Appendix 4C Quarterly Activity Report for Quarter Ended December 31, 2023

        

NEW YORK, Jan. 30, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an activity report for the second quarter ended December 31, 2023.

Mesoblast Chief Executive Silviu Itescu said: “It has been a very busy quarter in which we have made substantial operational progress across our three lead Phase 3 assets. We have generated significant new potency and characterization data for our lead product Ryoncil (remestemcel-L) for children with acute GVHD, as requested by FDA, and will submit these data ahead of our planned meeting with FDA this quarter.”

“Our second Phase 3 back pain trial with rexlemestrocel-L, aiming to confirm the durable pain reduction that was seen in the first Phase 3 trial, is underway. Finally, we were very pleased to have received a Rare Pediatric Disease (RPD) Designation from FDA for our cardiovascular product Revascor in children with life-threatening congenital heart disease, and plan to discuss the trial results in the context of a regulatory approval pathway.”

Dr Itescu added: “We raised additional capital during the quarter to support these important Phase 3 programs, and I would like to thank all shareholders that participated in the placement and entitlement offer. In combination with our previously announced cost reduction strategies and operational streamlining, which are on-track, this new capital will provide added balance sheet strength.”

ACTIVITY REPORT

Graft versus Host Disease – Pediatric and Adult Phase 3 Programs

• Mesoblast has requested a meeting with FDA this quarter to provide additional potency and characterization data for its product RYONCIL which it believes demonstrate that the product used as second-line after corticosteroids in the pivotal Phase 3 trial GVHD001 in children with SR-aGVHD, which successfully met its primary endpoint of Day 28 Overall Response, was made to a standard supporting the trial as being adequate and well controlled.
• The new potency assay data show that the RYONCIL product made with the current manufacturing process that has undergone successful inspection by FDA, demonstrates greater potency than the earlier generation product, providing context to its greater impact on survival.
• Showing that the product used in the completed pediatric Phase 3 trial was standardized as to potency and characterization could provide support for approval of the pediatric indication given the absence of any approved therapies for children.
• Survival in adults with SR-aGVHD who have failed at least one additional agent, such as ruxolitinib, remains as low as 20-30% by 100 days.^1,2 In contrast, 100-day survival was 63% after remestemcel-L treatment was used under expanded access in 71 patients aged 12 and older with SR-aGVHD who failed to respond to at least one additional agent, such as ruxolitinib.
• The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) in the United States, a body that is funded by the National Institutes of Health (NIH) and is responsible for approximately 80% of all US allogeneic BMTs, has agreed to develop and execute a pivotal trial of RYONCIL in adults who are refractory to both corticosteroids and a second line agent such as ruxolitinib.
• Mesoblast will provide the Phase 3 trial protocol to FDA ahead of the upcoming meeting this quarter.

Cardiovascular – Program in Pediatric Congenital Heart Disease, Adult Phase 3 Program in Chronic Heart Failure with Reduced Ejection Fraction (HFrEF)