MSB Trading 2020 - a new dawn, page-11993

Hey Team,

Here are some stats on the probability of success (PoS) for phase III trails between 2006 and 2015:



https://www.bio.org/sites/default/files/legacy/bioorg/docs/Clinical%20Development%20Success%20Rates%202006-2015%20-%20BIO,%20Biomedtracker,%20Amplion%202016.pdf

And here is a distribution of those failures:



https://www.parexel.com/application/files_previous/5014/7274/5573/ACT_Article.pdf

I'd probably group CLBP under metabolic but I will let you pick what you want to group it under.

Keeping in mind a few things

- We know Remestemcel-L has a clean safety profile (as do almost all MSC therapies to date), so failure due to safety is extremely unlikely.
- We have passed two futility checks for Covid-19 ARDS, so there seems like there is some kind of efficacy signal
- We can also be pretty confident to rule out commercial failures due to the fact there is a significant unmet medical need and a poor standard of care

Collectively, this boosts the success stats a bit as failures due to efficacy are <60%. As such, it wouldn't be outrageous to half the failure rates demonstrated in the above table. The only real thing that could screw us for Covid-19 ARDS is the sample size, will we see efficacy by the final readout or will placebo do well/ our cells be insufficient to achieve significance.

Plenty of things give us a lot of confidence regarding efficacy, such as:

- The EAP with 10/12 survival.
- The UAE stem cell studies
- Athersys phase II data
- Similar pathophysiology to GvHD where I am satisfied that we have demonstrated efficacy.
- Two passes for futility checks.

However it's not all rosey, a big factor that reduces the PoS relative to the above figures is that:

- There has never been a commercially approved stem cell therapy

3000 deaths a day is a pretty damned good motivator to fix that though right?