Mesoblast reported its fiscal 2025 results and, more importantly for the market, its first quarter of commercial sales for Ryoncil, the first FDA-approved mesenchymal stromal cell therapy in the United States, indicated for steroid-refractory acute graft-versus-host disease in children. Net product sales reached $11.3 million in the launch quarter, on $13.2 million in gross sales, with a 14.6% gross-to-net ratio, contributing to total cell therapy revenues of $17.2 million for the year. The company has onboarded 32 transplant centers and expects to reach the top 45 centers that conduct the most pediatric transplants by quarter-end. It has secured coverage for more than 250 million US lives and achieved mandatory fee-for-service Medicaid coverage, effective July 1, 2025. Cash on hand stood at $162 million, offset by a $102 million net loss and $50 million net operating cash spend.
This is more than a launch; it is a test of whether off‑the‑shelf MSCs can become a repeatable commercial category rather than a scientific curiosity. Early payer traction and center onboarding suggest a viable access pathway; however, the sales base remains small relative to burn, and hospital formulary adoption, site readiness, and utilization management will determine whether revenues scale. The strategic question is whether Mesoblast can convert a narrow pediatric foothold into a durable franchise before capital needs and evidence demands outpace momentum.
The stakes extend beyond one brand. For patients and transplant teams, a new option in pediatric steroid‑refractory aGVHD could shift survival expectations and treatment algorithms. For payers, the initial budget impact is modest in the rare pediatric population, but the calculus changes if adult label expansion proceeds; the target adult cohort is roughly three times larger than the pediatric population, and real-world data already highlight high non-response rates to second-line agents, such as ruxolitinib. For competitors, the 12-year biologic data exclusivity, which extends into 2036, and a deep IP wall through 2044 create real barriers to US entry, making category leadership attainable if manufacturing and potency assays remain aligned with FDA expectations.
The pipeline update highlights a broader industry shift toward de-risked regulatory pathways for novel cell therapies. Revascor for heart failure with reduced ejection fraction remains under RMAT, with FDA alignment on BLA components and a post-approval confirmatory trial design. This pragmatic path mirrors how several gene and regenerative programs have moved forward after receiving narrowly targeted, biomarker-enriched signals. In chronic low back pain, the 300‑patient confirmatory Phase 3 under RMAT aims to translate an earlier positive pain endpoint into an approvable claim, with particular resonance for opioid stewardship given the high opioid use in discogenic pain. If positive, this would place allogeneic cellular therapy at the center of a high-volume, payer-skeptical market, where durability, procedure logistics, and coding will dictate adoption as much as efficacy.
The following six to twelve months are critical for execution. Adult aGVHD development with the BMT-CTN could unlock a step-change in addressable demand that the commercial model needs. Ryoncil’s gross‑to‑net and cost of revenues will reveal whether inpatient administration and specialty distribution can support sustainable margins. RMAT-enabled milestones for Revascor and the back pain program will test whether Mesoblast can transform MSCs into a multi-indication platform business rather than a single-asset story. The key question for Commercial and Medical leaders across the sector: Are we witnessing the emergence of a scalable allogeneic cell therapy playbook, or a rare success that remains bound unless adult labels and payer evidence arrive on schedule?
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
