Japan is Breathing Life into the Stem Cell Space, Astellas Pharma (ALPMF) & Pluristem Therapeutics Inc. (PSTI) Are Benefiting

The program showed some good results in a Phase I run by Ocata, and now the AIRM is pushing through a Phase II in Japan and the US. From a timeline perspective, since Astellas took over the Ocata program things have gone pretty quiet. It’s a notoriously long-winded process to harvest pluripotent stem cells so chances are we are looking at a 3 to 5-year study rather than a quick turnaround. It’s worth keeping an eye on, however.

If Astellas Pharma (OTCMKTS:ALPMF) can demonstrate its ability to regenerate RPE cells in both SD and AMD patients, the potential wider indications are far beyond ophthalmology in scope. To keep an eye on perspective here in terms of value, Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) increased in value by over a factor of 10 thanks largely to its EYLEA treatment for wet AMD, a much rarer form of AMD with similar symptoms.

This is a pretty risk-mitigated exposure to the Japanese stem cell space, since Astellas has such a wide portfolio of products and R&D candidates. Next, let’s take a look at what we might consider a direct allocation.

Pluristem Therapeutics Inc. (NASDAQ:PSTI)

Pluristem has taken a very different approach to stem cell therapy, in that it uses stem cells derived from the placenta right after the birth of a baby. The main difference is that these cells are stromal cells, which have a far lower immunogenicity than, say, the cells Astellas is using. This means they are not normally affected by the immune system in any capacity.

Low immunogenicity means that there doesn’t need to be any immune matching before use. Cells with high immunogenicity would get recognized by the host immune system as foreign, and attacked. In traditional stem cell therapy, therefore, physicians need to carefully match donor cells with a host so as not to evoke an immune response. The placenta must have low immunogenicity because it protects two different immune systems – mother’s and baby’s – from attacking each other.

With Pluristem’s treatments, this matching isn’t necessary, and so the indications it targets are easier to go at from a logistical standpoint. This reduces trial time, cost etc., and should speed up the path to commercialization considerably. Couple this with the rapid regulatory pathway in Japan, and that path becomes even shorter.

Pluristem Therapeutics Inc. (NASDAQ:PSTI) is going after many indications right now, but one of the lead targets is a condition called critical limb ischemia. CLI is when fatty substances build up in the walls of the arteries generally in the legs and cause blockages. There are around 1.7 million CLI sufferers in the US, Europe and Japan, and prognosis is poor as things stand. The majority of patients with any sort of severe form of the condition will require amputation and many will die. It’s an unmet need, and it costs the US somewhere in the region of $10 billion annually.

Phase I data from two trials has led to the acceptance of Pluristem and its treatment – called PLX-PAD into accelerated regulatory programs in both Japan and Europe. It’s the only treatment in the world that is in both programs. What does this mean? For both, it means that the company likely won’t need Phase III data before it can pick up preliminary approval in Japan and possibly Europe. Instead, a pivotal trial demonstrating safety, and then an approval application combining the efficacy data from both the Phase I’s and the safety and efficacy data from the Phase II’s. This results in obvious cost and time savings. Both trials are expected to kick off before the close of this year, meaning Pluristem Therapeutics Inc. (NASDAQ:PSTI) could be targeting marketing approval in both Europe and Japan by early 2018, with enough cash on its balance sheet to get there to boot.

Note: This article is written by Mark Collins and was originally published at Market Exclusive.