NEW YORK, Aug. 28, 2025 (GLOBE NEWSWIRE) -- Mesoblast Limited (ASX:MSB; Nasdaq:MESO), global leader in allogeneic cellular medicines for inflammatory diseases, today provided financial results and an operational update for the period ended June 30, 2025.

Mesoblast Chief Executive Dr. Silviu Itescu said, "This has been a banner year for the Company, with achievement of FDA approval for Ryoncil$(R)$ , the first and only FDA approved mesenchymal stromal cell $(MSC)$ product in the United States and a successful commercial launch of the product for treatment of steroid-refractory acute graft-versus-host disease (SR-aGvHD) in pediatric patients aged 2 months and older ("children")."

"We are working tirelessly to transform Mesoblast into a fast-paced commercial biotechnology company with demonstrable sales of our first commercial product Ryoncil(R) . We intend to deliver additional approved indications for sales of Ryoncil(R) and to launch a next-generation platform technology into potential blockbuster indications including heart failure and chronic low back pain."

FINANCIAL AND CORPORATE HIGHLIGHTS FOR THE YEAR ENDED JUNE 30, 2025

   -- Revenue from cell therapy products was US$17.2 million, up 191% on prior 
      year. 
   -- Revenue growth was driven by successful launch of Ryoncil(R) in the final 
      quarter, with US$13.2 million gross sales and US$11.3 million in reported 
      net sales after 14.6% gross to net adjustment. 
   -- Net operating cash spend was US$50.0 million, a 3% increase on prior year, 
      inclusive of costs related to commercial team build and product launch. 
   -- Cash on hand at June 30, 2025, was US$162 million (A$247 million)1. 
   -- Mesoblast was added to the S&P Dow Jones Indices' S&P/ASX 200 Index 
      effective March 6, 2025, on the Australian Stock Exchange (ASX), and to 
      the Nasdaq Biotechnology Index (Nasdaq: NBI) as part of the annual 
      reconstitution of the 2024 Nasdaq index December 23, 2024. 
   -- Board of Directors was strengthened with appointments of Dr. Gregory 
      George MD PhD and Ms Lyn Cobley. 

INVESTMENT HIGHLIGHTS

Mission

Mesoblast is committed to bring to market innovative off-the-shelf allogeneic cellular medicines to treat serious and life-threatening inflammatory illnesses.

Product Portfolio

   -- Ryoncil(R) the only FDA-approved MSC therapy for any indication; 
      lifesaving for pediatric SR-aGvHD. 
 
   -- Revascor(R) has potential for FDA accelerated approval in end-stage 
      HFrEF. 
 
   -- Rexlemestrocel-L in Phase 3 trial for potential approval in CLBP. 
 
   -- Additional pipeline therapies targeting unmet medical needs. 

Market Opportunity

   -- Steroid-refractory acute GvHD $1 billion annual addressable market for 
      children & adults. 
 
   -- Biologic-refractory inflammatory bowel disease >US$5 billion. 
 
   -- Heart failure with reduced ejection fraction (HFrEF) >$10 billion 
      addressable market. 
 
   -- Chronic low back pain (CLBP) >$10 billion addressable market. 
 
   -- Additional potential multi-billion-dollar opportunities from existing and 
      future product pipeline based on existing technology platforms. 

Competitive Advantage

   -- Proven scientific approach, with deep understanding of mechanism of 
      action (MOA). 
 
   -- Robust and extensive intellectual property. 
 
   -- Extensive positive clinical trial results across multiple indications. 
 
   -- Demonstrated ability to meet regulatory requirements of FDA. 
 
   -- Scalability of manufacturing processes and proprietary next generation 
      technology. 

Financials and Projections

   -- Strong balance sheet to support Ryoncil(R) launch and product portfolio. 
 
   -- Current revenue streams anticipated to expand based on Ryoncil(R) 
      performance. 
 
   -- Continued prudent cash management strategy. 
 
   -- Long-term revenue potential from the existing and future pipeline. 

Upcoming Milestones

   -- Commence Ryoncil(R) registration trial for label expansion in adults with 
      severe SR-aGvHD. 
 
   -- Commence Ryoncil(R) registration trial for label expansion in adults and 
      children with inflammatory colitis. 
 
   -- Revascor(R) BLA submission for accelerated approval. 
 
   -- Rexlemestrocel-L CLBP Phase 3 completion and BLA submission. 
 
   -- Expansion into new indications and markets. 

OPERATIONAL HIGHLIGHTS

Ryoncil(R) (remestemcel-L-rknd) for Steroid-Refractory Acute Graft Versus Host Disease (SR-aGvHD) in Children

In December 2024, Ryoncil(R) became the first and only Food and Drug Administration (FDA) approved mesenchymal stromal cell (MSC) product in the Unites States. Ryoncil(R) became commercially available for purchase on March 28, 2025, within one quarter of receiving FDA approval for treatment of SR-aGvHD in children.

Coverage for Ryoncil(R) continues to expand with over 250 million US lives insured by commercial and government payers. Federal Medicaid coverage by Centers for Medicare and Medicaid $(CMS)$ is in place and mandatory fee-for-service Medicaid coverage for Ryoncil(R) became effective July 1, 2025 in all US states.

To date, we have onboarded 32 transplant centers and aim to have onboarded by the end of this quarter the top 45 centers that account for 80% of pediatric bone marrow transplants in the United States.

Assistance to Patients with SR-aGvHD and their Caregivers for Access to Ryoncil(R)

To assist patients and institutions with insurance coverage, financial assistance, and access programs, ensuring that no patient is left behind in receiving this potentially life-saving therapy, Mesoblast has established a patient access hub termed MyMesoblast$(TM)$, where Ryoncil(R) is available for ordering. Additional information is available on ryoncil.com, where valuable resources for healthcare providers, patients and caregivers can be found.

Ryoncil(R) Lifecycle Extension in Adults with Severe SR-aGvHD

Most of the sites already onboarded for use of Ryoncil(R) in children with SR-aGvHD also perform adult bone marrow transplants. We have an active compassionate care program to provide Ryoncil(R) to adults with SR-aGvHD who have failed other therapies.

Adults with severe SR-aGvHD have a high rate of non-responsiveness to second-line agents such as ruxolitinib. Survival in those with SR-aGvHD who have failed at least one additional agent, such as ruxolitinib, remains as low as 20-30% by 100 days.(2) (,) (3) In contrast, among 25 patients aged 12 and older with SR-aGvHD who failed ruxolitinib or other second-line agents, Day-100 survival was 76% after Ryoncil(R) treatment was used under expanded access. On the basis of these results, Mesoblast intends to seek label extension for Ryoncil(R) in adults with severe SR-aGvHD.

Mesoblast recently met with FDA to discuss a pivotal trial for Ryoncil(R) in adults with severe SR-aGvHD. Given the continued unmet need in adults with severe SR-aGvHD, Mesoblast intends to conduct a pivotal study of Ryoncil(R) on top of approved second-line therapy in patients with severe SR-aGvHD.

This trial will be conducted with the NIH-funded Bone Marrow Transplant Clinical Trials Network (BMT-CTN), the objective being to extend Ryoncil's(R) label from children to adults with SR-aGvHD, a population approximately three times the size of the pediatric SR-aGvHD population.

Ryoncil(R) Lifecycle Extension in Inflammatory Diseases

Inflammatory bowel disease $(IBD)$, including ulcerative colitis (UC) and Crohn's disease $(CD)$ remains a major unmet need across the adult and pediatric population where early and durable remission remains especially challenging.

According to recent estimates, more than three million people (1.3%) in the US alone have inflammatory bowel disease, with more than 38,000 new cases of ulcerative colitis and 33,000 new cases of Crohn's disease diagnosed every year.(4-6) Despite recent advances, approximately 30% of patients are primarily unresponsive to anti-TNF<ALPHA> agents and even among responders, up to 10% will lose their response to the drug every year(7,8) . Up to 80% of patients with medically-refractory Crohn's disease and 20% of patients with medically-refractory ulcerative colitis eventually require surgical treatment of their disease,(7,8) which can have a devastating impact on quality of life.

A recent pilot study in adults with inflammatory colitis demonstrated positive outcomes (rapid mucosal healing and disease remission) in biologic refractory patients receiving Ryoncil(R) by direct endoscopic injection to areas of inflammation.(9) (,10) This extends earlier Mesoblast data showing that intravenously delivered remestemcel-L can induce early remission in CD adults who have failed an anti-TNF agent.

Given the effectiveness of Ryoncil(R) in GI-related SR-aGvHD, and the existing data on adult inflammatory colitis, Mesoblast plans to initiate a pivotal study of Ryoncil(R) for early remission in patients with medically-refractory inflammatory colitis.

Ryoncil(R) Market Exclusivity

Ryoncil(R) received seven years of orphan-drug exclusive approval from FDA for treatment of SR-aGvHD in pediatric patients 2 months of age and older, meaning that the FDA will not approve another MSC product for this indication during the 7-year exclusivity period.

Mesoblast also has biologic exclusivity preventing another sponsor from referencing the Ryoncil(R) biologic license application (BLA) until December 2036, preventing market entry by a biosimilar. Mesoblast's strong U.S. intellectual property position on MSC composition of matter, manufacturing and indications, including SR-aGvHD, provide even further commercial barriers to entry against competitors through 2044.

Revascor(R) (rexlemestrocel-L) for Chronic Heart Failure with Reduced Ejection Fraction (HFrEF) and Persistent Inflammation

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