Appendix 4C Quarterly Activity Report for Quarter Ended December 31, 2024

NEW YORK, Jan. 30, 2025 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided highlights of its recent activities for the second quarter ended December 31, 2024.

ACTIVITY REPORT         

• On December 18, 2024, Ryoncil (remestemcel-L) became the first mesenchymal stromal cell (MSC) therapy approved by U.S. FDA for any indication.
• FDA approved Ryoncil as the first and only therapy for children aged 2 months and older, including adolescents and teenagers, with steroid-refractory acute graft versus host disease (SR-aGvHD), a life-threatening condition with high mortality rates.
• Commercial inventory has been manufactured and a distribution network has been established using Cencora, a leader in specialty pharmaceutical services and distribution. Cencora will leverage its cryogenic logistics capabilities and state-of-the art cryogenic storage infrastructure to enable the efficient and secure delivery of cryopreserved product to U.S. treatment centers.
• The confirmatory Phase 3 trial of rexlemestrocel-L in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration is actively enrolling and treating patients at multiple sites across the United States; the capital raise concluded this month will facilitate expansion of sites enrolling in the trial and acceleration of patient accrual.
• Under its Regenerative Medicine Advanced Therapy (RMAT) designation Mesoblast intends to meet with FDA to discuss data presentation, timing and FDA expectations for an accelerated approval filing in end-stage heart failure patients.
• In November 2024 a publication in the prestigious peer-reviewed European Journal of Heart Failure (EJHF), reported that a single intramyocardial injection of Revascor (rexlemestrocel-L), Mesoblast’s second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell therapy, results in improved survival in high-risk NYHA Class II/III patients with ischemic heart failure and inflammation.¹ This identifies the HFrEF population that is responsive to REVASCOR and will be the target of a confirmatory trial after accelerated approval, if received.
• FDA granted REVASCOR RMAT designation following submission of results from the randomized controlled trial in children with hypoplastic left heart syndrome (HLHS), a potentially life-threatening congenital heart condition.