Mesoblast (NASDAQ:MESO) reported $115 million in net revenue for fiscal 2026, its first full year of commercial sales for RYONCIL, the company’s FDA-approved allogeneic mesenchymal stromal cell therapy for children with steroid-refractory acute graft-versus-host disease, or GVHD.
Chief Executive Officer Silviu Itescu said fourth-quarter net revenue reached $36 million, while gross profit excluding amortization expenses totaled $110 million for the full year. The company described RYONCIL as the first and only FDA-approved mesenchymal stromal cell product in the U.S.
RYONCIL Commercial Progress
Mesoblast said more than 50 treatment centers have been onboarded for RYONCIL, and insurance coverage now extends to more than 98% of U.S. lives. The company also received a J-code in October 2025, which Itescu said contributed to revenue growth.
In real-world use, Itescu said RYONCIL has shown 84% early survival among treated children with acute GVHD. He said the company’s priorities over the next year include increasing adoption in the pediatric market, encouraging earlier use of the therapy, and expanding into adult GVHD.
Mesoblast is enrolling a 180-patient randomized trial in adults with Grade 3 or 4 steroid-refractory acute GVHD. The study compares ruxolitinib alone with ruxolitinib plus RYONCIL. Itescu said the trial is enrolling across more than 40 U.S. sites and is expected to take about 18 months to complete.
The company expects an interim analysis in the fourth quarter of 2027 after approximately 100 patients, or 57% of the planned enrollment, have been enrolled. Itescu said either a successful interim analysis or a positive result from the full trial could support a supplemental biologics license application, or sBLA, for an adult label expansion.
Discussing patients who have failed ruxolitinib, Itescu cited compassionate-use results showing 76% survival at day 100 among adolescents and adults treated with RYONCIL, compared with survival of roughly 20% to 30% in reports involving patients treated with other third-line agents after ruxolitinib failure.
When asked about revenue expectations, Itescu did not provide formal guidance but said the company expects “double-digit growth” over the coming 12 months. He said management expects to have a clearer view of growth by the middle of the fiscal year.
Pipeline Advances in Duchenne and Back Pain
The FDA cleared Mesoblast’s investigational new drug application for a Phase III trial of RYONCIL in pediatric Duchenne muscular dystrophy. Itescu said the company is working with U.S. clinicians, patient groups and families to establish trial sites and begin the study.
He said Mesoblast selected Duchenne muscular dystrophy because it believes its cell therapy could address inflammation early in the disease process, including in children as young as 3 or 4. The company expects the treatment could potentially be used alongside gene therapy approaches, according to Itescu.
Mesoblast also completed treatment of all 350 patients in its pivotal Phase III trial of rexlemestrocel-L for chronic low back pain associated with degenerative disc disease. The company is following participants through 12 months and expects trial results in the second half of calendar 2027.
If successful, Mesoblast plans to file a BLA, with a potential approval timeline in calendar 2028. Itescu said the pivotal study is designed to replicate an earlier Phase III trial in which a single injection produced pain reduction beginning at six months, peaking at 12 months and persisting through at least 36 months.
The company said it is targeting patients with moderate-to-severe chronic low back pain who have not responded to medical therapies. Itescu said an outreach effort among pain specialists found that 85% were more likely to recommend rexlemestrocel-L if the prior clinical results were replicated in a commercial product.
Heart Failure Program and Financial Outlook
Mesoblast is also pursuing approval of REVASCOR, a rexlemestrocel-L program for advanced heart failure patients supported by left ventricular assist devices. Itescu said a prior randomized trial showed reductions in major gastrointestinal bleeding and hospitalizations related to right heart failure, along with improved survival among higher-risk ischemic patients.
The company plans to seek full FDA approval in patients at risk of right heart failure and life-threatening bleeding. Itescu said a successful approval could potentially support later expansion into the larger Class II and III heart failure population.
O’Brien said Mesoblast’s fiscal 2027 cash burn is expected to be lower than in fiscal 2026, supported by anticipated RYONCIL growth and cash receipts. He declined to provide a specific quarter in which the company expects to become cash-flow positive from operations.
Mesoblast has a $125 million credit facility carrying an 8% interest rate, with no principal amortization for five years, according to O’Brien. He said the company intends to maintain financial flexibility while funding Phase III programs, manufacturing capabilities, regulatory filings and inventory needed to meet patient demand.
About Mesoblast (NASDAQ:MESO)
Mesoblast Limited is a global leader in allogeneic cellular medicines, focused on developing treatments for inflammatory and immunologic diseases. Founded in 2004 by Dr. Silviu Itescu, the company builds on proprietary mesenchymal lineage cell technology to create off-the-shelf, donor-derived therapies. These therapies are designed to modulate immune responses and promote tissue repair in conditions where existing medical options are limited or ineffective.
The company’s most advanced product, Alofisel® (darvadstrocel), has been approved in Europe for the treatment of complex perianal fistulas in adults with Crohn’s disease.
