Our current development effort focuses on the innovative M102 program. M102 is a proprietary disease-modifying drug candidate based on recently understood disease pathways in neurogenerative disease. Neurodegenerative disease represents a range of medical conditions which primarily affect the neurons in the human brain. Neurons are the building blocks of the nervous system which includes the brain and spinal cord. Normally, neurons do not reproduce or replace themselves, so when they become damaged or dead, they cannot be replaced by the body. Neurodegeneration is the progressive loss of structure or function of neurons, including death of neurons. Many neurodegenerative diseases occur as a result of neurodegenerative processes. Such diseases are incurable, resulting in progressive degeneration and/or death of neuron cells. As research progresses, many similarities appear that relate these diseases to one another on a sub-cellular level. There are many parallels between different neurodegenerative disorders, including atypical protein assemblies and induced cell death. Neurodegeneration can be found in many different levels of neuronal circuitry, ranging from molecular to systemic. Examples of neurodegenerative diseases include Parkinson's Disease, Huntington's Disease, Amyotrophic Lateral Sclerosis, Batten Disease, and Friedreich's Ataxia.
Parkinson's disease is a neurodegenerative disorder that affects predominately dopaminergic neurons in a specific area of the brain called substantia nigra. People with this disease may experience tremor, slowness of movements, limb rigidity, as well as gait and balance problems. Symptoms generally develop slowly over years. Every year, about 60,000 Americans are diagnosed with Parkinson's disease.
Huntington’s disease is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It deteriorates a person’s physical and mental abilities during their prime working years and has no cure. Today, there are approximately 30,000 symptomatic Americans and more than 200,000 at-risk of inheriting the disease.
AMYOTROPHIC LATERAL SCLEROSIS
Amyotrophic lateral sclerosis is a specific disease which causes the death of neurons controlling voluntary muscles. This disease is characterized by stiff muscles, muscle twitching, and gradually worsening weakness due to muscles decreasing in size. This results in difficulty speaking, swallowing, and eventually breathing. Amyotrophic lateral sclerosis afflicts about 25,000 patients in America.
Batten disease is an inherited neurodegenerative disease. Batten disease mainly affects infants, toddlers and children. The symptoms of Batten disease are caused by the disorder of neuronal ceroid lipofuscinoses (NCLs). As NCLs accumulate, they cause the death of cells called neurons in the brain, retina and central nervous system. Batten disease occurs in an estimated 2 to 4 of every 100,000 births in America.
Friedreich's ataxia is an autosomal recessive inherited disease that causes progressive damage to the nervous system. It manifests in initial symptoms of poor coordination such as gait disturbance; it can also lead to scoliosis, heart disease and diabetes, but does not affect cognitive function. Friedreich's ataxia affects around 1 in 50000 people in the United States.