NSI-566

Candidate & Indication Preclinical Phase 1 Phase 2 Phase 3
Cell Therapy*
NSI-566 US Amyotropic Lateral Sclerosis
Preclinical Phase complete
Phase 1 Phase complete
Phase 2 Phase in progress
Phase 3 Phase not started
NSI-566 US Chronic Spinal Cord Injury
Preclinical Phase complete
Phase 1 Phase in progress
Phase 2 Phase not started
Phase 3 Phase not started
NSI-566 China Ischemic Stroke
Preclinical Phase complete
Phase 1 Phase in progress
Phase 2 Phase not started
Phase 3 Phase not started

*Neuralstem corporate strategy is to seek partnerships/collaborations to fund the programs.

About Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis, or ALS, is a disease of the nerve cells in the brain and spinal cord that control voluntary muscle movement. ALS is an orphan condition in the U.S. and EU and approximately 6,400 people in the U.S. are diagnosed with the condition each year. The incidence of ALS is two per 100,000 people, and it is estimated that more than 20,000 Americans may be living with ALS at any given time. In ALS, nerve cells (neurons) waste away or die, and can no longer send messages to muscles. This eventually leads to muscle weakening, twitching, and an inability to move the arms, legs, and body. The condition slowly gets worse. When the muscles in the chest area stop working, it becomes hard or impossible to breathe.

NSI-566 as a Potential Treatment

NSI-566 is under development as a potential treatment for ALS by providing cells designed to nurture and protect the patients' remaining motor neurons; and possibly repair some motor neurons which have not yet died but which are diseased. Neuralstem received orphan designation by the FDA for NSI-566 in ALS. NSI-566 was evaluated in Phase 1 and Phase 2 safety studies in 30 patients. The data showed that the intraspinal transplantation of the cells was safe and well tolerated. Subjects from both the Phase 1 and Phase 2 continue to be monitored for long-term follow-up evaluations. To date, substantially all the clinical costs of our ALS studies have been funded by grants.

About Chronic Spinal Cord Injury

A spinal cord injury, or SCI, generally refers to any injury to the spinal cord that is caused by trauma instead of disease although in some cases, it can be the result of diseases. It is estimated that there are 12,500 new cases of SCI per year and that at any given time, there are between 240,000 and 337,000 people in the United States that are living with SCI. Chronic spinal cord injury generally refers to the phase beginning 6 months after the initial injury. SCIs are most often traumatic, caused by lateral bending, dislocation, rotation, axial loading, and hyperflexion or hyperextension of the cord or cauda equina. Motor vehicle accidents are the most common cause of SCIs, while other causes include falls, work-related accidents, sports injuries, and penetrations such as stab or gunshot wounds. In certain instances, SCIs can also be of a non-traumatic origin, as in the case of cancer, infection, intervertebral disc disease, vertebral injury and spinal cord vascular disease.

NSI-566 as a Potential Treatment

We believe that NSI-566 may provide an effective treatment for chronic spinal cord injury by "bridging the gap" in the spinal cord circuitry created in traumatic spinal cord injury and providing new cells to help transmit the signal from the brain to points at or below the point of injury. We are carrying out a Phase 1 trial to test this possibility.

The study has enrolled four AIS-A thoracic spinal cord injury subjects (motor and sensory complete), one to two years post-injury at the time of stem cell treatment in a Phase 1 safety study. The stem cell treatment was found to be safe and well-tolerated by the subjects enrolled and there were no serious adverse events at six-month post transplantation. Subjects are currently being monitored for long-term follow-up evaluations. The FDA has approved the protocol amendment to treat an additional cohort of four cervical spinal cord injury patients. Substantially all of the clinical costs of this study have been, and will continue to be, funded by grants arranged through UCSD.

About Ischemic Stroke

Ischemic strokes, the most common type of stroke, occur as a result of an obstruction within a blood vessel supplying blood to the brain. Approximately 15 million people worldwide suffer stroke of which it is estimated that 87% of all strokes are ischemic strokes. Post-stroke motor deficits include paralysis in arms and legs and can be permanent.

NSI-566 as a Potential Treatment

We believe that NSI-566 may provide an effective treatment for restoring motor deficits resulting from ischemic stroke by both creating new circuitry in the area of injury and through repairing and or nurturing diseased cells to improve function in patients. Currently Neuralstem is conducting a Phase 1 safety trial in 9 subjects with motor deficits due to ischemic stroke. The trial is being conducted at BaYi Brain Hospital in Beijing, China. Subjects are currently being monitored through their 24-month observational follow-up period. The trial is being conducted by Suzhou Neuralstem, a wholly owned subsidiary of Neuralstem in China.