Growing Confidence on COVID-19 Application, page-11

Yes very good point, this and the small subset population/trial size. If MSB were going for an BLA there is no way the data would be sufficient.

The product treatment strength, safety profile and data does However, in my opinion meet all the requirements for an EUA. There are 4 main criteria the FDA have for issuing an EUA:

1. Serious or Life-Threatening Disease or Condition

• Covid-19 ARDS has a mortality rate of ~42%+ with current SOC and increases with age.

2. Evidence of Effectiveness
Medical products that may be considered for an EUA are those that "may be effective"

• The results from MSB's P3 trial show significant treatment efficacy of 77% reduction in 90 day mortality with p=0.0037
• The small patient sub-set is the only weakness but this still shows that the product "may be effective" and provides significantly stronger evidence of efficacy compared to many EUA's issued

3. Risk-Benefit Analysis
A product may be considered for an EUA if the Commissioner determines that the known and potential benefits of the product, when used to diagnose, prevent, or treat the identified disease or condition, outweigh the known and potential risks of the product.

• Remestemcel-L has had no safety concerns with an impeccable safety profile similar to placebo in multiple p2 and p3 trials. So little to no side effects (potential risks) vs 77% reduction in 90 day mortality (known/potential benefits).

4. No Alternatives

• The only other treatment that has reduced mortality in COVID-19 ARDS is dexamethasone. However mortality rates still remain at 42%+. Remestemcel-L + dexamethasone appear to compliment each other providing significant mortality benefit never seen before in ARDS patients.

Given the recent Biogen Accelerated Approval for Alzheimer's (approved based on a subset trial population with a small effect size), I would also say there is a small chance of an AA for Rem-l, however I see this as unlikely.

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/emergency-use-authorization-medical-products-and-related-authorities