Core Viewpoint - Mesoblast Limited has reported strong initial sales of its FDA-approved product Ryoncil, generating gross revenue of US$13.2 million from March 28 to June 30, 2025, following its launch for treating steroid-refractory acute graft-versus-host disease in pediatric patients [1][6][10]. Financial Highlights - Gross revenue from Ryoncil sales reached US$13.2 million (unaudited) from March 28 to June 30, 2025 [6]. - The company reported US$1.6 million in revenue from royalties on sales of TEMCELL HS Inj. in Japan [6]. - Net operating cash spend for the quarter was US$16.6 million [6]. - Cash on hand as of June 30, 2025, was US$162 million (A$247 million) [6]. Operational Highlights - More than 25 transplant centers have been onboarded since the product launch, with plans to complete onboarding across all 45 priority centers that account for approximately 80% of U.S. pediatric transplants [6]. - Ryoncil's coverage is expanding, with over 250 million U.S. lives insured by commercial and government payers, and mandatory Medicaid coverage effective July 1 [6]. - A patient access hub, MyMesoblast™, has been established to assist patients with insurance coverage and access to Ryoncil [6]. Regulatory and Exclusivity Status - Ryoncil received seven years of orphan-drug exclusive approval from the FDA for treating SR-aGvHD in pediatric patients aged 2 months and older [6]. - Mesoblast holds biologic exclusivity preventing another sponsor from referencing Ryoncil's biologic license application until December 2036 [6]. - The company has a strong U.S. intellectual property position on MSC technology, providing commercial barriers to entry against competitors through 2044 [6][12]. Future Developments - Mesoblast held a Type B meeting with the FDA to discuss a pivotal trial for Ryoncil in adults with SR-aGvHD, aiming to extend the product's label [6].

Core Insights - Mesoblast is making progress with the FDA regarding the accelerated approval pathway for Revascor and label extension for Ryoncil [1][2][3] Group 1: FDA Interactions and Approvals - A Type B meeting was held with the FDA to discuss the potential filing for a Biologics License Application (BLA) for Revascor, with alignment on key components [2] - An upcoming meeting with the FDA is scheduled to discuss a pivotal trial of Ryoncil in adults with steroid refractory acute graft versus host disease (SR-aGvHD) [3] Group 2: Commercial Launch and Market Coverage - Ryoncil became commercially available in the U.S. on March 28, 2025, with over 20 transplant centers onboarded by the end of the quarter, exceeding expectations [4] - The company has expanded Ryoncil's coverage to over 220 million U.S. lives insured, with 37 of 51 states providing Medicaid coverage [4] Group 3: Company Overview and Future Developments - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing proprietary mesenchymal lineage cell therapy technology [6][8] - The company is committed to developing additional cell therapies for various indications, including inflammatory bowel disease and chronic low back pain [8]

Core Insights - Mesoblast Limited has successfully launched Ryoncil (remestemcel-L) for treating children with acute graft versus host disease (GVHD) shortly after receiving FDA approval, marking it as the first approved mesenchymal stromal cell therapy in the US [2][11] - The company is well-positioned financially, with a cash reserve of US$182 million (A$290 million), allowing for the expansion of Ryoncil's indications to other serious pediatric inflammatory diseases and adult acute GVHD [5][12] - Mesoblast is also preparing for a Type B meeting with the FDA to discuss the accelerated approval pathway for Revascor (rexlemestrocel-L) in treating ischemic chronic heart failure with reduced ejection fraction [5][12] Financial Highlights - The net operating cash spend for the quarter was US$12.7 million [5] - Cash on hand at the end of the quarter was US$182 million (A$290 million) [5] - Mesoblast raised A$260 million (US$161 million) through a global private placement primarily to existing major shareholders [12] Operational Highlights - Ryoncil became commercially available in the US on March 28, 2025, with Federal Medicaid coverage, and 15 infusion kits have been purchased for patient treatment [5] - The company has onboarded ten priority transplant centers, with plans to onboard an additional ten in the current quarter [5] - Mesoblast has expanded Ryoncil coverage to over 104 million US lives insured by commercial and government payers [5] Product Development - Ryoncil is being developed for additional indications, including adult SR-aGvHD and biologic-resistant inflammatory bowel disease [13] - Revascor is under development for chronic heart failure and has received RMAT designation from the FDA [12] - The confirmatory Phase 3 trial for rexlemestrocel-L in chronic low back pain is actively enrolling patients, with a focus on opioid cessation [12] Corporate Developments - Mesoblast has strengthened its Board of Directors with the appointment of Dr. Gregory George and Ms. Lyn Cobley [12] - The company was added to the S&P/ASX 200 Index effective March 6, 2025 [12] Intellectual Property - Mesoblast holds a strong global intellectual property portfolio with over 1,000 granted patents or applications covering its mesenchymal stromal cell technology [14] Manufacturing Capabilities - The company utilizes proprietary manufacturing processes to produce industrial-scale, cryopreserved, off-the-shelf cellular medicines [15] Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing its proprietary mesenchymal lineage cell therapy technology [10]

Core Insights - Mesoblast has expanded coverage for its product Ryoncil® (remestemcel-L) to 104 million insured lives in the U.S. through government and commercial payers [1][2] - Ryoncil® is the first FDA-approved mesenchymal stromal cell therapy for steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients aged 2 months and older [4] Coverage Expansion - 37 out of 51 states provide fee-for-service Medicaid coverage for Ryoncil®, covering 20 million lives, which is 80% of total Medicaid fee-for-service lives [2] - Mandatory coverage for an additional 24 million lives will begin on July 1, 2025 [2] - Commercial plans cover 84 million lives, excluding medical exceptions policies, indicating that the actual commercial coverage is likely higher [2] Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing a proprietary mesenchymal lineage cell therapy technology platform [3] - The company is also developing additional therapies for other inflammatory diseases and conditions such as heart failure and chronic low back pain [5] Intellectual Property - Mesoblast holds a strong global intellectual property portfolio with over 1,000 granted patents or applications, providing commercial protection expected to last until at least 2041 in major markets [6] Manufacturing Capabilities - The company has proprietary manufacturing processes that yield industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability to patients worldwide [7]