NEW YORK, USA / MELBOURNE, Australia, July 29–30, 2026
Mesoblast Limited reported strong commercial and clinical progress for the quarter ended June 30, 2026, highlighted by RYONCIL® (remestemcel-L-rknd) generating US$36 million in net revenue during the fourth quarter and US$115 million during its first full year following FDA approval. The company also advanced multiple late-stage development programs, including expansion of RYONCIL® into adult steroid-refractory acute graft-versus-host disease (SR-aGvHD) and other severe inflammatory conditions. In addition, Mesoblast reached its enrollment target of more than 300 patients in its pivotal Phase 3 trial evaluating rexlemestrocel-L for chronic low back pain (CLBP) associated with degenerative disc disease. The company ended the fiscal year with US$103 million in cash, supporting continued commercialization and regulatory development across its pipeline.
Ryoncil Commercial Growth Supports Expansion Into New Indications
Following its FDA approval for pediatric steroid-refractory acute graft-versus-host disease, RYONCIL® continues to gain adoption across major pediatric transplant centers in the United States. Building on this commercial momentum, Mesoblast has initiated a registrational trial to expand the therapy into adult SR-aGvHD, a market estimated to be three times larger than the current pediatric indication. Up to 40 clinical sites are expected to participate in the U.S. trial, representing approximately 60% of the annual adult allogeneic bone marrow transplant population. The company also received FDA Investigational New Drug (IND) clearance to begin a registrational trial evaluating RYONCIL® in children aged 5–9 years with Duchenne muscular dystrophy (DMD), further broadening the product’s potential use in life-threatening inflammatory diseases.
Late-Stage Pipeline Advances Across Pain and Cardiovascular Programs
Beyond RYONCIL®, Mesoblast achieved another major milestone by completing enrollment of more than 300 patients in the MSB-DR004 pivotal Phase 3 trial of rexlemestrocel-L for chronic low back pain caused by degenerative disc disease. Patients will now be followed through the study’s 12-month primary endpoint to evaluate pain reduction versus placebo. The company also received a Biologics License Application (BLA) filing number from the U.S. FDA for rexlemestrocel-L in preventing life-threatening gastrointestinal bleeding in left ventricular assist device (LVAD) patients with end-stage heart failure, while requesting a modular review to potentially accelerate regulatory assessment. These developments strengthen Mesoblast’s diversified late-stage regenerative medicine portfolio.
Advertisement
Mesoblast Expands Cell Therapy Innovation While Maintaining Strong Financial Position
During the quarter, Mesoblast also unveiled its next-generation mesenchymal stromal cell (MSC) platform, including an exclusive worldwide license for a patented CAR-MSC technology designed to enhance the precision and potency of future cell therapies. The technology is expected to support new treatments for ulcerative colitis, Crohn’s disease, lupus nephritis, and other autoimmune disorders. Financially, the company maintained a solid balance sheet with US$103 million in cash at June 30, 2026, after refinancing debt through a US$125 million five-year credit facility. Chief Executive Dr. Silviu Itescu stated that the strong first-year commercial performance of RYONCIL®, combined with advancing regulatory and clinical milestones, positions Mesoblast to pursue additional FDA approvals while supporting the long-term growth of its regenerative medicine portfolio.
Source: Mesoblast, press release
