Intellect Neurosciences, Inc. a biopharmaceutical company engaged in the discovery and development of disease-modifying therapeutic agents for Alzheimer's disease and other serious neurodegenerative conditions, with an internal diversified pipeline and licenses with major pharmaceutical companies covering products in late-stage clinical trials, announced that it has filed an orphan drug application with the Office of Orphan Products Development of the United States Food and Drug Administration (FDA) to have Orphan Drug Designation granted to its clinical stage drug candidate, OX1 (Oxigon) for the treatment of Friedreich's Ataxia (FA). The United States Orphan Drug Act of 1983 is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders.
FA is a rare, hereditary, progressive, neurodegenerative disease caused by a defective gene affecting energy production. About six thousand people in the United States are estimated to suffer from the disease. Orphan Drug designation would provide a seven-year term of market exclusivity for OX1 if ultimately approved by the FDA pending successful outcome of planned clinical trials and would enable the Company to take advantage of various financial and regulatory benefits, including government grants for conducting clinical trials, waiver of FDA user fees and certain tax credits.
The company anticipates filing for Orphan Drug designation in Europe as well. It recently reported obtaining a positive clinical data report from human phase I trials indicating that OX1 appears to be safe and well tolerated.
Dr Daniel Chain, chairman & CEO of Intellect commented: “The filing of an orphan drug application is an important step in our drug development strategy. Our decision pending grant of an Orphan Drug Designation, to develop OX1 as a potential ground-breaking treatment for FA, results from the convergence of two independent lines of inquiry over several years: one relates to the general properties and mechanisms of action of the drug and the other relates to a greater understanding of the pathogenic mechanisms underlying FA, especially the important role of oxidative stress. These parallel developments spanning more than a decade of research by numerous investigators, helped bring to light the strong potential of OX1 as a disease-modifying treatment for FA based on its unique multimodal antioxidant properties preventing damage to cell membranes as well as the oxidation of proteins and DNA. We believe that OX1 will ultimately prove disease modifying for FA and other neurodegenerative diseases. We are pleased by the interest shown in the program from numerous experts in FA research and grateful for the amount of assistance we received from FARA bringing us up to date on developments and clinical trial experience in this important field of biomedical research.”
Jennifer Farmer, MS, CGC, executive director, FARA commented: “FARA is excited that Intellect Neurosciences has decided to advance clinical research of OX1 in Friedreich's Ataxia. There is no treatment for this devastating disease. Research into the underlying mechanism of FA and damage that occurs in the cells strongly supports such a therapeutic candidate. FARA has supported the necessary clinical research infrastructure, such as a worldwide patient registry and clinical research network, to expedite planning and execution of clinical trials. We look forward to partnering with Intellect Neurosciences as their research efforts move forward.”
Friedreich's Ataxia (FA) is rare hereditary disease caused by a mutation in a gene which encodes frataxin, a protein essential for proper functioning of mitochondria, the energy pumps of the cell. In the absence of frataxin, iron in the cytoplasm builds up and causes free radical damage. It causes progressive damage to the nervous system, resulting in symptoms ranging from gait disturbance to speech problems; it can also lead to heart disease and diabetes. FA results from the degeneration of nerve tissue in the spinal cord, in particular sensory neurons essential for directing muscle movement of the arms and legs. The spinal cord becomes thinner and nerve cells lose some of their myelin sheath. The primary site of pathology is spinal cord and peripheral nerves. Symptoms typically begin sometime between the ages of 5 and 15 years, but may occur in the 20s or 30s. The disease usually presents with progressive staggering or stumbling and frequent falling. The symptoms are slow and progressive. The median age of death is 35 years. Currently there are no FDA approved drugs for FA.