Notes from Neuralstem Presentation at Biotech Showcase Conference, January 13, 2015 (CUR, Buy, $3.09)
Richard Gar, CEO of Neuralstem (CUR), made a presentation at Biotech Showcase Conference in San Francisco on January 13, 2015. So far, everything seems to be trending positively in the trials. The Company is roughly on schedule with its clinical trials that are currently ongoing in four different disease states. The NSI-566 neural stem cells are being tested in ALS, ischemic stroke and chronic spinal cord injury and will soon start a trial in acute spinal cord injury. The small molecule drug NSI-189 is being studied in major depressive disorder.
There was only one thing in the presentation that was really new to me and that was a plan to begin clinical trials of CUR’s stem cells to deliver large molecules into the brain. Using gene transfer technology, a DNA sequence encoding for a protein is incorporated into a stem cell which is then implanted in the brain; this allows for the secretion of the protein for a sustained period of time. The first protein selected is the growth factor IGF-1 which will begin a phase 1 trial in Alzheimers and Parkinson’s patients in 2016.
The lockdown of data on the phase 2 trial of NSI-566 neural stem cells in ALS will occur in late January and it may take a few weeks to collect and analyze the data. This points to seeing results in late February or March. The next trial in ALS will begin in April 2015. The Company has not yet discussed the trial design and size of the trial and whether it may be a potential registration trial. We should hear more of this in February or March. The ALS drug will be commercialized by Neuralstem if it is successful in clinical trials.
Date was shown for Ted Harrada and two other patients from his cohort that showed that their ALSFRS-r scores were stable for 1150 or more days (3+ years) post-surgery. ALS is a disease that inexorably progresses in almost all patients. It is unheard of except in a small number of atypical patients (i.e. Stephen Hawking) to see stability. The ALSFRS-r scale is 48 for perfectly normal people and newly diagnosed ALS patients are in the 35 to 40 range. Typically, patients deteriorate at a rate of about 0.5 to 0.7 points per month on this scale and dies at ALSFRS-r scores of 15 to 20. Stability for a three year period of time is extraordinary.
The trial of the small molecule drug NSI-189 in major depressive disorder will start in April. It should be about 150 patients in 20 centers. It is expected to take 18 to 24 months to complete. This product will be partnered after the results of the phase 2 trial are known assuming success in the trial. Mr. Gar discussed the positive phase 1 results for NSI-189. I analyzed them in a report last June if you are interested.
Mr. Gar mentioned briefly the ischemic stroke trial in China in which the same NSI-566 neural stem cells used in the ALS trial are injected directly into the brains of stoke victims. The initial phase of the trial that is aimed at determining dosing is being done in 18 patients and is a single arm trial in ischemic stroke patients. If results warrant, the trial will roll into a phase 2 randomized study in about 100 patients. No comment was made on results to date but I did publish an anecdotal report recently that was encouraging.
The chronic spinal cord injury trial involving four patients with thoracic injuries will complete enrollment in 1Q, 2015 and there will be a six month follow-up so that we may learn results in 3Q, 2015. It will be followed by surgeries in patients with cervical injuries.
The acute spinal cord injury trial could begin in a few weeks in South Korea. It will also use NSI-566 neural stem cells.
Tagged as CUR, Inc., Neuralstem, Inc., NSI-189 in major depressive disorder, NSI-566 neural stem cells in ALS + Categorized as Smith On Stocks Blog
Thought Garr stated in the present he wasn’t expecting topline until end of Q1 / beginning of Q2. Any reason you expect data in feb/march?
Thanks, Larry. Appreciate your work.
Larry-
The large molecule delivering IGF-1 into the brain, for treating Alzheimer’s (and Parkinson’s)… I thought that there was some talk earlier about the small molecule NSI-189 for that purpose, after research on depression… has the company stopped talking about that, or was that actually your musings at the time?
I appreciate your work as well. -L.
They are advancing NSI-189 into phase 2 in April. It is a small molecule. This is a new technology that enables large molecules to be delivered that would otherwise be blocked by the blood brain barrier.
I listened to only the last third or so of the call but heard Mr. Gar say something along the lines of the small molecule part of the company comprising a separate unit from the stem-cell part and that this unit had equal standing in the company and he expects it will eventually become the larger of the two because the markets are larger.
Is this a new thing? Do you see this as a significant statement or is it merely an attempt to get the market to assign some value to the NSI-189 program?
Thanks very much.