@col69, many thanks for providing this transcription of this...

@col69, many thanks for providing this transcription of this significant conference call.

As others have said, the comment from Dr. Krause regarding the CRL#2 not questioning the efficacy of Ryoncil is critically important!

IMHO, SI's comment on 63% survival rates in adults and adolescents in the EAP, against a background survival rate of 20%, is also highly significant!! I had previously been concerned about the risk of reduced efficacy in an adult trial, particularly from the apparent relative difference in the results between children and adults in Protocol 280. However, this more recent EAP survival rate (and with n=71) is fantastic!! SI intimated that each arm of an adult trial may be in the order of 60-70 persons. If the EAP survival rates and control mortality rates are repeated in an adult trial with a commensurate primary end point, it should reach very high levels of statistical significance (see sample chi square analysis below with very low p-values). This may open up immediate access to the marketing approval for adults (~5x larger market) without requiring a secondary trial.

SI didn't directly answer the analyst question on the trial duration. However, his comments regarding the trial infrastructure being already in place, the EAP results providing excitement / incentive to enrol, the relatively small trial size and cost (within the cost containment measures for the next six months) all imply that this will likely be implemented in a period closer to six months as opposed to years.

It will be interesting to watch how markets digest this information in coming days and weeks, let alone the next conference call to debrief on the Type A meeting.