early preclinical studies encouraged us to seek out Medtronic, an internationally recognized leader in the delivery of drugs with implantable devices, to study jointly the therapeutic potential of AXOKINE for Huntington's disease."

"Our first steps will include conducting certain preclinical efficacy and toxicology studies. While we cannot predict the results of any experiment, we are looking forward to joining with Medtronic in this important and challenging effort."

Commenting on the announcement of the collaboration, Nancy Wexler, Ph.D., president of the Hereditary Disease Foundation and Higgins professor of neuropsychology at Columbia University, said, "Today is an important moment for families with Huntington's disease. Regeneron and Medtronic are launching a collaboration that may lead to the development of a new treatment for this catastrophic disease. Right now, there is a gene test that can tell people whether they will develop Huntington's disease in the future, but there is no way to prevent or even delay the onset of symptoms and nothing to ameliorate them once they begin. Huntington's disease devastates body and mind and can even destroy a person's healthy years by filling them with dread anticipation." Wexler continued, "We are very pleased that Regeneron and Medtronic are combining their unique capabilities to attack this disease. We look forward to working with them in their attempts to bring a new therapy to families with Huntington's disease."

Kent C. Westbrook, M.D., chairman of the board of trustees of the Huntington's Disease Society of America (HDSA), said, "Patients and families affected by Huntington's disease have become close observers of the science arena looking for hope. Currently, there is no treatment for the disease. While the discovery of the gene permits prediction of the development of the disease, it has not provided a basis for treatment. Until a treatment based on the genetic abnormality is available, other treatments must be explored." Westbrook also said, "The announcement of a collaborative effort by Regeneron and Medtronic brings hope to many people throughout the United States and the world. We look forward to their investigation of this new potential treatment. HDSA will cooperate in any way possible to facilitate this research."

John A. Meslow, president of Medtronic's Neurological Business, noted that the Regeneron collaboration is another step in the implementation of Medtronic's strategy to develop applications of its technologies to address unmet medical needs. "We are extremely pleased to collaborate with Regeneron in the development of a potential therapy for the thousands whose lives are so severely restricted by Huntington's disease." said Meslow.

Regeneron's recent news releases can be obtained by dialing (800) 331-0841 for fax copies or by accessing the Internet at

Send comments to Renette Davis by clicking here.

Last updated: Dec. 5, 2010