Mesoblast Limited, a global leader in allogeneic cellular medicines for inflammatory diseases, announced its operational update and financial results for the period ending June 30, 2024. The company highlighted significant progress in its clinical development programs, focusing on its key products, RYONCIL (remestemcel-L) and REVASCOR (rexlemestrocel-L).
RYONCIL (REMESTEMCEL-L) FOR ACUTE GRAFT VERSUS HOST DISEASE IN CHILDREN
Mesoblast resubmitted its Biologics License Application (BLA) for approval of RYONCIL on July 8, 2024, addressing outstanding concerns raised by the U.S. Food and Drug Administration (FDA) in a Complete Response Letter (CRL) issued in August 2023. The FDA has accepted the resubmission and is currently reviewing it. Mesoblast anticipates a decision from the FDA before or on the Prescription Drug User Fee Act (PDUFA) goal date of January 7, 2025. The company is concurrently implementing a comprehensive go-to-market strategy to make RYONCIL accessible to children suffering from steroid-refractory acute graft versus host disease (SR-aGVHD).
RYONCIL (REMESTEMCEL-L) FOR ACUTE GRAFT VERSUS HOST DISEASE IN ADULTS
Mesoblast plans to pursue a label extension for RYONCIL in adults after gaining pediatric approval. To facilitate this, the company is collaborating with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) to conduct a pivotal trial in adults with SR-aGVHD. This is crucial considering the low survival rate (20-30%) within 100 days for adults with SR-aGVHD who have failed other treatments.
REXLEMESTROCEL-L FOR CHRONIC LOW BACK PAIN ASSOCIATED WITH DEGENERATIVE DISC DISEASE
Mesoblast has commenced enrollment for a confirmatory Phase 3 trial of its second-generation allogeneic stromal cell product, rexlemestrocel-L, in patients with chronic low back pain (CLBP) caused by inflammatory degenerative disc disease (DDD). The FDA has previously approved the trial design, with a 12-month primary endpoint focused on pain reduction. This trial is especially significant as discogenic back pain accounts for a significant portion of prescription opioid usage in the U.S., and Mesoblast’s previous Phase 3 trial demonstrated substantial pain reduction and opioid cessation. The FDA has designated rexlemestrocel-L a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of CLBP, which provides expedited review and approval pathways.
REVASCOR (REXLEMESTROCEL-L) FOR PEDIATRIC CONGENITAL HEART DISEASE: HYPOPLASTIC LEFT HEART SYNDROME (HLHS)
REVASCOR has received both Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) from the FDA. These designations stem from the successful results of a randomized controlled trial in children with hypoplastic left heart syndrome (HLHS), a life-threatening congenital heart condition. The trial demonstrated that a single intramyocardial administration of REVASCOR at the time of staged surgery resulted in significantly larger increases in left ventricular (LV) end-systolic and end-diastolic volumes compared to controls over 12 months. This indicates clinically important growth of the small left ventricle, facilitating a successful surgical correction known as full biventricular (BiV) conversion, which allows for normal two-ventricle circulation. Mesoblast may be eligible for a Priority Review Voucher (PRV) upon FDA approval of a BLA for REVASCOR for the treatment of HLHS.
REVASCOR (REXLEMESTROCEL-L) FOR CHRONIC HEART FAILURE WITH REDUCED EJECTION FRACTION (HFrEF) AND PERSISTENT INFLAMMATION
Heart failure with low ejection fraction (HFrEF) affects approximately 50% of heart failure patients and carries high mortality risk. REVASCOR has shown promise in reducing major adverse cardiac events (MACE) in a completed Phase 3 trial in ischemic HFrEF patients. The company is pursuing an accelerated approval pathway for REVASCOR for patients with end-stage ischemic HFrEF and a left ventricular assist device (LVAD) based on data demonstrating reduced inflammation, strengthened left ventricular function, reduced right ventricular failure, and reduced hospitalizations in these patients.
FINANCIAL RESULTS
Mesoblast reported a cash balance of US$63.3 million at June 30, 2024, with an additional US$10.0 million available from an existing facility upon FDA approval of RYONCIL. The company achieved a 23% reduction in net cash usage for operating activities in FY2024 compared to FY2023, driven by reduced manufacturing activities and lowered payroll. Mesoblast remains committed to prudent cash management for operational activities as it prepares for the commercial launch of RYONCIL and related supply chain activities.
CONCLUSION
Mesoblast continues to make significant strides in its clinical development programs, with promising results for its key product candidates. The company’s focus on cost containment and prudent cash management positions it strategically for future growth and commercialization efforts.
