autologous v allogeneic

life scientist interviewing jason mc carthy JM: Celyad is an interesting company. This company is working on cardiopoietic stem cells for congestive heart failure (CHF), but it's also involved in CAR T-cell therapy. The company is developing C-Cure (autologous bone marrow-derived mesenchymal cardiopoietic cells) for CHF, and is expecting topline data from its 240-patient Phase 3 trial called CHART-1 sometime in mid-2016. This will be a pivotal milestone for the company and the regenerative medicine space. It's a European study, but Celyad is expecting to do a U.S. study, CHART-2, and the company is in discussions with the FDA right now to determine the final protocol. We expect CHART-2 to start sometime before the end of this year, which would be another milestone for the company.

This CHF study has some parallels with and some implications for Mesoblast Ltd. (MSB:ASE; MBLTY:OTCPK), an Australian company partnered with Israel-based Teva Pharmaceutical Industries Ltd. (TEVA:NYSE). Mesoblast is conducting a 1,700-patient study in CHF with Revascor (mesenchymal precursor cells) that is not ready to read out yet—it's still a few years away. If Celyad can show some progress, or even just a signal that autologous cardiac cells can work for CHF, that could do a lot for other companies in this space, like Mesoblast and Teva.
TLSR: Revascor is an allogeneic, or "off-the-shelf," therapy, whereas C-Cure is an autologous therapy, in which cells must be harvested from and processed for each patient individually. Does Celyad's autologous therapy, with its much higher cost of goods sold (COGS), give you pause?
JM: I think anything autologous is going to be a cause for concern in terms of valuation, and that extends all the way to CAR T-cell therapies. We think of autologous cell therapy as first-generation technology, and allogeneic cell therapy, which is a few years away still, as second generation. Allogeneic is where the space wants to go, but I think the first successes will be in autologous cell technology. If you can harness the body's endogenous repair mechanisms with an autologous regenerative cell, that opens the door to allogeneic therapies.
Mesoblast has shown, so far, that its allogeneic cells are safe. I think these two companies can complement each other. If Celyad shows that, yes, autologous cells do work in CHF, and then Mesoblast comes along and says, "Well, now we can do it allogeneic," the progression of regenerative medicine can move forward from there.
TLSR: Celyad was originally scheduled to collect its final data on this 240-patient Phase 3 trial in December 2014. Does it have the data yet? [Editor's Note (9/29/15): Per Celyad, the company expected to complete enrollment of 240 patients for the Phase 3 CHART-1 trial in December 2014 and release the data readout mid-2016.]
JM: No. It just dosed the last patient sometime over the summer. It's going to take 18 months or so. We need time to see how the patients do. No data have been released yet. Cheers Vin
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