Amyotrophic Lateral Sclerosis is a progressive neurodegenerative disease that attacks nerve cells in the brain and spinal cord, leading to complete paralysis, and eventually, death. Also known as Lou Gehrig's disease, Amyotrophic Lateral Sclerosis, or ALS, is said to affect as many as 30,000 Americans, with 5,600 new cases being diagnosed each year.
Currently, there are two FDA-approved drugs to treat ALS namely, Sanofi-Aventis' (SNY) Riluzole, which prolongs life by 2-3 months, and Avanir Pharmaceuticals Inc.'s (AVNR) Nuedexta, which treats emotional instability that accompanies this disease.
Developing a neural stem cell therapy for ALS is Rockville, Maryland-based biotechnology company Neuralstem Inc. (CUR).
For readers who are new to Neuralstem, here's a brief overview of the company's pipeline and the upcoming events to watch out for...
The company is testing its cell product - NSI-566 human spinal cord stem cells, via transplantation technique, in the treatment of ALS symptoms. The phase I NSI-566 study was completed as recently as August of this year. This groundbreaking trial, the first to be approved by the FDA to test neural stem cells in patients with ALS, began in January 2010.
The trial was designed to enroll up to 18 patients, the last of which was treated in August of this year. The entire trial concludes six months after the final surgery.
The interim data on the NSI-566 ALS trial will be updated on October 8, 2012, according to the company.
NSI-566 will also be evaluated in treating motor deficits due to ischemic stroke. The company has received approval to commence a combined phase I/II ischemic stroke trial with NSI-566 in China, and it is expected to begin early next year.
The trial is designed to test up to 118 patients who have suffered an ischemic stroke with chronic residual motor disorder with NSI-566 cell line, 4-24 months post-stroke. The duration of the combined trial, including patient monitoring and data collection, is approximately two years.
Ischemic strokes, the most common type of stroke, occur as a result of an obstruction within a blood vessel supplying blood to the brain. After a stroke, many patients suffer from paralysis in arms and legs, which can be permanent.
Neuralstem's request for U.S. approval to begin a phase I study with NSI-566 neural stem cells for spinal cord injury, filed in August 2010, is pending approval. According to the company website, the spinal cord injury trial is expected to commence in South Korea in 2013.
Also under clinical development is a pharmaceutical compound - NSI-189 for the treatment of major depressive disorder, which is under phase I study. The phase Ia portion of the trial was initiated last February and was completed in October of 2011. The phase Ib portion of the trial got its first patient dosed in June of this year. The company anticipates a total of 24 patients to be dosed in the phase Ib portion.
Apart from major depressive disorder, the company plans to develop NSI-189 for other psychiatric and cognitive disorders as diverse as chronic traumatic encephalopathy, Alzheimer's disease, anxiety, and post-traumatic stress disorder.
Last August, the company was selected as the primary subcontractor under the U.S. Department of Defense contract to develop its human neural stem cell technology for the treatment of cancerous brain tumors. Neuralstem received $625,000 under the contract. The contract expired in June of 2012, and whether or not the DOD will exercise its option for future years under this contract remains to be seen.
A quick look at Neuralstem's balance sheet...
Since inception in 1996, the company has had a history of losses, and at June 30, 2012, had accumulated losses totaling $103 million. Historically, Neuralstem has derived revenue primarily from selling treated samples for gene expression data from stem cell experiments; providing services under various grants and contracts, and through the licensing of the use of its intellectual property.
In the second quarter ended June 30, 2012, Neuralstem's net loss narrowed to $2.37 million or $0.04 per share from $3.65 million or $0.08 per share in the year-ago quarter. The company's revenue in Q2, 2012 was about $78 thousand for its services as principal subcontractor under the DOD contract while no revenue was recognized in Q2, 2011.
The company ended Q2, 2012 with cash and cash equivalents of $2.54 million. In August, $2.76 million in gross proceeds was raised in a public offering, and in the following month, $7 million was raised in a registered direct offering.
Neuralstem shares have thus far hit a 52-week low of $0.42 and a 52-week high of $1.96. CUR closed Thursday's trading at $1.28, up 12.28%. The stock has gained over 90% in the last one month.
by RTT Staff Writer
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