Mesoblast Maintains Momentum With FDA on Accelerated Approval Pathway for Revascor ® in Ischemic Heart Failure and Label Extension for Ryoncil ® in Adults With GvHD

Mesoblast provided an update on its interactions with the FDA regarding its two lead products, Revascor and Ryoncil. The company held a Type B meeting with the FDA regarding the BLA filing for Revascor for ischemic chronic heart failure, achieving general alignment on key aspects. Additionally, Mesoblast is scheduled to meet with the FDA in early July to discuss a pivotal trial for Ryoncil in adults with steroid refractory acute graft versus host disease, aiming to expand its label. Ryoncil's commercial launch is exceeding expectations, with significant hospital onboarding and payer coverage.

Analysis

Mesoblast (MESO) has reported significant positive momentum in its interactions with the U.S. Food and Drug Administration (FDA) for its key therapeutic candidates. Following a Type B meeting in early June under its Regenerative Medicines Advanced Therapy (RMAT) designation for Revascor® (rexlemestrocel-L), intended for ischemic chronic heart failure with reduced ejection fraction (HFrEF) and inflammation, Mesoblast indicated general alignment with the FDA on crucial aspects for a potential Biologics License Application (BLA), including chemistry, manufacturing & controls (CMC), potency assays for commercial product release, and the proposed design and primary endpoint for the confirmatory trial post-approval. The Company will await the final minutes from FDA for detailed feedback and timelines for a potential filing. Concurrently, Mesoblast is advancing Ryoncil® (remestemcel-L-rknd), with an FDA meeting scheduled in early July to discuss a pivotal trial in adults with steroid-refractory acute graft versus host disease (SR-aGvHD), aiming to extend its current pediatric label. This trial will be conducted with the NIH-funded Bone Marrow Transplant Clinical Trials Network (BMT-CTN). The commercial launch of Ryoncil®, which became available for purchase in the United States on March 28, 2025, for children with SR-aGvHD, is exceeding the company’s expectations. More than 20 transplant centers are expected to be onboarded by the end of the current quarter, surpassing launch targets, and payer coverage has expanded to over 220 million US lives. This includes 37 of 51 States providing fee-for-service Medicaid coverage, with the remainder, covering an additional 24 million lives, anticipated to provide mandatory coverage by July 1, 2025. Mesoblast Chief Executive Dr. Silviu Itescu highlighted the encouraging momentum with the FDA and the strength of the Ryoncil® commercial launch, noting that hospital onboarding, physician adoption, and payer coverage have exceeded expectations in the ten weeks since launch. An update on Ryoncil® sales is scheduled for the quarterly activities report at the end of July. Mesoblast's intellectual property portfolio, comprising over 1,000 granted patents or patent applications, is stated to provide commercial protection extending through at least 2041.

Key Decisions for Investors

• Investors should monitor upcoming FDA communications, specifically the final minutes for Revascor and the outcome of the July meeting for Ryoncil, as these are critical near-term catalysts for Mesoblast.
• The reported outperformance of Ryoncil's commercial launch, evidenced by exceeding expectations in hospital onboarding (over 20 centers by quarter-end) and securing broad payer coverage (over 220 million US lives), signals positive early market adoption and potential revenue impact, with sales figures anticipated at the end of July.
• While the company highlights a strong IP position with protection through at least 2041, continued successful execution in clinical development, securing regulatory approvals, and effectively scaling commercial operations remain paramount for sustained value creation and de-risking future prospects.