Ann: 3 Articles on RYONCIL GvHD Trial Results Published in BBMT, page-29

To summarise the results:

- All three trials showed that RYONCIL (remestemcel-L) demonstrated safety and efficacy in patients with the greatest level of inflammation and most severe GVHD.

- Study 275 used RYONCIL as a salvage treatment for 241 children exhibiting acute GVHD who failed to respond to steroids and a range of other treatments, with 67% overall survival at Day 100. This is compared to 10% survival in severe forms of acute GVHD cases with standard care.

- Study GVHD001/002 used RYONCIL as a first line treatment for 55 children exhibiting acute GVHD who failed to respond to steroid treatments, with 74.1% survival at Day 100 and 68.5% overall survival at Day 180. This is compared to 57% survival rate at Day 100 for a matched placebo group from a Mount Sinai database with standard care.

- Study 280 used RYONCIL as a second line treatment for 232 adults and 28 children with steroid-refractory acute GVHD who failed to respond to steroid treatment. At Day 28 overall survival rate of 57% compared to placebo at 37%. At Day 28 overall survival rate for children was 64% compared to placebo at 36%. Adults did not respond as well as children to the treatment.

Conclusion: RYONCIL works well for children and high risk adults with acute steroid-refractory GVHD, but there was no significant benefit of using RYONCIL in standard risk patients. There is currently no FDA approved treatment for children with steroid-refractory acute GVHD in the USA.

Dr Jacques Galipeau concluded that “after more than a decade of clinical study involving three distinctadvanced trials, it appears that remestemcel-L might well have finally met theregulatory requirements for marketing approval in the United States forsteroid-refractory acute GVHD in children”.