The adult case presentation at Tandem will be exciting. The Aug 2024 slide deck had << Label extension strategy for Ryoncil In adult patients with SR-aGVHD >> survival in patients who fail Rux remains a dismal 20-30% by 100 days, and that these patients who fail Rux have no other approved therapies. In constrast the 100-day survival was 67% after Ryoncil was used in the EAP in 51 adults and children with SR-aGVHD who failed to respond to at least one additional agent, such as Rux. Meso were still talking about doing the adult trial with the BMT-CTN last Aug, but it is quite possible that it be run as a confirmatory trial post label extension in adults - i.e. the data presented at the Tandem meeting for the 25 adults in this EAP population looks to be compelling given that there is no alternative treatment for people failing Rux and that Ryoncil has now been approved, and the results are so good. Life is much easier with the FDA once you have the first approval. The FDA would probably insist that the trial in adults be started, before granting conditional approval, but that's not a big ask as we have BMT-CTN to run it for us and the trial design has already been done. The cost would probably be around $US5m - which is nothing post the recent $US160m CR.Expansion into adults could happen quite quickly.Joanne Kurtzberg will also be speaking at the event - she is inspiring, and now her trial has led to approval, and she is investigating other expansions of Ryoncil. Excitement!
E Cool.
Old EC is finding it hard to keep bagging SI out with the Share price upward movement. Hence he is changing Tact.
Good Post from him.
Aloha.
MSB Price at posting:
$3.17 Sentiment: Buy Disclosure: Held
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