The cell therapy biotech Capricor just released new Phase 3 data from its Duchenne Muscular Dystrophy (DMD) program
The firm is trialing its stromal cell product deramiocel for DMD.
These new results are very encouraging and stronger than past data.
Why am I excited here?
These newest deramiocel results released in a PR show statistically significant, big improvements for treated trial participants versus placebo controls. This was also a randomized, double-blind trial.
About a year ago, the company released some more preliminary data that also were encouraging. The stock jumped, but the firm then ran into headwinds at the FDA. The agency rejected the BLA just this July, a decision of CBER Director Vinay Prasad.
Now, there’s a good chance the FDA will approve deramiocel for DMD, probably by the middle of next year. The stock is way up again.
New Deramiocel data suggest strong benefit
What do the newest deramiocel data show? The cell therapy strongly slows DMD progression as reflected in preserved heart and limb muscle function. Both are important for DMD patients to maintain active lives.
For example, see the data above from Capricor. Note that these results have not yet been peer-reviewed or published.
Craig McDonald, M.D., my colleague here at UC Davis, who is the lead PI on all of this work, said of the new data, “We believe the HOPE-3 PUL results show statistically and clinically meaningful and significant treatment effects on both upper limb function and cardiomyopathy.” I’m not involved in this research.
It’s also important to note the crucial role of CIRM here in funding this clinical trial work.
Possible deramiocel mechanisms
One thing that still isn’t clear to me is how this cell therapy would lead to meaningful, lasting benefits. What’s the mechanism? The firm has said that deramiocel secretes beneficial exosomes or extracellular vesicles so perhaps that’s a factor here and works via immunomodulation? There could be other mechanisms as well.
In thinking more about such possible mechanisms, what exactly is CAP-1002 or deramiocel? The firm defined it in an earlier paper this way:
“Cardiosphere-derived cells (CDCs) are a type of stromal or progenitor cell, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. Exosomes secreted by CDCs also reprogramme fibroblasts, rendering them antifibrotic.”
This sounds a lot like the possible mechanisms related to MSCs being studied for various conditions.
If this mechanism holds up, there’s a beautiful, useful irony in fibroblast-type cells having an antifibrotic effect since fibroblasts can be part of harmful scar formation too.
The fact that cardiac stromal cells, especially allogeneic ones, are probably not going to engraft in a meaningful way, explains the need for continuous infusion. Patients get the cellular drug four times a year.
What will the FDA do?
As a STAT article on the new Capricor data points out, we should consider where deramiocel fits into things in the context of Sarepta’s experience with their investigational gene therapy for DMD. CBER Director Vinay Prasad has been a big part of the Sarepta rollercoaster with Elevidys.
Although I’d like to see more data from Capricor, its data to my eye seems far superior to anything we saw from Sarepta. Will the FDA see it that way?
From STAT:
“Capricor CEO Linda Marbán said she thinks the new, positive results from a larger, placebo-controlled study are sufficiently persuasive to reverse the FDA’s prior decision.
“We believe we can answer the issues that were raised by the FDA,” Marbán told STAT. “We’re going to be submitting these data to the FDA, and I don’t really see how they could possibly sling arrows at it.”
I hope the data, including once published, continue to look strong and Capricor gets its Deramiocel approval soon.