Core Insights - Mesoblast has received seven years of orphan-drug exclusive approval from the FDA for Ryoncil (remestemcel-L) to treat steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients aged 2 months and older [1][5] - The FDA's approval grants Mesoblast statutory exclusivity, preventing the approval of other mesenchymal stromal or stem cell products for this indication during the exclusivity period [2] - Mesoblast holds biologic exclusivity that prevents other sponsors from referencing the Ryoncil biologic license application until December 2036, providing a barrier against biosimilar market entry [2] - The company has a strong U.S. intellectual property position on MSC composition, manufacturing, and indications, providing commercial protection against competitors through 2044 [3][7] Company Overview - Mesoblast is a leader in developing allogeneic cellular medicines for severe inflammatory conditions, utilizing proprietary mesenchymal lineage cell therapy technology [4] - Ryoncil is the first FDA-approved MSC therapy for SR-aGvHD in pediatric patients [5] - The company is also developing additional cell therapies for other inflammatory diseases and has established commercial partnerships in Japan, Europe, and China [6] Intellectual Property and Manufacturing - Mesoblast has over 1,000 granted patents or patent applications covering various aspects of MSC technology, expected to provide commercial protection through at least 2044 [7] - The company's proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability to patients worldwide [8]

Company Overview - Mesoblast is a global leader in developing allogeneic cellular medicines for severe inflammatory diseases, utilizing proprietary mesenchymal lineage cell therapy technology [4][6] - The company has received FDA approval for RYONCIL (remestemcel-L), the first mesenchymal stromal cell therapy for treating steroid-refractory acute graft versus host disease in pediatric patients [5][6] Recent Developments - Mesoblast appointed Lyn Cobley to its Board of Directors, who brings over 30 years of experience in the financial services industry, including senior roles at major banks [1][3] - Cobley expressed enthusiasm about Mesoblast's recent FDA approval and the company's potential for future growth, including commercialization and new indications for prevalent diseases [3] Strategic Initiatives - The company is focused on developing additional cell therapies for various indications, including adult SR-aGvHD and biologic-resistant inflammatory bowel disease, as well as rexlemestrocel-L for heart failure and chronic low back pain [6] - Mesoblast has established commercial partnerships in key markets such as Japan, Europe, and China [6] Intellectual Property and Manufacturing - 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 [7] - The company utilizes proprietary manufacturing processes to produce industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability for patients worldwide [8]

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]

Core Viewpoint - Mesoblast, a leader in allogeneic cellular medicines for inflammatory diseases, announced that its products, including Ryoncil® and Revascor®, are not expected to be affected by the U.S. Government's tariffs on imported products [1]. Company Overview - Mesoblast specializes in developing allogeneic products using proprietary remestemcel-L and rexlemestrocel-L mesenchymal lineage stromal and precursor cell platform technologies [2]. - The company is recognized for its allogeneic cellular products manufactured from U.S. donors, which are classified as U.S. origin products [2]. Product Information - Ryoncil® (remestemcel-L) is the only FDA-approved allogeneic mesenchymal stromal cell therapy for any indication, specifically for pediatric patients aged 2 months and older with steroid-refractory acute graft versus host disease (SR-aGvHD) [3][5]. - Ryoncil® is designated as a 'U.S. Country of Origin' product, complying with FDA and Customs regulatory guidance [3]. Therapeutic Focus - 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 heart failure and chronic low back pain [6]. - The company has established commercial partnerships in Japan, Europe, and China to expand its market reach [6]. Intellectual Property - Mesoblast holds a robust global intellectual property portfolio with over 1,000 granted patents or patent applications, providing commercial protection expected to last until at least 2041 in major markets [7]. Manufacturing Capabilities - The company utilizes proprietary manufacturing processes to produce industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability to patients worldwide [8].

Core Viewpoint - Mesoblast Limited has been added to the S&P/ASX 200 Index, effective March 6, 2025, highlighting its significance in the Australian market as a leader in allogeneic cellular medicines for inflammatory diseases [1][2]. Company Overview - Mesoblast is a global leader in developing allogeneic cellular medicines aimed at treating severe and life-threatening inflammatory conditions, utilizing its proprietary mesenchymal lineage cell therapy technology [3][5]. - The company has a strong intellectual property portfolio with over 1,000 granted patents or patent applications, providing commercial protection expected to last until at least 2041 in major markets [6]. Product Development - Mesoblast's RYONCIL® (remestemcel-L) 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 [4]. - The company is developing additional therapies for various inflammatory diseases, including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease, as well as rexlemestrocel-L for heart failure and chronic low back pain [5]. Manufacturing Capabilities - Mesoblast employs proprietary manufacturing processes that yield industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability to patients worldwide [7]. Market Presence - Mesoblast operates in Australia, the United States, and Singapore, and is listed on both the Australian Securities Exchange (ASX: MSB) and Nasdaq (NASDAQ: MESO) [8].

Financial Data and Key Metrics Changes - The cash balance at December 31, 2024, was USD 38 million, with pro forma cash of approximately USD 200 million after a global private placement that raised USD 161 million [8] - Net operating cash spend was USD 20.7 million for the first half of FY 2025, representing a 22% reduction compared to the first half of FY 2024 [8] - The company reported a loss after tax of USD 47.9 million for the half year, with total operating cash flows of USD 20.7 million, a reduction of USD 5.9 million from the previous half year [13] Business Line Data and Key Metrics Changes - RYONCIL, the first FDA-approved mesenchymal stromal cell therapy, is now available for children with steroid-refractory acute graft-versus-host disease [15][25] - The FDA approval led to a reversal of a $23 million provision against inventory, now recognized as an inventory asset on the balance sheet [9][12] - The overall response rate for RYONCIL in the Phase 3 trial was 70% at day 28, significantly higher than other therapies for the same condition [17] Market Data and Key Metrics Changes - The addressable market in the US for RYONCIL is approximately 375 new children per year with life-threatening steroid-refractory acute graft-versus-host disease [16] - The cost of treating a child who dies within 12 months of a transplant due to steroid-refractory GVHD is approximately $2.5 million, which is $1.8 million higher than for those who survive [19] - The company is targeting 45 key transplant centers that account for 80% of patients [25][60] Company Strategy and Development Direction - The company aims to expand RYONCIL's label to include inflammatory bowel disease, targeting both children and adults [27] - There is a strategy to establish RYONCIL for adult patients with GVHD, particularly those who fail ruxolitinib, which has no approved therapies [31] - The company is preparing for accelerated approval for Revascor in adults with heart failure with low ejection fraction [49] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the rapid delivery of RYONCIL to children in need and emphasized the extensive efforts across all company levels to ensure a successful launch [86] - The management is confident in the positive feedback from payers regarding RYONCIL's pricing and its clinical efficacy [80] - Future meetings with the FDA are anticipated to clarify the pathway for accelerated approval for heart failure treatments [82] Other Important Information - The company has a scalable manufacturing capability that has been FDA inspected, allowing it to meet global commercial needs [3] - The company has over 1,000 patents and patent applications supporting its products [2] Q&A Session Summary Question: How large is the sales force for RYONCIL and how many centers are trained? - The sales force consists of nine key account managers focusing on 45 key transplant centers, which represent 80% of the potential market. Onboarding has already started [60][61] Question: How far along is the enrollment for the chronic lower back pain trial? - Currently, there are approximately 15 centers onboard, with plans to increase to about 40. The company expects to ramp up enrollment significantly in the coming weeks [68] Question: What feedback has been received from payers regarding RYONCIL's pricing? - Feedback has been positive, with an appreciation for the low number of affected children and the long-term survival benefits RYONCIL offers [80]

mesoblast Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases ASX: MSB; Nasdaq: MESO Financial Results and Operational Update for the Year Ended June 30, 2024 August 2024 CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This presentation includes forward-looking statements 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 achieveme ...

Financial Performance - Cash balance as of December 31, 2024, was US$38 million (A$61 million), with pro-forma cash approximately US$200 million (A$322 million) after a global private placement raising US$260 million (A$400 million) [20] - Total revenue for the half year ended December 31, 2024, was US$3.2 million, a slight decrease from US$3.4 million in the same period of 2023 [21] - Loss after tax for H1 FY2025 was US$47.9 million, compared to a loss of US$32.5 million in H1 FY2024, indicating a 47% increase in losses [24] - Net operating cash spend was US$20.7 million for H1 FY2025, a 22% reduction compared to H1 FY2024 [20] Research and Development - Research and development expenses increased to US$20.6 million in H1 FY2025 from US$12.6 million in H1 FY2024, reflecting a 63% increase [21] - Mesoblast plans to commence a Phase 3 trial of Ryoncil in adults with steroid-refractory acute graft-versus-host disease (SR-aGVHD), where 100-day survival rates were 73% after treatment [59] - The Phase 3 trial for Rexlemestrocel-L in chronic low back pain (CLBP) is actively enrolling 300 patients in a randomized, placebo-controlled study [82] Product Approvals and Efficacy - FDA approval of RYONCIL in December 2024 led to a US$23.0 million reversal of inventory provision, now recognized as an asset on the balance sheet [20] - RYONCIL is the first FDA-approved off-the-shelf cell therapy for pediatric patients aged 2 months and older with steroid-refractory acute graft versus host disease (SR-aGvHD) [28] - RYONCIL delivered an overall response rate of 95% at Day 28 in a Phase 3 study, indicating strong efficacy in treating SR-aGvHD [32] - The wholesale acquisition cost (WAC) of RYONCIL is US$194,000 per intravenous infusion, regardless of body weight [45] Market Needs and Opportunities - There is a significant unmet need in pediatric inflammatory bowel disease (IBD), with over 60% of adult and pediatric Crohn's disease patients unable to achieve remission on anti-TNF agents [53] - The annual pediatric incidence of Crohn's disease in the U.S. is approximately 7,000 children, with a prevalence of 60,000-80,000 [54] - Chronic low back pain (CLBP) affects over 7 million patients in the U.S. and Europe, with minimal treatment options available for those refractory to conservative therapy [63] - Heart failure with low ejection fraction (HFrEF) affects approximately 6.5 million patients in the U.S., with a high mortality rate of about 50% at five years [70] Future Plans and Designations - Mesoblast expects to advance its multiple product pipeline toward FDA approvals over the next six to twelve months [82] - The FDA has granted REVASCOR Rare Pediatric Disease Designation and Regenerative Medicine Advanced Therapy designation, indicating its potential for treating serious conditions in children [80] - Ryoncil® will be available for use in steroid-refractory acute graft-versus-host disease (SR-aGvHD) at U.S. hospitals [82] - Studies for inflammatory diseases will commence for both pediatric and adult label extensions [82] - The company is preparing for accelerated approval filing for heart failure treatments [82] Intellectual Property - The company has an extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets [9]

Core Viewpoint - Mesoblast Limited (MESO) has shown a downtrend recently, losing 10.8% over the past week, but a hammer chart pattern suggests a potential trend reversal as buying interest may be emerging to counteract selling pressure [1][2]. Technical Analysis - The hammer chart pattern indicates a possible bottoming out, with reduced selling pressure, suggesting a bullish outlook for the stock [2][4]. - A hammer pattern forms when there is a small difference between opening and closing prices, with a long lower wick, indicating that the stock found support after hitting a new low during a downtrend [3][4]. - The occurrence of a hammer pattern at the bottom of a downtrend signals that bears may have lost control, indicating a potential trend reversal [4]. Fundamental Analysis - There has been a positive trend in earnings estimate revisions for MESO, which is a bullish indicator, as it typically leads to price appreciation [6]. - The consensus EPS estimate for MESO has increased by 2.5% over the last 30 days, reflecting analysts' agreement on the company's potential for better earnings [7]. - MESO currently holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks, which historically outperform the market [8].

Core Insights - Mesoblast Limited has appointed Dr. Gregory George, its largest shareholder, to the Board of Directors, which is expected to enhance the company's operational efficiency as it transitions into a commercial organization [1][3]. Company Overview - Mesoblast is a global leader in developing allogeneic cellular medicines aimed at treating severe inflammatory diseases, utilizing its proprietary mesenchymal lineage cell therapy technology [4]. - The company has a strong intellectual property portfolio with over 1,000 granted patents or applications, providing commercial protection expected to last until at least 2041 in major markets [7]. Leadership and Expertise - Dr. Gregory George has a background as a medical scientist and founded the largest privately owned ambulatory surgical center company in the U.S., bringing valuable operational experience to Mesoblast [2][3]. - The CEO, Dr. Silviu Itescu, emphasized that Dr. George's operational skills will be crucial for Mesoblast's growth and efficiency [3]. Product Development - Mesoblast's RYONCIL® (remestemcel-L) 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 [5]. - The company is developing additional therapies for various inflammatory diseases and conditions, including heart failure and chronic low back pain, leveraging its allogeneic stromal cell technology platforms [6]. Manufacturing Capabilities - Mesoblast employs proprietary manufacturing processes that yield industrial-scale, cryopreserved, off-the-shelf cellular medicines, ensuring availability to patients worldwide [8].