Core Insights - Mesoblast Limited will host its inaugural R&D Day on April 8, 2026, in New York City, featuring presentations from senior leadership and key opinion leaders [1][2] - The event will discuss the company's corporate strategy, the commercialization of its flagship product Ryoncil, and its multi-billion-dollar pipeline opportunities in inflammatory pain and cardiovascular disease [2][3] Company Overview - Mesoblast is a global leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing a proprietary mesenchymal lineage cell therapy technology platform [3][5] - The company has a strong intellectual property portfolio with over 1,000 granted patents or applications, providing commercial protection until at least 2044 in major markets [6] Product Information - Ryoncil (remestemcel-L-rknd) is the first FDA-approved mesenchymal stromal cell therapy for treating steroid-refractory acute graft versus host disease in pediatric patients aged 2 months and older [4] - The company is developing additional therapies based on its technology platforms, targeting conditions such as heart failure and chronic low back pain [5] Manufacturing and Distribution - Mesoblast's proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf cellular medicines, which are planned to be readily available to patients worldwide [7] - The company has established commercial partnerships in Japan, Europe, and China to enhance its market reach [5]

Core Insights - Mesoblast Limited has appointed Teresa Montagut MD, PhD as the Head of Clinical Development and Medical Affairs to enhance its medical affairs organization and clinical collaborations [1][4] - Teresa Montagut brings extensive experience in medical leadership and pharmaceutical development, having previously worked at Regeneron and led cancer immunotherapy programs at major pharmaceutical companies [2][3] Company Overview - Mesoblast is a global leader in developing allogeneic cellular medicines for severe inflammatory diseases, utilizing a proprietary mesenchymal lineage cell therapy technology platform [5] - The company’s FDA-approved product, Ryoncil, is the first mesenchymal stromal cell therapy for treating steroid-refractory acute graft versus host disease in pediatric patients aged 2 months and older [6] - Mesoblast is developing additional therapies for various inflammatory diseases and has established commercial partnerships in Japan, Europe, and China [7] Intellectual Property and Manufacturing - Mesoblast holds a strong global intellectual property portfolio with over 1,000 granted patents or applications, providing commercial protection until at least 2044 in major markets [8] - The company’s proprietary manufacturing processes enable the production of industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability to patients worldwide [9]

Core Insights - Mesoblast Limited has appointed Teresa Montagut MD, PhD as the Head of Clinical Development and Medical Affairs, a newly established position aimed at enhancing clinical collaborations and advancing cell therapy programs for inflammatory diseases [1][4] Company Overview - Mesoblast is a global leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing a proprietary mesenchymal lineage cell therapy technology platform [5] - The company’s lead product, Ryoncil® (remestemcel-L-rknd), is the first FDA-approved mesenchymal stromal cell therapy for treating steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients aged 2 months and older [6] Leadership and Expertise - Teresa Montagut brings extensive experience in medical leadership and pharmaceutical development, having previously held significant roles at Regeneron and other major pharmaceutical companies [2][3] - Her expertise in executing investigator-initiated clinical trials is expected to be crucial for expanding the indications of Ryoncil® and advancing Mesoblast's pipeline of cellular therapies [4] Product Development and Pipeline - Mesoblast is committed to developing additional cell therapies for various indications, including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease, as well as rexlemestrocel-L for heart failure and chronic low back pain [7] - The company has established commercial partnerships in key markets including Japan, Europe, and China [7] Intellectual Property and Manufacturing - Mesoblast holds a strong global intellectual property portfolio with over 1,000 granted patents or applications, providing commercial protection until at least 2044 in major markets [8] - The company’s proprietary manufacturing processes enable the production of industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability to patients worldwide [9]

Core Insights - Mesoblast Limited reported strong operational and financial performance for H1 FY2026, driven by the successful commercial launch of Ryoncil® [7][8] Financial Highlights - Total revenue for H1 FY2026 reached US$51.3 million (A$78.3 million), a significant increase from US$3.2 million in the prior year [5] - Ryoncil® generated gross sales of US$57.0 million, with net revenue of US$48.7 million after adjustments [5] - Gross profit from Ryoncil® was US$44.2 million, compared to nil in the previous year [5] - Reported net loss improved to US$40.2 million from US$47.9 million, marking a year-over-year improvement of US$7.8 million [5] - The company anticipates full-year fiscal 2026 Ryoncil® net revenue to range between US$110 million and US$120 million [6] Operational Highlights - The successful launch of Ryoncil® has led to onboarding 49 transplant centers, with a target of 64 centers covering 94% of U.S. transplants [5] - Coverage for Ryoncil® extends to 280 million U.S. lives, including Federal Medicaid coverage [5] - 84% of patients in a real-world clinical setting completed the initial 28-day treatment regimen as per FDA approval [5] - Mesoblast is expanding Ryoncil®'s clinical indications for lifecycle extension in both adults and children with life-threatening inflammatory conditions [5] Product Development - Mesoblast is advancing its second-generation product, rexlemestrocel-L, targeting chronic discogenic low back pain and inflammatory chronic heart failure [4] - The company received positive feedback from the FDA regarding the potential filing of a Biologics License Application (BLA) for rexlemestrocel-L [12] - A confirmatory study for end-stage patients with chronic heart failure is being planned, with new data supporting the efficacy of rexlemestrocel-L [12] Strategic Positioning - The company has improved its financial position through positive cash flow from Ryoncil® sales and strategic refinancing [8] - Mesoblast is focused on accelerating commercial uptake and advancing regulatory opportunities while maintaining financial discipline [8]

Financial Data and Key Metrics Changes - Total revenues for the first half of FY 2026 were $51.3 million, driven by the successful launch of Ryoncil, with net product revenues of $49 million and a gross margin of 93% [9][10] - R&D expenses increased to $46.1 million compared to $5.1 million in the prior year, primarily due to trials and preparations for BLA [9][10] - The net loss for the period was $40.2 million, an improvement from $48 million in the prior year [10] - Cash at the end of December 2025 was $130 million, supported by a $125 million non-dilutive credit line facility [12][13] Business Line Data and Key Metrics Changes - Ryoncil sales are expected to continue strong growth, with projected full-year net revenues ranging from $110 million to $120 million [14][36] - The company onboarded 49 treatment centers, with Ryoncil now listed on the formulary of 30 centers, indicating strong market adoption [17][19] Market Data and Key Metrics Changes - Ryoncil is now covered by insurance plans representing over 280 million lives, with Medicaid coverage in all states and favorable coverage policies from major payers [19] - The company aims to achieve a 20% market share in the pediatric population by the end of the fiscal year [17][45] Company Strategy and Development Direction - The company plans to expand Ryoncil label indications and seek approval for Revascor and Ryoncil products, focusing on operational efficiency and strategic partnerships [5][22] - The strategic priorities include identifying appropriate patients for Ryoncil therapy, reinforcing superior patient outcomes, and empowering caregivers [20] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving significant market penetration and highlighted the importance of physician education for Ryoncil's adoption [62] - The company is optimistic about the upcoming BLA filings for Revascor and chronic back pain programs, with a focus on mortality as a key endpoint [67][72] Other Important Information - The company is actively optimizing manufacturing logistics to support commercialization of both Ryoncil and the second-generation rexlemestrocel-L pipeline [36] - The company has received positive feedback from the FDA regarding the approvability of endpoints for chronic lower back pain trials [71] Q&A Session Summary Question: Guidance for the upcoming year - The company projects full fiscal year net revenues to range from $110 million to $120 million [41] Question: Ryoncil penetration in pediatrics - The company aims for a 20% penetration based on a patient range of 300 to 375, with a realistic peak share of 40% [45] Question: Revascor BLA and FDA input - The company plans to seek a label for the entire patient population, emphasizing the efficacy across all patients [50] Question: Chronic back pain data submission - The ongoing phase three trial will be the primary dataset for the BLA submission, with previous trials serving as supportive data [54] Question: FDA confirmation on chronic lower back pain endpoint - The company has received confirmation from the FDA that the 12-month pain endpoint is sufficient for approval [71] Question: Outstanding items for Class 4 heart failure programs - The company is engaged in commercial manufacturing and expects to finalize necessary confirmations with the FDA [72]

Financial Data and Key Metrics Changes - Total revenues for the first half of FY 2026 were $51.3 million, driven by the successful launch of Ryoncil, with net product revenues of $49 million and a gross margin of 93% [9][10] - R&D expenses increased to $46.1 million from $5.1 million in the prior year, primarily due to trials and preparations for BLA [9][10] - Sales and general administrative expenses rose to $28.5 million from $18 million, reflecting increased sales and marketing efforts [10] - The net loss for the period was $40.2 million, down from $48 million in the prior year [10][11] - Cash at the end of December 2025 was $130 million, with a new $125 million non-dilutive credit line facility established [12][13] Business Line Data and Key Metrics Changes - Ryoncil, the first FDA-approved allogeneic mesenchymal stromal cell product, generated $49 million in net revenue in the first half of FY 2026 [7][9] - The company aims to achieve a 20% market share for Ryoncil in the pediatric population by the end of the fiscal year [19][56] Market Data and Key Metrics Changes - Ryoncil is now covered by insurance plans representing over 280 million lives, with Medicaid coverage in all states [22] - The product has been included in the formularies of 30 treatment centers, facilitating its adoption [20] Company Strategy and Development Direction - The company plans to expand Ryoncil's label indications and seek approval for Revascor and Ryoncil products, focusing on cash flow and operational efficiency [5][6] - Strategic priorities include identifying appropriate patients for Ryoncil therapy, reinforcing patient outcomes, and empowering caregivers [23][24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving full-year Ryoncil net revenues between $110 million and $120 million [15][49] - The company is focused on building infrastructure to support Ryoncil's market potential and is optimistic about the upcoming BLA filing for Revascor [46][44] Other Important Information - The company is evaluating multiple indications for its second-generation platform, rexlemestrocel-L, targeting chronic discogenic low back pain and chronic ischemic heart failure [29][30] - The company has received positive feedback from the FDA regarding the potential filing of a BLA based on ongoing trials [30][83] Q&A Session Summary Question: Guidance for the upcoming year - The company projects full fiscal year net revenues ranging from $110 million to $120 million [53] Question: Ryoncil penetration in pediatrics - The company aims for a 20% penetration in the pediatric population by the end of the fiscal year, based on a patient range of 300 to 375 [56][57] Question: Revascor BLA and FDA input - The company plans to seek a label for the entire patient population, emphasizing the efficacy of Revascor across all patients [62] Question: Chronic back pain data submission - The ongoing phase III trial will be the primary dataset for the BLA submission, with previous trials serving as supportive data [66][70] Question: FDA confirmation on chronic lower back pain endpoint - The company has received confirmation from the FDA that the 12-month pain endpoint is sufficient for approval [83]

Financial Data and Key Metrics Changes - Total revenues for the first half of FY 2026 were $51.3 million, driven by the successful launch of Ryoncil, with net product revenues of $49 million and a gross margin of 93% [7][10] - R&D expenses increased to $46.1 million from $5.1 million in the prior year, primarily due to adult GVHD trials and other programs [7][9] - Sales and general administrative expenses rose to $28.5 million from $18 million in the prior year, reflecting increased sales and marketing efforts [9] - The net loss for the period was $40.2 million, down from $48 million in the prior year [9] Business Line Data and Key Metrics Changes - Ryoncil, the first FDA-approved allogeneic mesenchymal stromal cell product, generated $49 million in net revenue in the first half of FY 2026, with significant market adoption expected [5][36] - The company aims to achieve a 20% market share for Ryoncil in the pediatric population by the end of the fiscal year [15][45] Market Data and Key Metrics Changes - Ryoncil is now covered by insurance plans representing over 280 million lives, with Medicaid coverage in all states and favorable coverage policies from major payers [17] - The company has onboarded 49 treatment centers, with 30 centers listing Ryoncil on their formularies, facilitating adoption [15][16] Company Strategy and Development Direction - The company plans to expand Ryoncil's label indications and seek approval for Revascor and Ryoncil products, focusing on operational efficiency and cash flow management [4][36] - Strategic priorities include identifying appropriate patients for Ryoncil therapy, reinforcing superior patient outcomes, and empowering caregivers [18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving full-year Ryoncil net revenues between $110 million and $120 million, supported by ongoing market adoption and operational efficiencies [12][36] - The company is optimistic about the potential for Ryoncil in adult indications and other inflammatory diseases, with ongoing clinical trials [20][21] Other Important Information - The company entered into a $125 million non-dilutive credit line facility, enhancing financial flexibility and supporting growth initiatives [12][13] - The company is actively optimizing manufacturing logistics to support commercialization of both Ryoncil and its second-generation products [36] Q&A Session Summary Question: Guidance for the upcoming year - The company projects full fiscal year net revenues ranging from $110 million to $120 million [41] Question: Ryoncil penetration in pediatrics - The company aims for a 20% penetration of the pediatric population by the end of the fiscal year, based on a patient range of 300 to 375 [45][46] Question: Revascor BLA submission details - The company plans to submit data from the ongoing phase three trial as the primary dataset, with previous trials serving as supportive data [55] Question: Chronic back pain trial data submission - The ongoing phase three trial will be included in the filing, with the aim to complete enrollment soon [56][68]

Financial Results and Operational Update for the period ended December 31, 2025 February 2026 ASX: MSB; Nasdaq: MESO For personal use only For personal use only CAUTIONARY NOTE REGARDING FORWARD LOOKING STATEMENTS This presentation includes forward-looking statements and forecasts that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ mat ...

Core Insights - Ryoncil has demonstrated high survival rates in patients with steroid-refractory acute graft-versus-host disease, highlighting the necessity for earlier intervention [1][5] Group 1: Ryoncil's Efficacy - Ryoncil (remestemcel-L-rknd) achieved high survival outcomes in both children and adults with steroid-refractory acute graft-versus-host disease, regardless of treatment line or patient resistance status [2] - In a study involving 53 patients with SR-aGvHD who received Ryoncil as a third-line treatment, 15% died before completing the treatment course, compared to only 2% in the Phase 3 trial when used as a second-line treatment [4] - The results underscore the importance of administering Ryoncil as early as possible after steroid resistance to maximize survival rates [5] Group 2: Regulatory Approval - Ryoncil is the first mesenchymal stromal cell product approved by the U.S. FDA for any indication and is the only product approved for children under age 12 with steroid-refractory acute graft-versus-host disease [3] Group 3: Market Performance - The stock is currently trading 1.8% above its 20-day simple moving average but 2.3% below its 50-day simple moving average, indicating short-term strength but medium-term resistance [6] - Over the past 12 months, shares have decreased by 1.29% and are closer to their 52-week highs than lows [6] - The RSI is at 54.28, indicating neutral territory, while the MACD is above its signal line, suggesting bullish momentum [7] Group 4: Price Action - Mesoblast shares were down 3.06% at $17.73 during premarket trading, with key resistance at $19.50 and key support at $17.50 [8]

Core Insights - Mesoblast Limited presented data on Ryoncil (remestemcel-L-rknd) at the February 2026 Tandem Meetings, highlighting its effectiveness in treating steroid-refractory acute graft-versus-host disease (SR-aGvHD) in both children and adults [1][2]. Group 1: Treatment Efficacy - Ryoncil demonstrated high survival outcomes in SR-aGvHD patients, regardless of age or treatment line, and is the first FDA-approved mesenchymal stromal cell product for this indication [2][6]. - In a study of 53 patients receiving Ryoncil as a third-line treatment, 15% died before completing the treatment, compared to only 2% in the Phase 3 trial for second-line treatment, indicating the importance of early intervention [3]. - Adult patients in the Emergency Investigational New Drug program showed survival rates comparable to children, supporting the rationale for a pivotal trial of Ryoncil in adults [4]. Group 2: Future Developments - A pivotal trial for Ryoncil's use in adults with severe SR-aGvHD is expected to begin enrollment this quarter, pending protocol approval [4]. - If successful, this trial could lead to an extension of Ryoncil's label for adult use, which represents a market approximately three times larger than that of pediatric patients [4]. Group 3: Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing proprietary mesenchymal lineage cell therapy technology [5][7]. - The company has a robust intellectual property portfolio with over 1,000 patents or applications, providing commercial protection until at least 2044 [8]. - Mesoblast is also developing additional therapies for other inflammatory diseases and has established commercial partnerships in Japan, Europe, and China [7].