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https://www.gamida-cell.com/wp-content/uploads/TCT_2021_horw...

Phase 3 results above.

As a general rule is a study like this best avoided? They are not measuring survivability, but "Time to neutrophil engraftment"

The market responded well to the report, as the stock is up 70% after hours.

Survivability seemed to be their secondary end point.
Secondary Endpoint: Days Alive and Out of the Hospital
in the First 100 Days Post-Transplant (ITT Population)
Omidubicel: Median 60.5 days
Control: Median 48.0 days
p = 0.005

Disease-free and Overall Survival
(ITT population)
This does not seem very impressive to me.
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hey there! Great point!

Time to engraftment is actually somewhat important since these patients are kept in a hospital awaiting to have an intact immune system. basically, if you let them into the wild, they (may or may not) catch something out there. So what youre really doing is (hopefully?) decreasing time to hospital discharge.

Now...

you are correct that overall survival favors Gamida, but its not statistically significant. A larger trial might show this.

With that said...
A wild card could be the relapse rate. This does *not* favor Gamida, but is not statistically significant.

One way to look at this from a 10,000 foot view financial aspect is that Gamida, in theory, would have to show a financial advantage to their treatment when you take into account a potentially higher relapse rate. That would likely need to be better than an 8% cost savings (25% vs 17% relapse).

FWIW, I think this is likely positive, and had posted on GMDA before. I think this might have some room for investors; personally, I would have liked to see the relapse rate favor GMDA. Then I think it has a near bulletproof argument until more data comes out.


Back to your question: is engraftment a good endpoint. Typically, I would prefer a patient oriented outcome (like mortality!), and engraftment seems to be a proxy for a patient oriented outcome: it would (potentially) stand to reason that a better time to engraftment would reduce hospital length of stay.

Improved engraftment times are a better proxy than say, "better lab values that dont necessarily correlate with outcomes," but not as good as "improved engraftment times as well as reduced length of stay."

does that make more sense? GMDA could still be potentially useful in a portfolio and a potential buyout. I think improved myelablative therapy / pretreatments are going to be a hot item for *somebody*.
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