While heading the Israeli company, Mindset BioPharmaceuticals, Dr. Daniel Chain developed a drug compound known as OX1. The drug has now resulted in a $126 million global licensing deal for Intellect Neurosciences which undertook further testing. The deal covers the use of the clinical stage drug candidate for the treatment of Friedreich’s Ataxia and other neurodegenerative diseases.
“The preclinical development work on the compound was done at Mindset in Jerusalem many years ago – before the investors pulled the plug,” commented Dr. Chain in an interview with BioIsrael. Dr. Chain, who did his doctoral studies in biochemistry at the Weizmann Institute, founded Mindset in Jerusalem in 1999 and during his tenure at that company also laid the groundwork for Intellect Neurosciences’ ANTISENILIN technology for Alzheimer’s disease which underlies products in Phase 1, Phase 2 and Phase 3 clinical trials by major pharmaceutical companies who have purchased royalty-bearing licenses from Intellect.
Dr. Chain currently is the Chairman and CEO of Intellect Neurosciences, Inc. (OTCBB: ILNS), a Manhattan-based company. The ViroPharma deal will bring the company an upfront payment of $6.5 million and milestone payments of $120 million. Under the agreement, Intellect will also receive two-tiered royalty payments up to low single digits from net sales of OX1.
OX1 is a multimodal, metal-binding, extremely potent antioxidant molecule, which has been demonstrated to protect nerve cells from highly oxidizing neurotoxins. ViroPharma plans to develop and commercialize OX1 as a treatment of Friedreich’s Ataxia and possibly other diseases for which OX1 may qualify for orphan drug designation.
Intellect recently announced completion of Phase 1 clinical trials for OX1. Dr. Chain has played a leadership role in the development of OX1 since its therapeutic potential was originally discovered by scientists at NYU Medical School and University of South Alabama in the late 1990s.
In addition to OX1, the company’s pipeline includes Alzheimer’s monoclonal antibody products licensed to major pharmaceutical companies, melatonin for treatment of amyloidosis-related diseases, Recall-Vax, a novel Alzheimer’s vaccine, IN-N01 a humanized monoclonal antibody targeting beta amyloid and tau immunotherapy approaches.
Friedreich’s Ataxia is rare hereditary disease affecting 1 in 50,000 individuals of European descent 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. The disease 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. The ataxia of Friedreich’s ataxia 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.