Brainstorm Cell Therapeutics Inc (NASDAQ:BCLI) is running up on the back of a data presentation for the companies lead amyotrophic lateral sclerosis (ALS) asset. The asset in question, NurOwn, is currently under investigation as part of a phase II program, and the data came in the form of a presentation by lead investigator Dr. James Berry, at the 27th International Symposium on ALS/MND in Dublin, Ireland.
ALS is a huge unmet need, and any company with a potential pathway to commercialization in the space is going to attract considerable speculative interest. With the most recent data, Brainstorm has laid some pretty strong and numerous stones along this pathway, and that’s why the company is seeing its market cap boost in the way it is.
With a number of catalysts near to medium term, and based on the inference of the latest data, we think there’s plenty of run room left on this one before the trial concludes and NurOwn advances through to a pivotal investigation.
Here’s what you need to know.
ALS was made famous a few years ago with the ice bucket challenge, where a number of high profile celebrities, business leaders, etc poured a bucket of ice water over their heads. Before this, however, it was also well known for being the condition with which Stephen Hawking has suffered since his twenties. The condition is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. You can break down the etymology of the name for a pretty clear description of what it is, and what it does. The Amyotrophic element derives from Greek. “A” means no. “Myo” refers to muscle, and “Trophic” means nourishment – “No muscle nourishment.” Lateral refers to the region in the spine where the movementrelated nerve cells are located, and sclerosis refers to the scarring that comes about as the result of muscle degeneration. Essentially, the muscles waste away (usually quite gradually, and painlessly) to a point that they are no longer functional.
There’s no cure, and as illustrated by Stephen Hawking’s condition, is completely debilitating in its later stages.
So, what is BrainStorm doing about it?
As mentioned, its lead ALS asset is called NurOwn, and it’s rooted in what are called mesenchymal stem cells (MSCs). These are pretty widely used in the medical space already (things like bone grafts, etc.) What BrainStorm does is takes MSCs and, by growing them in proprietary conditions, converts them into what can best be described as biological factories. These secrete a variety of neurotrophic factors (NTFs). NTFs are growth factors known to support the survival of neurons in a variety of conditions, and in animal models of many neurodegenerative diseases.
With the investigation, it’s using a sort of surrogate approach. Basically, if the company can demonstrate that NurOwn results in an increase of certain NTPs in patients with ALS, the assumption is that this is going to improve the symptoms of (or at lease slow or halt the degeneration associated with) the condition.
And – here’s the important bit – it did.
In the samples of those patients treated with NurOwn, a statistically significant increase in levels of VEGF, HGF and LIF (all of which are NTPs) was observed from pre- to post-transplantation. The treatment also resulted in a statistically significant reduction in inflammatory markers (MCP-1 and SDF-1) over this period. This was not observed in the placebo group. So, the inference here, is that there’s some strong evidence of efficacy in a condition that has – perhaps until now – eluded any efficacious therapy.
It’s early days, of course, and just as with many biotechs at this end of the space, Brainstorm doesn’t have much cash on its books. We’re going to see some dilution, then, and there’s no guarantee that a) the company can replicate these data on a larger scale next trial or b) that this replication will translate to improved ALS symptoms (as opposed to just increased NTF levels).
There’s a lot of potential here, however, and we think the asset associated value boost is going to mitigate the impact of any potential raise on the share base.
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Disclosure: We have no position in BCLI and have not been compensated for this article.