Mesoblast (MESO)2025-02-27 23:45Financial Performance - Cash balance as of December 31, 2024, was US61 million), with pro-forma cash approximately US322 million) after a global private placement raising US400 million) [20] - Total revenue for the half year ended December 31, 2024, was US3.4 million in the same period of 2023 [21] - Loss after tax for H1 FY2025 was US32.5 million in H1 FY2024, indicating a 47% increase in losses [24] - Net operating cash spend was US20.6 million in H1 FY2025 from US23.0 million reversal of inventory provision, now recognized as an asset on the balance sheet [20] - RYONCIL is the first FDA-approved off-the-shelf cell therapy for pediatric patients aged 2 months and older with steroid-refractory acute graft versus host disease (SR-aGvHD) [28] - RYONCIL delivered an overall response rate of 95% at Day 28 in a Phase 3 study, indicating strong efficacy in treating SR-aGvHD [32] - The wholesale acquisition cost (WAC) of RYONCIL is US$194,000 per intravenous infusion, regardless of body weight [45] Market Needs and Opportunities - There is a significant unmet need in pediatric inflammatory bowel disease (IBD), with over 60% of adult and pediatric Crohn's disease patients unable to achieve remission on anti-TNF agents [53] - The annual pediatric incidence of Crohn's disease in the U.S. is approximately 7,000 children, with a prevalence of 60,000-80,000 [54] - Chronic low back pain (CLBP) affects over 7 million patients in the U.S. and Europe, with minimal treatment options available for those refractory to conservative therapy [63] - Heart failure with low ejection fraction (HFrEF) affects approximately 6.5 million patients in the U.S., with a high mortality rate of about 50% at five years [70] Future Plans and Designations - Mesoblast expects to advance its multiple product pipeline toward FDA approvals over the next six to twelve months [82] - The FDA has granted REVASCOR Rare Pediatric Disease Designation and Regenerative Medicine Advanced Therapy designation, indicating its potential for treating serious conditions in children [80] - Ryoncil® will be available for use in steroid-refractory acute graft-versus-host disease (SR-aGvHD) at U.S. hospitals [82] - Studies for inflammatory diseases will commence for both pediatric and adult label extensions [82] - The company is preparing for accelerated approval filing for heart failure treatments [82] Intellectual Property - The company has an extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets [9]