Ann: Update on BLA for Graft Versus Host Disease, page-237

Hard pill to swallow, but manufacturing box is now ticked with efficacy outsatnding despite a 9-1 ODAC vote and new analysis + data submitted.

As I see it, next steps are :

1. Meet with the FDA in 45 days (Type A meeting), pushing for accelerated approval on pediatric (FDA could recommend that MSB submit a BLA for accelerated approval, which could be lodged prior to the start / completion of the adult study (but would be ancilliary to the accelerated approva BLA))
2. Restructure debt + sort out funding
3. Minutes from FDA Type A meeting received to confirm next steps
4. Adult trial likely to be funded as a research study (no cost to MSB)
5. CLBP US partnership may be sooner rather than later, but unlikely to commence until clarity is obtained from the above (or partner is announced)
6. CHF meeting with FDA
7. COVID trial may commence with CMC issues resolved from Ryoncil BLA

Once the adult trial is completed, MSB could well have access to both adult and children .. and with this outcome today, IMO this will be priority #1.

Goodluck all