Upbeat prospects for some California clinical trial efforts from CIRM

Over at the California Stem Cell Report, David Jensen is reporting on some good news from CIRM for California on the stem cell clinical trial front.

Stem cell biotechs Asterias and Capricor have stem cell trials supported by 20+ million in CIRM funding each and have been hitting milestones. These trials are progressing and so far have good safety profiles. Asterias and CIRM have mentioned some possibly encouraging early hints at efficacy as well in its trial, and apparently there are hopeful hints from the Capricor trial too.

See the posts from CIRM here (a weekly summary kind of post that begins discussing Asterias) and here. For background, also see past posts I’ve done on both companies here and here in the archives, and see especially my interview with Asterias leadership from a few months back.

It’s early days for these trials and at these phases they are not really designed to look for efficacy so a conservative approach to discussing such trials is in order given the stage, but at this phase of the game for early clinical trials the news has been all one could hope for so far in both cases.


Asterias cells

The Asterias and Capricor trials are for spinal cord injury and Duchenne muscular dystrophy, respectively. The latter trial utilizes the Capricor CAP-1002 product, which is a cool allogeneic cardiosphere technology made from donor human heart tissue. A beating cardiosphere from a different source (IPSCs) can be seen in the video above. Asterias’ trial employs their OPC product made from hESCs, which is also inherently allogeneic. The idea of potentially repairing the injured spine via stem cells is intriguing.

I’m hoping in the next month or so to do a broader update on the stem cell and regenerative medicine biotech arena. By way of disclosure, I do not have any financial stake in either company discussed here.

J&J Bets Up to $325 million on Capricor: Big Pharma Stem Cell Move as Predicted for 2014

linda marban CapricorBig Pharma Giant Johnson & Johnson (J & J; $JNJ) announced today a $12.5 million investment in stem cells & regenerative medicine via the small biotech Capricor ($CAPR) and the potential for the bet to go into the hundreds of millions for the heart disease program.

It was only 8 days ago that I made my stem cell predictions for 2014 and #3 on the list was “Big announcement  from “Big Pharma” on stem cells or regenerative medicine.”

Chalk that up for the crystal ball of this blog at one for one so far for 2014.

According to the FierceBiotech today:

Later this week a little biotech named Capricor Therapeutics is set to begin a Phase II study of its new stem cell therapy with about $20 million in support from the California Institute for Regenerative Medicine (CIRM).

J&J’s role could shoot up to over $300 million depending on how things go.

I’d call that big money. Investors seem enthused as well as the CAPR stock is up nearly 50% today at the close.

The Capricor adult stem cell product is CAP-1002, which is in clinical trial and consists of allogeneic cardiosphere-derived cells. The trial may enroll up to 274 patients, which is impressive.

FierceBiotech quotes Capricor CEO Linda Marban (pictured above) as follows:

“One of the reasons why I was motivated to work on this deal is because of the statement it makes in the field,” says the CEO. “It says, OK, somebody very large and powerful is taking a look at this technology and saying there’s something there, and that’s the most exciting thing for me.”

I’ll be very curious to see what else big pharma might have in store on the stem cell/regenerative medicine front.

Disclosure: To my knowledge, I have no financial interests in the companies discussed in this piece.