Annuncio • Aug 17
Mesoblast Limited Completes Patient Treatment In Pivotal Phase 3 Trial Of Rexlemestrocel-L For Chronic Low Back Pain Mesoblast Limited announced that it has completed patient treatment in the MSB-DR004 pivotal randomized controlled Phase 3 trial of rexlemestrocel-L for chronic low back pain (CLBP) associated with the inflammatory condition of degenerative disc disease. The major milestone was achieved with 350 patients randomized and treated with either an intra-discal injection of rexlemestrocel-L or sham injection. Total patient numbers treated increased from 300 to 350 after strong demand from trial investigators to have their patients enrolled in the innovative program. The trial’s primary endpoint aims to confirm the durable pain reduction at 12 months from a single intra-discal injection of rexlemestrocel-L seen in the earlier MSB-DR003 trial. With 350 treated patients, the trial is well-powered for showing a greater treatment benefit in patients receiving rexlemestrocel-L compared with controls. Secondary endpoints include improvements in function, quality of life, and cessation of pain medication, including opioids. CLBP caused by inflammation and degenerative disc disease is a serious condition with a prevalence of over 7 million people in the U.S. alone. The indication has potential peak year revenue of >USD 10 billion for Mesoblast even with just single digit market penetration. Rexlemestrocel-L has Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for treatment of CLBP due to degenerative disc disease providing eligibility for priority review once the Biologics License Application (BLA) has been filed. Top-line results are expected in mid-CY2027 after the last treated patient has completed 12 months follow-up. Mesoblast's second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell product candidate rexlemestrocel-L is being evaluated in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration. Mesoblast has agreement with FDA on the design of the randomized, placebo-controlled pivotal Phase 3 trial and on the trial's 12-month primary endpoint of pain reduction, previously successfully met in Mesoblast’s first Phase 3 trial, as an approvable indication. Key secondary measures include improvement in quality of life and function. A further secondary endpoint will be reduction in opioid use since discogenic back pain accounts for approximately 50% of opioid prescriptions in the U.S. Back pain is the leading cause of disability in Americans under 45 years, with an annual prevalence in the general US adult population of 10-30%. CLBP caused by inflammation and degenerative disc disease (DDD) is a serious condition with a prevalence of over 7 million people in the US alone. CLBP due to DDD is a leading cause of disability, and is associated with impaired quality of life, severe limitations in ability to perform activities of daily living, reduced ability to work, and negative impacts on mental health. CLBP accounts for approximately 50% of prescription opioid usage in the US, making the condition a significant contributor to the opioid epidemic. Annuncio • Jul 14
Mesoblast Limited Achieves Target of 300 Treated Patients in Pivotal Phase 3 Trial for Chronic Low Back Pain Mesoblast Limited has achieved its target of at least 300 patients treated in the MSB-DR004 pivotal Phase 3 randomized controlled trial of rexlemestrocel-L for chronic low back pain (CLBP) associated with degenerative disc disease. The trial aims to confirm the durable pain reduction from a single intra-discal injection of rexlemestrocel-L seen in the earlier MSB-DR003 trial. Commercial manufacturing is proceeding in parallel so that the company can file for approval as soon as possible after trial results readout. CLBP caused by inflammation and degenerative disc disease is a serious condition with a prevalence of over 7 million people in the U.S. alone. The indication is a potential blockbuster for Mesoblast with potential peak year revenue of more than USD 10 billion with single digit market penetration. The trial's primary endpoint is powered to show a significant difference in reduction of low back pain at 12 months between rexlemestrocel-L and sham controls. Secondary endpoints include improvements in function, quality of life, and cessation of pain medication, including opioids. Rexlemestrocel-L has Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA) for treatment of CLBP due to degenerative disc disease providing eligibility for priority review once the Biologics License Application (BLA) has been filed. Top-line results are expected in mid-calendar year 2027 after the last treated patient has completed 12 months follow-up. The 300-patient randomized controlled confirmatory Phase 3 trial of Mesoblast's second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell product candidate rexlemestrocel-L in combination with hyaluronic acid (HA) as delivery agent for injection into the lumbar disc is actively enrolling in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration at multiple sites across the U.S. FDA has previously agreed on the design of this 300-patient randomized, placebo-controlled confirmatory Phase 3 trial, and the 12-month primary endpoint of pain reduction as an approvable indication. This endpoint was successfully met in Mesoblast's first Phase 3 trial. Key secondary measures include improvement in quality of life and function. A particular focus is on treatment of patients on opioids, since discogenic back pain accounts for approximately 50% of prescription opioid usage in the U.S. Significant pain reduction and opioid cessation were observed in Mesoblast's first Phase 3 trial. FDA has designated rexlemestrocel-L a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of chronic low back pain. RMAT designation provides all the benefits of Breakthrough and Fast Track designations, including rolling review and eligibility for priority review on filing of a Biologics License Application (BLA). Back pain is the leading cause of disability in Americans under 45 years, with an annual prevalence in the general U.S. adult population of 10-30%. CLBP caused by inflammation and degenerative disc disease (DDD) is a serious condition with a prevalence of over 7 million people in the U.S. alone. CLBP due to DDD is a leading cause of disability, and is associated with impaired quality of life, severe limitations in ability to perform activities of daily living, reduced ability to work, and negative impacts on mental health. CLBP accounts for approximately 50% of prescription opioid usage in the U.S., making the condition a significant contributor to the opioid epidemic. Annuncio • Jul 01
Mesoblast Limited Receives BLA Filing Number And Requests Modular Review For Rexlemestrocel-L In Patients With End-Stage Heart Failure And LVADs Mesoblast Limited had received a Biologics License Application (BLA) filing number from the U.S. Food and Drug Administration (FDA) and had requested a modular review of its BLA for rexlemestrocel-L in prevention of life-threatening gastrointestinal bleeding due to right ventricular dysfunction in end-stage heart failure patients with a left ventricular assist device (LVAD). Rexlemestrocel-L had received Orphan Drug Designation for prevention of life-threatening major mucosal bleeding events and had Regenerative Medicine Advanced Therapy (RMAT) designation for this patient population, providing eligibility for rolling and priority reviews of the BLA. The new FDA leadership this past week provided additional guidance to how it approaches regulatory flexibility for products which address orphan rare diseases with high mortality and irreversible morbidity. The new draft guidance to industry titled ‘Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products’ highlights FDA's flexible approach to substantial evidence of effectiveness. This follows the May guidance from FDA titled ‘Chemistry, Manufacturing, and Controls Flexibilities for Developing Human Cellular and Gene Therapy Products for a Biologics License Application.’ Rexlemestrocel-L is an allogeneic preparation of immunoselected and culture-expanded mesenchymal precursor cells (MPC) and is being developed as an immunomodulatory therapy to address the high degree of inflammation in the heart and in the circulation that is present across the spectrum of heart failure and reduced ejection fraction (HFrEF) patients, from New York Heart Association (NYHA) class II through end-stage CHF, in order to reduce the high rate of major cardiac events and complications. This investigational therapy had been trialled in two large placebo-controlled randomized studies in patients with CHF, a 565-patient trial in NYHA class II/III HFrEF patients and a 159-patient trial in end-stage HFrEF patients implanted with a left ventricular assist device (LVAD). Rexlemestrocel-L had US Food and Drug Administration (FDA) Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations for patients with end-stage HFrEF implanted with an LVAD. Chronic heart failure (CHF) is characterized by poor heart function resulting in insufficient blood flow to the body’s vital organs and extremities. This condition affects approximately 6,500,000 people in the United States and 26,000,000 people globally with increasing prevalence and incidence. CHF patients are commonly classified according to the New York Heart Association (NYHA) categories based on the patient’s physical limitations. Class I (mild) patients have no limitations while Class IV patients (severe/end stage) experience symptoms even at rest. The mortality rate approaches 50% at 5 years as patients progress beyond NYHA early class II disease in parallel with increasing inflammation in the heart and in the circulation. Despite recent approvals of new therapies for HFrEF, NYHA class II/III HFrEF patients with inflammation remain at high risk for cardiac death, heart attacks and strokes. Every year in the United States over 100,000 patients progress to end-stage HFrEF, with a one-year mortality as high as 50%. In these patients, more than 2,500 life prolonging LVADs are implanted in the US annually, of whom approximately 80% undergo the procedure as destination or permanent therapy. Most patients receiving LVADs as destination therapy have an ischemic HFrEF etiology. Compared to patients with non-ischemic HFrEF, patients with ischemic HFrEF have a 76% lower likelihood of LV functional recovery following LVAD implantation, and increased mortality over the initial 1-2 years. Resistance to functional recovery in ischemic HFrEF patients is thought to be due to excessive inflammation and microvascular insufficiency in the ischemic myocardium. Mesoblast’s Ryoncil (remestemcel-L-rknd) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA-approved mesenchymal stromal cell (MSC) therapy. Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Ryoncil is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China. Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications provide commercial protection extending through to at least 2044 in all major markets. The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide. Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). Annuncio • Apr 29
Mesoblast Limited Achieves Patient Recruitment Target in Pivotal Phase 3 Trial for Chronic Low Back Pain Mesoblast Limited announced that its pivotal Phase 3 clinical trial evaluating rexlemestrocel-L for the treatment of chronic low back pain (CLBP) associated with degenerative disc disease has achieved its patient recruitment target. The placebo-controlled study (MSB-DR004) will follow at least 300 patients randomized to a single intra-discal injection of rexlemestrocel-L or sham control for 12 months. The trial seeks to confirm results from the earlier MSB-DR003 trial where a single intra-discal injection of rexlemestrocel-L resulted in clinically meaningful reductions in pain and opioid usage for up to three years. The trial’s primary endpoint is powered to show a significant difference in reduction of low back pain at 12 months between rexlemestrocel-L and sham controls. Secondary endpoints include improvements in function, quality of life, and cessation of pain medication, including opioids. Top-line results are expected in mid-CY2027 after the last randomized patient has completed follow-up. A positive readout will be used in support of an expected regulatory filing in Third Quarter CY2027 for United States Food and Drug Administration (FDA) approval. Rexlemestrocel-L has Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for treatment of CLBP due to degenerative disc disease providing eligibility for priority review once the Biologics License Application (BLA) has been filed. CLBP caused by inflammation and degenerative disc disease is a serious condition with a prevalence of over 7 million people in the U.S. alone. The indication is a potential blockbuster for Mesoblast with potential peak year revenue of >USD 10 billion with single digit market penetration. The 300-patient randomized controlled confirmatory Phase 3 trial of Mesoblast’s second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell product candidate rexlemestrocel-L in combination with hyaluronic acid (HA) as delivery agent for injection into the lumbar disc is actively enrolling in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration at multiple sites across the U.S. FDA has previously agreed on the design of this 300-patient randomized, placebo-controlled confirmatory Phase 3 trial, and the 12-month primary endpoint of pain reduction as an approvable indication. This endpoint was successfully met in Mesoblast’s first Phase 3 trial. Key secondary measures include improvement in quality of life and function. A particular focus is on treatment of patients on opioids, since discogenic back pain accounts for approximately 50% of prescription opioid usage in the US. Significant pain reduction and opioid cessation were observed in Mesoblast’s first Phase 3 trial. FDA has designated rexlemestrocel-L a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of chronic low back pain. RMAT designation provides all the benefits of Breakthrough and Fast Track designations, including rolling review and eligibility for priority review on filing of a Biologics License Application (BLA). Back pain is the leading cause of disability in Americans under 45 years, with an annual prevalence in the general US adult population of 10-30%. CLBP caused by inflammation and degenerative disc disease (DDD) is a serious condition with a prevalence of over 7 million people in the US alone. CLBP due to DDD is a leading cause of disability, and is associated with impaired quality of life, severe limitations in ability to perform activities of daily living, reduced ability to work, and negative impacts on mental health. CLBP accounts for approximately 50% of prescription opioid usage in the US, making the condition a significant contributor to the opioid epidemic. Annuncio • Mar 12
Mesoblast Limited Appoints Dr. Teresa Montagut As Head Of Clinical Development And Medical Affairs Mesoblast Limited announced the appointment of Teresa Montagut MD, PhD in the newly established position of Head of Clinical Development and Medical Affairs reporting to the Chief Medical Officer Dr. Eric Rose. In this role, Teresa will lead Mesoblast’s medical affairs organization, fostering clinical collaborations and spearheading investigator-initiated trials, enhancing clinical and medical communications, and engaging with healthcare professionals. She will play a critical role in unlocking the value of Mesoblast's cell therapy programs in new pediatric and adult inflammatory conditions in partnership with investigators and key opinion leaders. Teresa brings extensive experience in medical leadership and pharmaceutical development, with a strong background in translating clinical science into meaningful patient outcomes. Teresa joined Mesoblast from Regeneron where she was Global Head of Early Pipeline Studies in Oncology and Head of Medical Affairs for Investigator Sponsored Studies in gastrointestinal and genitourinary areas. She previously led multiple cancer immunotherapy programs across Novartis, Genentech, and Atara Biotherapeutics. Teresa earned her MD from Universidad Nacional Autónomade México and her PhD in Tumor Immunology from Memorial Sloan Kettering Cancer Center/Cornell University. She completed fellowships at Massachusetts General Hospital, Howard Hughes Medical Institute, and Rockefeller University. Teresa also serves on the Board of Directors of the Global Pediatric Alliance, supporting maternal and pediatric healthcare in under-served indigenous communities in Latin America, particularly Mexico. Annuncio • Feb 27
Mesoblast Limited Provides Revenue Guidance for Fiscal Year 2026 Mesoblast Limited provided revenue guidance for fiscal year 2026. Mesoblast anticipates full-year fiscal 2026 Ryoncil net revenue to range between USD 110 million and USD 120 million. Annuncio • Feb 12
Mesoblast Limited Provides Data Presented at the February 2026 Tandem Meetings of the American Society for Transplantation and Cellular Therapy and the Center for Blood and Marrow Transplant Research in Salt Lake City, Utah Mesoblast Limited provided data presented at the February 2026 Tandem Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for Blood and Marrow Transplant Research (CIBMTR) in Salt Lake City, Utah. The study results showed that Ryoncil®? (remestemcel-L-rknd) achieved similarly high survival outcomes in steroid-refractory acute graft-versus-host disease (SR-aGvHD) irrespective whether used in children or adults, as second or third line, and in ruxolitinib naive or resistant patients. Ryoncil®? is the first mesenchymal stromal cell (MSC) product approved by the U.S. Food and Drug Administration (FDA) for any indication, and the only product approved for children under age 12 with steroid-refractory acuteraft-versus-host disease ("SR-aGvHD"). Importantly, of the 53 patients with SR-aGvHD who received Ryoncil®? as third line treatment under the Emergency Investigational New Drug (EIND) program (89% grade III/IV disease), 15% died before being able to complete a full treatment course of Ryoncil®? compared with only 2% of patients who received Ryoncil®®? as second-line in the Phase 3 trial MSB-GVHD001. These results emphasize the importance of using Ryoncil®? as early as possible after steroid resistance in acute GVHD in order to complete a full treatment course and maximize survival. Additionally, adult patients in the EIND program of Ryoncil®? had at least as favorable day 100 survival as children in the EIND program, providing strong support and rationale for the planned pivotal trial of early use of Ryoncil®? as part of the second-line treatment regimen in adults with severe SR-aGvHD. The trial is expected to commence enrollment this quarter after protocol approval by the trial's central Institutional Review Board (IRB). If successful, the trial will support label extension of Ryoncil® for use in adults - a population approximately three times the size of the pediatric SR-aGvHD population. Annuncio • Jan 27
Mesoblast Limited Announces Update on Ryoncil (remestemcel-L-rknd) for Pediatric SR-aGvHD and Plans for Adult Trials Mesoblast Limited provided an update on use of Ryoncil (remestemcel-L-rknd) since commercially available in March 2025 for the approved label in children 2 months and older with steroid-refractory acute graft-versus-host disease (SR-aGvHD). Ryoncil is the first mesenchymal stromal cell (MSC) product approved by the U.S. Food & Drug Administration (FDA) for any indication. Of the first 25 patients treated with Ryoncil in a 'real-world' clinical setting post launch, 21 were alive (84%) and completed the initial 28-day treatment regimen as per the FDA approval label. The four patients who did not complete the 28-day treatment course had been offered and failed other therapies prior to use of Ryoncil and died of severe SR-aGvHD within 28 days. To ensure that no patient is left behind in receiving this potentially life-saving therapy, Mesoblast has established a patient access hub termed MyMesoblast, where Ryoncil is available for ordering. To date 45 transplant centers have been onboarded, with a target of 64 centers which account for 94% of transplantants performed in the U.S. Ryoncil coverage by government and commercial payers already extends to over 260 million U.S. lives with Federal Medicaid coverage by U.S. Centers for Medicare & Medicaid Services (CMS) and mandatory fee-for-service Medicaid coverage in all U.S. states. Issuance on October 1, 2025, of a specific Healthcare Common Procedure Coding System (HCPCS) J-Code by CMS for billing and reimbursement resulted in greater usage of Ryoncil under CMS coverage versus commercial coverage in the last quarter. These commercial activities will continue to serve the company well as it seeks to expand the FDA label for Ryoncil to adults with severe SR-aGv HD, a market size approximately three times that of the pediatric SR-aGvHD population. A pivotal trial of Ryoncil in adults with severe SR-a GvHD will be conducted with the NIH-funded Bone Marrow Transplant Clinical Trials Network (BMT-CTN) and is expected to commence site enrollment this quarter. Annuncio • Jan 21
Mesoblast Limited Provides Feedback Received from the U.S. Food & Drug Administration on Potential Filing of a Biologics License Application for its Allogeneic Cell Therapy Product Rexlemestrocel-L in Patients with Chronic Low Back Pain Mesoblast Limited provided feedback received from the U.S. Food & Drug Administration (FDA) on potential filing of a Biologics License Application (BLA) for its allogeneic cell therapy product rexlemestrocel-L in patients with chronic discogenic low back pain (CLBP). This follows FDA's Type B meeting review of data from Mesoblast's first randomized controlled Phase 3 trial (MSB-DR003) on pain reduction and relationship to decreased use or elimination of opioids for up to three years following a single rexmestrocel-L administration. Mesoblast is seeking FDA approval for rexlemestrocel- L based on reduction in CLBP through 12 months. Comparing outcomes between rexlemestrocel- Ltd. and placebo from MSB-DR003 trial, FDA acknowledged that the effects on pain intensity appear to favor the active arm. FDA also confirmed that a clinically meaningful reduction in pain intensity in the active arm versus placebo at 12 months can support product efficacy and stated that the robust results on opioid reduction from at least one adequate and well controlled trial could be included in the Clinical Studies section of product labeling. CLBP is a major contributing factor to the U.S. opioid crisis, and rexlemestrocel-l has received Regenerative Medicine Advanced Therapy (RMAT) designation from FDA for treatment of CLBP. The 300-patient randomized controlled confirmatory Phase 3 trial of Mesoblast's second generation allogeneic, STRO3-immunoselected, and industryally manufactured stromal cell product candidate rexlemestrocel- lumbar disc is actively enrolling in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration at multiple sites across the U.S. FDA has previously agreed on the design of this 300-patient randomized, placebo-controlled confirmatory Phase 3 trial, and the 12-month primary endpoint of pain reduction as an effective indication. In light of the devastating opioid crisis that continues to rage in the U.S., in September 2025 FDA provided new Guidance to Industry on Development of Non-Opioid Agents for Treatment of Chronic Pain. Significant pain reduction and opioid cessation were observed in Mesoblast's first Phase 3 trial. FDA has designated rexlemestrocel-S a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of chronic low back pain. The therapies from the Company's proprietary mesenchymal line cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process. Mesoblast's Ryoncil (remestemcel-L-rknd) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA-approved FDA-approved mesenchymal stromal cell (MSC) therapy. Mesoblast is committed to developing additional cell therapies for distinct indications based on itsremestemcel-L and rexlemestrocel -L allogeneic stromal cell technology platforms. Ryoncil is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologics. Annuncio • Jan 19
Mesoblast Limited Provides Feedback Received from the U.S. Food & Drug Administration on Potential Filing of A Biologics License Application for Its Allogeneic Cell Therapy Product Rexlemestrocel-L in Patients with Chronic Low Back Pain Mesoblast Limited provided feedback received from the U.S. Food & Drug Administration (FDA) on potential filing of a Biologics License Application (BLA) for its allogeneic cell therapy product rexlemestrocel-L in patients with chronic discogenic low back pain (CLBP). This follows FDA's Type B meeting review of data from Mesoblast's first randomized controlled Phase 3 trial (MSB-DR003) on pain reduction and relationship to decreased use or elimination of opioids for up to three years following a singlerexlemestrocel-L administration. Mesoblast is seeking FDA approval for rexlemestrocel- L based on reduction in CLBP through 12 months. Comparing outcomes between rexlemestrocel- Ltd. and placebo from MSB-DR003 trial, FDA acknowledged that the effects on pain intensity appear to favor the active arm. FDA also confirmed that a clinically meaningful reduction in pain intensity in the active arm versus placebo at 12 months can support product efficacy and stated that the robust results on opioid reduction from at least one adequate and well controlled trial could be included in the Clinical Studies section of product labeling. A second randomized controlled Phase 3 trial, MSB-DR004, is actively recruiting across 40 sites in the U.S., is over 50% enrolled, and is expected to complete the 300-patient enrollment target in the coming three months. CLBP is a major contributingory factor to the U.S. opioid crisis, and rexlemestrocel- lumbar disc has received Regenerative Medicine Advanced Therapy (RMAT) designation from FDA for treatment of CLBP. The 300-patient randomized controlled confirmatory Phase 3 trial of Mesoblast's second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell product candidate rexlemestemcel-L in combination with hyaluronic acid (HA) as delivery agent for injection into the lumbar disc is actively enrolling in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration at multiple sites across the U.S. FDA has previously agreed on the design of this 300-patient randomized, placebo-controlled confirmatory Phase 3 trial, and the 12-month primary endpoint of pain reduction as an approvable indication. This endpoint was successfully met in Mesoblast's first Phase 3 trial. A particular focus is on treatment of patients on opioids, since discogenic back pain accounts for approximately 50% of prescription opioid usage in the U.S. In light of the devastating opioid crisis that continues to rage in the U.S., in September 2025 FDA provided new Guidance to Industry on Development of Non-Opioid Agents for Treatment of Chronic Pain. Significant pain reduction and opioid cessation were observed in Mesoblast's first phase 3 trial. In Mesoblast's first randomizedcontrolled Phase 3 trial of 404 patients, 168 of whom were taking opioids at baseline, more than 3-fold higher numbers of patients treated with a single intra-discal injection ofrexlemestrocel-L + HA were able to cease use of all opioids by 36 months compared with saline-treated controls (p=0.008). FDA has designated rexlemestrocel-lknd) for the treatment of chronic low back pain. The therapies from the Company's proprietary mesenchymal lineage cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process. Mesoblast's Ryoncil®? (remestemcel-L-rknd) for the treatment the treatment of steroid-refractory acute graft versus placebo at 12 months. Annuncio • Dec 12
Mesoblast Limited Announces Independent Study Presented at American Society of Hematology (ASH) Annual Meeting Concludes Remestemcel-L Superior to Ruxolitinib in Clinical Outcomes as Treatment for SR-aGvHD Mesoblast Limited announced that an independent peer-reviewed comparative analysis of efficacy and safety between remestemcel-L and ruxolitinib for treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) was presented at the 67th ASH Annual meeting in Florida this past week. The independent study authors concluded that remestemcel-L showed superior outcomes in complete and overall remission compared with ruxolitinib. The meta-analysis involved 2,732 patients (1,993 in the treatment arms and 523 in the control arms) across 11 studies. Among treatment groups, 644 patients received remestemcel-L. and 1,349 received ruxolitinib. While both ruxolitinib and remestemcel-L significantly improved quality of life in treating SR-aGvHD, remestemcel-L demonstrated superior outcomes in complete and Overall remission as well as differences in hematology, cardiac and hepatic adverse events. The authors also concluded that while both therapies exhibit favorable safety profiles, clinical decisions should consider the differences in adverse events. Ryoncil®? is the first mesenchymal stromal cell (MSC) product approved by the U.S. Food and Drug Administration (FDA) for any indication, and the only product approved for children under age 12 with SR-aGvHD. The Spotlight segment of Blood, the journal of ASH, featured FDA approval of Ryoncil®? as an important advance in the field for treatment of acute GvHD. Annuncio • Nov 25
Mesoblast Limited Provides Revenue Guidance for the Quarter Ending December 31, 2025 Mesoblast Limited provided revenue guidance for the quarter ending December 31, 2025. For the period, the company expects gross revenue of more than USD 30.0 million from sales of Ryoncil® (remestemcel-L-rknd). This represents more than 37% increase on the USD 21.9 million in gross revenue from Ryoncil® in the prior quarter ended September 30, 2025. Annuncio • Nov 23
Mesoblast And BMT CTN Initiate Pivotal Trial Of Ryoncil For Severe Acute GVHD In Adults Mesoblast Limited announced a collaboration with the NIH-funded Blood and Marrow Transplant Clinical Trials Network (BMT CTN) to initiate a pivotal trial of Ryoncil® (remestemcel-L-rknd) as part of a first-line regimen in adults with severe acute graft versus host disease (aGvHD) refractory to corticosteroids. This trial aims to address the high rate of non-responsiveness and mortality in these patients. The trial will explore the use of Ryoncil®, already FDA-approved for children and adolescents, in adults with severe SR-aGvHD. The trial protocol will be submitted to the FDA to initiate enrollment in the first quarter of 2026. Annuncio • Nov 17
Mesoblast Limited Appoints James M. O’Brien as Chief Financial Officer Mesoblast Limited announced that in connection with the Company’s transition to a fully integrated commercial organization it has appointed James M. O’Brien as its US-based Chief Financial Officer (CFO). Jim has extensive experience in all aspects of financial management and planning having spent the majority of his career with multi-national public and private companies in the life sciences, biotechnology, and pharmaceutical industries. Jim has had global responsibility overseeing Corporate and Business Unit finance, Reporting, Internal Controls and Accounting Operations at Actavis plc which subsequently acquired Allergan plc prior to its acquisition by AbbVie Inc. Jim created a world-class financial budgeting and forecasting process to track business performance and trends, and developed financial tools and analyses supporting a range of corporate activities including Business Strategy and Corporate Development, integrating international business acquisitions and managing activities for transactions of nearly $10 billion. During his tenure, Actavis had proforma revenues of over $8 billion. Previously, Jim built a finance team overseeing all supply chain and revenue management for Nycomed, a private equity owned company with $750 million revenues that was acquired by Sandoz for $1.5 billion. Jim’s recent CFO roles included NASDAQ-listed biopharmaceutical company Cognition Therapeutics Inc., overseeing the company’s successful initial public offering, and at dual-listed Finnish biotechnology company Faron Pharmaceuticals Ltd. He has also held finance roles at Bristol-Myers Squibb (BMS). Jim is a certified public accountant and previously worked at PriceWaterhouseCoopers in New York and Washington DC. Annuncio • Nov 05
Mesoblast Limited to Meet with FDA Next Month to Discuss Rexlemestrocel-L and Opioid Cessation Mesoblast Limited announced that the U.S. Food & Drug Administration (FDA) has scheduled a meeting in early December to discuss Mesoblast's data on opioid reduction and cessation from its first Phase 3 study (MSB-DR003) of rexlemestrocel-L in patients with chronic low back pain (CLBP). In Mesoblast's first randomized controlled Phase 3 trial of 404 patients, 168 of whom were taking opioids at baseline, more than 3-fold higher numbers of patients treated with a single intra-discal injection of Rexlemestrocel-L + HA were able to cease use of all opioids by 36 months compared with saline-treated controls (p=0.008). In light of the devastating opioid crisis that continues to rage in the US, in September 2025 FDA provided new Guidance to Industry on Development of Non-Opioid Agents for Treatment of Chronic Pain.1 Since CLBP is the principal cause of chronic prescription opioid use, there is a significant need for a safe, effective, and durable opioid-sparing treatment in patients with CLBP associated with degenerative disc disease. Rexlemestrocel-L has received Regenerative Medicine Advanced Therapy (RMAT) designation for treatment of CLBP due to degenerative disc disease, a serious condition that is a major contributory factor to the nation's opioid crisis. Mesoblast is seeking FDA approval based on reduction in CLBP through 12 months and is actively recruiting a 300-patient confirmatory Phase 3 trial across 40 sites in the US, with enrollment expected to be completed in the coming quarter. Annuncio • Oct 27
Mesoblast Limited, Annual General Meeting, Nov 25, 2025 Mesoblast Limited, Annual General Meeting, Nov 25, 2025, at 10:30 AUS Eastern Standard Time. Agenda: Annual General Meeting. Annuncio • Oct 03
Mesoblast Limited Receives J-Code from Medicare & Medicaid Services (CMS) Facilitating Reimbursement and Broader Patient Access Mesoblast Limited announced that a specific Healthcare Common Procedures Coding System (HCPCS) J-Code assigned to Ryoncil®? (remestemcel-L-rknd) by United States Medicare & Medicaid Services (CMS) became active for billing and reimbursement on October 1, 2025.1 Formal recognition by CMS is a significant milestone for Ryoncil®? as the product becomes easier to bill and pay for. The new permanent J-Code, J3402, provides a standardized, clear, permanent, and specific billing pathway for Ryoncil®? by Medicaid, facilitating reimbursement and broader patient access for this important therapy. Additionally, commercial payers look to the permanent J-code to update their coverage systems. Ryoncil®? is the first mesenchymal stromal cell (MSC) product approved by the U.S. Food and Drug Administration (FDA) for any indication, and the only product approved for children under age 12 with steroid-refractory acute graft-versus-host disease (SR-aGvHD). Healthcare providers can begin using J3402 for claims submitted on or after October 1, 2025. Annuncio • Sep 26
Mesoblast Limited Announces its Allogeneic Cell Therapy Products Are Designated U.S. Country of Origin Mesoblast Limited reiterated that its allogeneic cell therapy products are manufactured from U.S. donors in the U.S. and designated as U.S. origin products not subject to tariffs on imported branded or patented pharmaceutical products. Ryoncil®? (remestemcel-L) is the only allogeneic mesenchymal stromal cell therapy approved by U.S. Food and Drug Administration (FDA) for any indication. As documented in the Company's Biologic License Application (BLA), Ryoncil®? is designated a 'U.S. Country of Origin' product in line with U.S. FDA and Customs regulatory guidance. Ryoncil®? is approved for treatment of pediatric patients 2 months and older, including adolescents and teenagers, with steroid-refractory acute graft versus host disease (SR-aGvHD), a condition with high mortality rates. Mesoblast continues to ensure that all its products, whether for SR-aGvHD, chronic heart failure, chronic back pain, or other inflammatory indications, are manufactured from U.S., donors at U.S. sites. Annuncio • Sep 15
Mesoblast Limited Announces Successful Commercial Launch of Ryoncil Mesoblast Limited announced that the successful commercial launch of Ryoncil®? (remestemcel-L-rknd), the only mesenchymal stromal cell (MSC) product approved by the United States Food and Drug Administration (FDA), was highlighted this past week in presentations at the Morgan Stanley and Cantor Fitzgerald Annual Global Healthcare Conferences held in New York. Mesoblast Chief Executive Silviu Itescu reiterated the strong first quarter launch of Ryoncil®®? and product sales in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGvHD) as recently reported in the Company's full year earnings and discussed the continued drive to onboard new sites and increase product usage. Dr. Itescu additionally highlighted plans for expanding Ryoncil®? into additional indications including adult SR-aGvHD and inflammatory bowel disease (IBD), as well as key Phase 3 milestones for its second-generation product Rexlemestrocel-L. Annuncio • Sep 04
Mesoblast Limited announced that it expects to receive AUD 76.8 million in funding Mesoblast Limited announced it has entered into convertible note subscription agreement to issue 5% unsecured convertible notes for gross proceeds of (AUD 76,800,000) $50,000,000 on September 3, 2025. The transaction includes participation from SurgCenter principals and existing Mesoblast shareholders, Gregory George and William Gueck. The convertible notes have a coupon of 5% per annum on the face value of issued notes. The maturity date of the convertible notes will be 5 years after the first issuance of notes (unless redeemed or converted earlier). At any time up to the maturity date, the Investors may elect to convert the notes issued into fully paid ordinary shares or ADRs of Mesoblast at the conversion price of $16.25 per ADR (American Depositary Receipt) equivalent to AUD 2.501 per ASX-listed share. As consideration, the Investors collectively will receive a commitment fee of ($100,000) AUD 153,846.15. and, subject to shareholder approval, of 2 million warrants over 2 million ordinary shares (or 200,000 Mesoblast ADRs) for entering into the convertible note option, and a further 3 million warrants over 3 million ordinary shares (or 300,000 Mesoblast ADRs) should Mesoblast exercise this option. The warrants shall have the same exercise price as the conversion price of the notes and a maturity date of 4 years from the date of first issuance of the warrants. The conversion price is subject to adjustment mechanisms in the event of future share issues, capital reductions, share consolidations and other corporate actions in accordance with customary adjustment rules. The company may issue notes in tranches of AUD 15,384,615.38. ($10,000,000) to the Investors. The transaction is subject to to shareholder approval at the upcoming Annual General Meeting (AGM). Annuncio • Jul 01
Mesoblast and FDA Align on Key Items for Revascor Biologic License Application in Ischemic Heart Failure Mesoblast announced alignment with the United States Food and Drug Administration (FDA) on items required for filing a Biologics License Application (BLA) for Revascor®? (rexlemestrocel-L) in the treatment of patients with ischemic heart failure with reduced ejection fraction (HFrEF) and inflammation. In follow-up to the successful Type B meeting in early 2024 under the existing Regenerative Medicine Advanced Therapy (RMAT) designation for REVASCOR in end-stage HFrEF patients with a left ventricular assist device (LVAD), where FDA stated that the results of the presented studies could support accelerated approval, Mesoblast met with FDA on June 3, 2025 to align on key items for BLA filing. FDA provided formal minutes to the company following the most recent Type B meeting. The minutes confirm alignment with FDA on items regarding chemistry, manufacturing & controls (CMC), potency assays for commercial product release, and proposed design and primary endpoint for the confirmatory trial post-approval. Mesoblast intends to file by the end of the year for accelerated approval in patients with end-stage ischemic HFrEF implanted with an LVAD. Annuncio • Jun 12
Mesoblast Maintains Momentum With FDA on Accelerated Approval Pathway for Revascor® in Ischemic Heart Failure and Label Extension for Ryoncil® in Adults With GvHD Mesoblast provided an update on continued momentum with United States Food and Drug Administration (FDA) regarding both accelerated approval pathway for Revascor (rexlemestrocel-L) in the treatment of patients with ischemic chronic heart failure with reduced ejection fraction (HFrEF) and inflammation, and label extension for Ryoncil (remestemcel-L-rknd) in adults with steroid refractory acute graft versus host disease (SR-aGvHD). In the first week of June, Mesoblast held a Type B meeting with FDA under its Regenerative Medicines Advanced Therapy (RMAT) designation for REVASCOR to discuss components of a potential filing for a Biologics License Application (BLA). There was general alignment on items regarding chemistry, manufacturing & controls (CMC), potency assays for commercial product release, and proposed design and primary endpoint for the confirmatory trial post-approval. The Company will await the final minutes from FDA in order to provide detailed feedback and timelines for potential filing. In early July, Mesoblast has an upcoming meeting with FDA to discuss a pivotal trial of Ryoncil in adults with SR-aGvHD. This trial will be conducted with the NIH-funded Bone Marrow Transplant Clinical Trials Network (BMT-CTN), the objective being to extend the product’s label from children to adults with SR-aGvHD. Ryoncil is the first and only mesenchymal stromal cell product approved by the FDA for any indication.Ryoncil became commercially available for purchase in the United States on March 28, 2025, within one quarter of receiving FDA approval to treat children with SR-aGvHD. More than 20 transplant centers will have been onboarded by the end of the quarter, exceeding the company’s expectations at product launch. Mesoblast has continued to expand coverage for Ryoncil to over 220 million US lives insured by commercial and government payers. To date, 37 of the 51 States provide fee-for-service Medicaid coverage for Ryoncil through Orphan Drug Lists or medical exception /prior authorization (PA) process. The remainder will come online July 1, 2025, with mandatory coverage for all 24 million lives. Annuncio • Apr 29
Mesoblast Limited Appoints Lyn Cobley to Its Board of Directors Mesoblast announced that it has appointed Lyn Cobley to its Board of Directors. Ms Cobley has been a global leader in the financial services industry with over 30 years' experience in senior positions at international and domestic banks. Ms Cobley has served as CEO of Westpac Institutional Bank, Group Treasurer of Commonwealth Bank of Australia, and held senior roles at Barclays Capital and Citibank Limited. Ms Cobley is currently a director of Commonwealth Bank of Australia, is a member of Chief Executive Women, a member of the Macquarie University Council, where she chairs the Finance and Facilities Committee, and an Advisory Member, EXL APAC Advisory Council. She is a former director of the Australian Financial Markets Association. Annuncio • Apr 17
Mesoblast Extends Payment Coverage for Ryoncil to over 100 Million USives Mesoblast announced that it has continued to expand coverage for Ryoncil (remestemcel-L), the first mesenchymal stromal cell (MSC) therapy approved by U.S. Food and Drug Administration (FDA) for any indication, to 104 million US lives insured by government and commercial payers. To date, 37 of the 51 states provide fee-for-service Medicaid coverage for Ryoncil through Orphan Drug Lists or medical exception /prior authorization (PA) process, representing 20 million covered lives, or 80% of the total Medicaid fee-for-service lives covered. The remainder will come on line July 1, 2025 with mandatory coverage for 24 million lives. Many plans do not publish policies for ultra rare diseases such as steroid-refractory acute graft versus host disease (SR-aGvHD) and manage through PA and medical exception, so the number to date is an underestimate of the total commercial coverage already achieved. Annuncio • Apr 03
Mesoblast Provides an Update on Its Plans to Meet with the United States Food and Drug Administration to Discuss Accelerated Approval Pathway for Revascor in Ischemic Heart Failure Mesoblast provided an update on its plans to meet with the United States Food and Drug Administration (FDA) to discuss the accelerated approval pathway for Revascor®? (rexlemestrocel-L) in the treatment of patients with ischemic chronic heart failure with reduced ejection fraction (HFrEF) and inflammation. Mesoblast filed a request for a Type B meeting during the last quarter, in line with previous guidance, and expects to have the meeting during this quarter. The meeting will be held under Mesoblast's Regenerative Medicines Advanced Therapy (RMAT) designation for REVASCOR. In a Type B meeting last year, FDA provided guidance to Mesoblast that the company was eligible to file for accelerated approval of REVASCOR in patients with end-stage HFrEF based on the totality of data across two randomized controlled trials. FDA also guided that a single confirmatory trial in class II/III patients with ischemic HFrEF and inflammation will need to be completed after any accelerated approval is obtained. The key objectives of the meeting are to obtain FDA feedback on relevant chemistry, manufacturing & controls (CMC), alignment on potency assays for commercial product release, and Mesoblast's proposed design and primary endpoint for the confirmatory trial. Annuncio • Mar 27
Mesoblast Limited Announces That Ryoncil is Now Available for Purchase in the United States Mesoblast Limited announced that Ryoncil (remestemcel-L) the first mesenchymal stromal cell (MSC) therapy approved by U.S. Food and Drug Administration (FDA) for any indication, is now available for purchase in the United States. Ryoncil is approved for treatment of pediatric patients 2 months and older, including adolescents and teenagers, with steroid-refractory acute graft versus host disease (SR-aGvHD), a condition with high mortality rates. The recommended dosage of Ryoncil for treatment of pediatric SR-aGvHD is 2 106 MSC/kg body weight per intravenous infusion given twice per week for 4 consecutive weeks. 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, where Ryoncil is now available for ordering. Additional information is available on ryoncil.com, where valuable resources for healthcare providers, patients and caregivers can be found. The company have been engaging with commercial and government insurers to expedite patient access to therapy. To date nine commercial payers have published favorable medical policies representing approximately 37 million covered lives in the United States. The company continue to broaden the commercial payer access landscape on a daily basis. The Company will enter into the National Drug Rebate Agreement with Medicaid and expects to be enrolled in the program in short order. Annuncio • Mar 16
Mesoblast Limited Announces Availability of RYONCIL Product Information in United States Mesoblast Limited announced that pricing information for Ryoncil is now available in all four major drug pricing compendia in the United States. These pricing compendia provide the official source for all information required to order product, including product manufacturing details, National Drug Codes (NDC) for kit purchase, and kit pricing. The pricing compendia listing followed approval and publication by the U.S. Food and Drug Administration (FDA) of the revised Ryoncil label, which includes revisions to Section 16 (How Supplied /Storage and Handling) of the product prescribing information. The revisions included acceptance of eight new Ryoncil kits, each with its own NNDC, based on allocation of product dosage according to patient weight-bands. Treatment centers will be able to order kits tailored to the appropriate dosage for each patient, with pricing applied uniformly, regardless of patient weight. The four compendia comprise Merative Micromedex RED book, First Databank FDB MedKnowledge Drug Pricing, Elsevier Gold Standard Drug Database, and Wolters Kluwer Medi-Span Price Rx. Annuncio • Feb 24
Mesoblast Limited Announces Appointment of Dr. Gregory George to its Board of Directors Mesoblast Limited announced the appointment of Dr. Gregory George MD PhD, Mesoblast’s to its Board of Directors. Dr. George founded and managed the largest privately owned ambulatory surgical center company in the United States, SurgCenter Development. Dr. George brings to the Board his background as a medical scientist with unique operational experience having built a start-up company in the medical field and turning it into a highly-efficient multi-billion-dollar commercial organization. Annuncio • Feb 15
Mesoblast Limited Announces Approve Product Ryoncil® (Remestemcel-L) Mesoblast Limited announced that its recently approved product Ryoncil® (remestemcel-L) is being highlighted at the 2025 Transplantation & Cellular Therapy Tandem Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for Blood and Marrow Transplant Research (CIBMTR), the industry's premier conference taking place this week in Honolulu, HI. Ryoncil® was approved in December 2024 by the United States Food and Drug Administration (FDA) for steroid-refractory acute graft-versus-host disease (SR-aGvHD) in pediatric patients 2 months and older, becoming the first mesenchymal stromal cell (MSC) therapy approved in the U.S. for any indication. Mesoblast has a significant presence at the Tandem meetings including an information hub to engage with and educate healthcare providers in support of Ryoncil's® upcoming launch. a key presentation by Mesoblast executives of scientific and clinical results for Ryoncil®, and hosting two investigator-led advisory panel meetings discussing Ryoncil® for acute SR-aGvHD. Annuncio • Feb 08
Mesoblast Limited to Report First Half, 2025 Results on Feb 26, 2025 Mesoblast Limited announced that they will report first half, 2025 results on Feb 26, 2025 Annuncio • Jan 31
Mesoblast Limited Announces Ryoncil® Commercial Launch Update and Product Pipeline Mesoblast Limited provided an update on the progress of the U.S. commercial launch of Ryoncil® (remestemcel-L) for steroid-refractory acute graft-versus-host disease (SR-aGvHD) in pediatric patients 2 months and older and key upcoming milestones for its late-stage pipeline. 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. Market Opportunity: Steroid-refractory acute GvHD >$1 billion annual market potential. Heart failure with reduced ejection fraction (HFrEF) >$10 billion addressable market potential. Chronic low back pain (CLBP) >$10 billion addressable market potential. Additional potential multi-billion-dollar opportunities from existing and future product pipeline based on existing technology platforms. Product Portfolio: Ryoncil® the only FDA-approved MSC therapy for any indication; lifesaving for pediatric SR-aGvHD. Revascor® 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. 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® launch and product portfolio. Current revenue streams anticipated to expand based on Ryoncil® performance. Continued prudent cash management strategy. Long-term revenue potential from the existing and future pipeline. Annuncio • Jan 24
Mesoblast Limited has completed a Follow-on Equity Offering in the amount of AUD 260 million. Mesoblast Limited has completed a Follow-on Equity Offering in the amount of AUD 260 million.
Security Name: Ordinary Shares
Security Type: Common Stock
Securities Offered: 97,680,000
Price\Range: AUD 2.5
Security Name: Ordinary Shares
Security Type: Common Stock
Securities Offered: 3,095,000
Price\Range: AUD 2.5
Security Name: Ordinary Shares
Security Type: Common Stock
Securities Offered: 3,225,000
Price\Range: AUD 2.5
Transaction Features: Subsequent Direct Listing Annuncio • Dec 21
Mesoblast Limited has completed a Follow-on Equity Offering in the amount of AUD 1.511944 million. Mesoblast Limited has completed a Follow-on Equity Offering in the amount of AUD 1.511944 million.
Security Name: Ordinary Shares
Security Type: Common Stock
Securities Offered: 5,039,814
Price\Range: AUD 0.3
Transaction Features: Subsequent Direct Listing Annuncio • Dec 19
Mesoblast Limited’s RYONCIL® is the First U.S. FDA-Approves Mesenchymal Stromal Cell (MSC) Therapy Mesoblast Limited announced the Food and Drug Administration (FDA) approved Ryoncil (remestemcel-L) as the first mesenchymal stromal cell (MSC) therapy in the United States. RYONCIL is the only MSC therapy approved in the U.S. for any indication, and the only approved therapy for steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 months and older, including adolescents and teenagers. Annually in the United States approximately 10,000 patients undergo an allogeneic bone marrow transplant, 1,500 of whom are children. Approximately 50% develop aGvHD and almost half of those do not respond to steroids, the recognized first-line treatment. In a single-arm multi-center Phase 3 trial of children with SR-aGvHD, 89% of whom had high severity Grade C or Grade D disease, 70% achieved an overall response by Day 28 of treatment with RYONCIL, a measure that predicts survival in aGVHD. RYONCIL treatment was not discontinued or interrupted in any patient for any laboratory abnormality, and the full course was completed without interruption in more than 85% of patients. The full Phase 3 clinical study results are available in Biology of Blood and Marrow Transplantation. Annuncio • Dec 05
FDA Grants Revascor (Rexlemestrocel-L) Regenerative Medicine Advanced Therapy (RMAT) Designation in Children with Congenital Heart Disease Mesoblast Limited announced the United States Food and Drug Administration (FDA) has granted its second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell therapy Revascor (rexlemestrocel-L) Regenerative Medicine Advanced Therapy (RMAT) designation following submission of results from the randomized controlled trial in children with hypoplastic left heart syndrome (HLHS), a potentially life threatening congenital heart condition. Earlier this year, FDA granted REVASCOR both Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) for treatment of children with HLHS. RPDD demonstrates that the disease is serious or life-threatening and the manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents, and that the disease is a rare disease or condition. On FDA approval of a BLA for REVASCOR for the treatment of HLHS, Mesoblast may be eligible to receive a Priority Review Voucher (PRV) that can be redeemed for any subsequent marketing application or may be sold or transferred to a third party. RMAT designations aim to expedite the development of regenerative medicine therapies intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition where preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for the disease or condition. An RMAT designation for rexlemestrocel-L provides all the benefits of Breakthrough and Fast Track designations, including rolling review and eligibility for priority review on filing of a Biologics License Application (BLA). Results from a blinded, randomized, placebo-controlled prospective trial of REVASCOR conducted in the United States in children with HLHS were published in the December 2023 issue of the peer reviewed The Journal of Thoracic and Cardiovascular Surgery Open (JTCVS Open). In the HLHS trial conducted in 19 children, a single intramyocardial administration of REVASCOR at the time of staged surgery resulted in the desired outcome of significantly larger increases in left ventricular (LV) end-systolic and end-diastolic volumes over 12 months compared with controls as measured by 3D echocardiography, (p=0.009 & p=0.020 respectively). These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide. Mesoblast is developing product candidates for distinct indications based on its remestemcel-L andrexlemestrocel-L allogeneic stromal cell technology platforms. Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid refractory acute graft versus host disease, and biologic-resistant inflammatory bowel disease. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast's preclinical and clinical studies, and Mesoblast's research and development programs; Mesoblast's ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast's ability To advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals (including any future decision that the FDA may make on the BLA for remestemcel-L for pediatric patients with SR-aGVHD), manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast's product candidates, if approved; regulatory or public approval of the RMAT designation, the company's approval of the RMAT designation. Annuncio • Dec 03
Mesoblast Limited Announces Publication in the November 2024 Online Issue of the European Journal of Heart Failure Mesoblast Limited announced a key publication in the November 2024 online issue of the prestigious peer-reviewed European Journal of Heart Failure (EJHF), which reports that a single intramyocardial injection of the Company's allogeneic cell therapy Revascor®? (rexlemestrocel-L) results in improved survival in high-risk patients with ischemic heart failure and inflammation. Results from the randomized, controlled DREAM-HF trial in patients with chronic heart failure with reduced injection fraction (HFrEF) identified the control group at highest risk of cardiovascular death as being those with ischemic etiology and inflammation and showed that a single intramy Cardial injection of Mesoblast's mesenchymal precursor cell therapy (MPCs; rexlemestrocel-L) resulted in a sustained reduction in cardiovascular mortality in these high-risk patients. This identifies the target HFrEF population that is responsive to REVASCOR therapy. The newly published results showed that over a mean follow-up of 30 months in the DREAM-HF trial: Factors portending the greatest risk for cardiovascular death in control patients were inflammation (baseline plasma high-sensitivity C-reactive protein =2 mg/L; p=0.003) and ischemic HFrEF etiology (p=0.097), with increased cardiovascular death risk of 61% and 38%, respectively. A single intra-myocardial MPC administration significantly lowered the risk of cardiovascular death in HFrEF patients with inflammation regardless of whether plasma hsCRP or plasma IL-6 was used as inflammatory biomarker by 80% (p=0.003) and 60% (p=0.037) respectively. MPCs reduced 2-point MACE (heart attack or stroke) by 57% (p=0.016) and 3-point MACE (cardiovascular death, heart attack, stroke) by 35% (p=0.049) in patients with ischemic HFrEF (n=303) compared to controls. MPCs reduced 2-point and 3-point MACE by 88% (p=0.005) and 52% (p=0.018) respectively, in patients with ischemic HFrEF and inflammation (n=158) compared to controls. Annuncio • Oct 17
Mesoblast Limited, Annual General Meeting, Nov 15, 2024 Mesoblast Limited, Annual General Meeting, Nov 15, 2024. Location: minterellison, collins arch, level 20, 447 collins street, melbourne victoria 3000 Australia Annuncio • Jul 24
Mesoblast Limited Announces the United States Food and Drug Administration Has Accepted Its Biologics License Application (Bla) Resubmission for Ryoncil® Mesoblast Limited announced that the United States Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) resubmission for Ryoncil® (remestemcel-L) in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD). FDA considers the resubmission to be a complete response and Mesoblast anticipates a decision on or before the FDA’s Prescription Drug User Fee Act (PDUFA) goal date of January 7, 2025. Mesoblast’s resubmission on July 8, 2024 addressed remaining CMC (Chemistry, Manufacturing, and Control) items after being informed by FDA at the end of March 2024 that, following additional consideration, the available clinical data from the Phase 3 study MSB-GVHD001 appears sufficient to support submission of the proposed BLA for remestemcel-L for treatment of pediatric patients with SR-aGVHD. In May 2023, FDA conducted the Pre-License Inspection (PLI) of the manufacturing process for remestemcel-L, and this did not result in the issuance of any Form 483. Mesoblast’s lead product candidate, Ryoncil® (remestemcel-L), is an investigational therapy comprising culture expanded mesenchymal stromal cells derived from the bone marrow of an unrelated donor. It is administered to patients in a series of intravenous infusions. RYONCIL has immunomodulatory properties which counteract the inflammatory processes that are implicated in SR-aGVHD by inhibiting activation and proliferation of effector T cells, down-regulating the production of pro-inflammatory cytokines, and enabling recruitment of anti-inflammatory cells to involved tissues. FDA granted remestemcel-L Fast Track designation, a process to facilitate the development and expedited review of therapies for serious conditions that fill unmet medical needs, and Priority Review designation, which is given to drugs that treat a serious condition and provide a significant improvement in safety or effectiveness over existing treatments. The Phase 3 Study GVHD001/002 was conducted in 54 children (89% Grade C/D) across 20 centers in the US where RYONCIL was used as the first line of treatment for children who failed to respond to steroids for acute GVHD.1 The trial met its pre-specified primary endpoint, Day 28 Overall Response (OR), 70.4% versus 45%, p=0.0003. An overall response at day 28 was highly predictive of improved survival through day 100 (87% compared to 47% in patients that did not achieve day 28 OR p= 0.0001). Compared with a matched control group of pediatric subjects from the contemporaneous database of the Mount Sinai Acute GVHD International Consortium (MAGIC) treated with best available therapy, treatment with Ryoncil achieved higher Day 28 OR (70% vs 43%) and higher Day 100 survival (74% vs 57%). A propensity-matched study of outcomes in 25 children from Mesoblast’s Phase 3 trial and 27 control children who received best available treatment, including ruxolitinib, from the MAGIC database showed that 67% of high-risk children (MAP scores >0.29) who received Ryoncil achieved a Day 28 overall response and were alive after 180 days compared to just 10% in both categories in the MAGIC group. In addition, results of a 4-year survival study performed by the Center for International Blood and Marrow Transplant Research (CIBMTR) on 51 evaluable patients with SR-aGVHD who were enrolled in the Phase 3 trial, demonstrated durability of the survival benefits, with 67% survival at 6 months, 63% survival at 1 year, 51% at 2 years, and 49% survival through 4 years in children with expected 2 year survival of just 25-38% using best available therapy. Acute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, including about 20% in pediatric patients.5,6 SR-aGVHD is associated with mortality as high as 90% and significant extended hospital stay costs.7,8 There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD. Survival outcomes have not improved over the past two decades for children or adults with the most severe forms of SR-aGVHD.2,9-10 The lack of any approved treatments for children under 12 means that there is an urgent need for a therapy that improves the dismal survival outcomes in children. Annuncio • Jul 22
Mesoblast Limited Announces Patient Enrollment Commences in Pivotal Phase 3 Trial of Rexlemestrocel-L for Chronic Low Back Pain Mesoblast Limited announced that the confirmatory Phase 3 trial of its allogeneic, immunoselected, and industrially manufactured stromal cell product rexlemestrocel-L in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease of less than five years duration has commenced enrollment at multiple sites across the United States. The United States Food and Drug Administration (FDA) has previously confirmed alignment with Mesoblast on the design of the 300-patient randomized, placebo-controlled trial and the 12-month primary endpoint of pain reduction as an approvable indication. Key secondary measures include improvement in quality of life, function, and reduced opioid usage. FDA has designated rexlemestrocel-L a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of chronic low back pain. RMAT designation provides all the benefits of Breakthrough and Fast Track designations, including rolling review and eligibility for priority review on filing of a Biologics License Application (BLA). Annuncio • Jul 10
Mesoblast Limited Resources Biologics Licence Application (BLA) with United States Food & Drug Administration (FDA) for Approval of Ryoncil in the Treatment of Children with SR-aGVHD Mesoblast Limited announced it has resubmitted its BLA for approval of Ryoncil (remestemcel-L) in the treatment of children with SR-aGVHD. The filing comes after Mesoblast was informed by FDA at the end of March that, following additional consideration, the available clinical data from the Phase 3 study MSB-GVHD001 appears sufficient to support submission of the proposed BLA for remestemcel-L for treatment of pediatric patients with SR- aGVHD. As a result, the filing addresses remaining CMC (Chemistry, Manufacturing, and Control) items. FDA granted remestemcel-L Fast Track designation, a process to facilitate the development and expedited review of therapies for serious conditions that fill unmet medical needs, and Priority Review designation, which is given to drugs that treat a serious condition and provide a significant improvement in safety or effectiveness over existing treatments. The BLA resubmission upon acceptance is expected to have a review period of between two and six months from receipt. In addition, results of a 4-year survival study performed by the Center for International Blood and Marrow Transplant Research (CIBMTR) on 51 evaluable patients with SR-aGVHD who were enrolled in the Phase 3 trial, demonstrated durability of the survival benefits, with 67% survival at 6 months, 63% Exhibit 99.1. Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, including about 20% in pediatric patients.5,6 SR-aGVHD is associated with mortality as high as 90% and significant extended hospital stay costs.7,8 There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD". These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide. Mesoblast is developing product candidates for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid refractory acute graft versus host disease, and biologic-resistant inflammatory bowel disease. Annuncio • Jul 01
Mesoblast to File Biologics License Application for Ryoncil FDA Approval Next Week Mesoblast Limited confirmed that it will file its Biologics License Application (BLA) for approval of Ryoncil (remestemcel-L) in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD) with the U.S. Food and Drug Administration (FDA) next week. Annuncio • Mar 27
United States Food & Drug Administration Notifies Mesoblast Limited of Available Clinical Data from Phase 3 Trial Appear Sufficient to Support BLA Submission for Remestemcel-L in Children with Steroid-Refractory Acute Graft Versus Host Disease Mesoblast Limited announced that U.S. FDA has informed the company that following additional consideration the available clinical data from its Phase 3 study MSB-GVHD001 appears sufficient to support submission of the proposed Biologics License Application (BLA) for remestemcel-L for treatment of pediatric patients with steroid-refractory acute graft versus host disease (SR-aGVHD). Mesoblast intends to file the resubmission during the next quarter, seeking to address all remaining product characterization issues. Annuncio • Mar 23
Mesoblast Limited has completed a Follow-on Equity Offering in the amount of AUD 0.93 million. Mesoblast Limited has completed a Follow-on Equity Offering in the amount of AUD 0.93 million.
Security Name: Ordinary Shares
Security Type: Common Stock
Securities Offered: 3,100,000
Price\Range: AUD 0.3 Annuncio • Mar 14
Mesoblast Limited has completed a Follow-on Equity Offering in the amount of AUD 97.041224 million. Mesoblast Limited has completed a Follow-on Equity Offering in the amount of AUD 97.041224 million.
Security Name: Ordinary Shares
Security Type: Common Stock
Securities Offered: 119,855,720
Price\Range: AUD 0.3
Security Name: Ordinary Shares
Security Type: Common Stock
Securities Offered: 63,404,141
Price\Range: AUD 0.3
Discount Per Security: AUD 0.012
Security Name: Ordinary Shares
Security Type: Common Stock
Securities Offered: 17,877,551
Price\Range: AUD 0.3
Discount Per Security: AUD 0.012
Security Name: Ordinary Shares
Security Type: Common Stock
Securities Offered: 122,333,333
Price\Range: AUD 0.3
Transaction Features: Rights Offering Annuncio • Mar 11
Mesoblast Limited Announces United States Food & Drug Administration Supports Accelerated Approval Pathway for Rexlemestrocel-L in End-Stage Heart Failure Patients with A Left Ventricular Assist Device Mesoblast Limited announced that U.S. FDA supports an accelerated approval pathway for rexlemestrocel-L, Mesoblast's allogeneic mesenchymal precursor cell (MPC) product, in patients with end-stage ischemic heart failure with reduced ejection fraction (HFrEF) and a left ventricular assist device (LVAD). FDA provided this feedback in formal minutes to the company following the Type B meeting held with FDA on February 21, 2024 for rexmestrocel-L (Revascor®?) under the existing Regenerative Medicine Advanced Therapy (RMAT) designation. Every year in the United States over 100,000 patients progress to end-stage HFrEF. Only 4.9% of ischemic patients treated with a single administration of rexlemestrocel- Ltd. died from month 2 through month 12, as compared with 26.9% of ische Micmic controls, an 82% reduction (p = 0.02). In feedback provided to Mesoblast regarding potential pathways to licensure for rexlemestrocel -L, FDA's comments indicated that the presented results may support a reasonable likelihood of clinical benefit of MPCs against mortality in LVAD patients, consistent with the criteria for accelerated approval. Mesoblast intends to request a pre-BLA meeting with FDA to discuss data presentation, timing and FDA expectations for an accelerated approval filing in end-stage ischemic HFrEF patients with LVAD implantation. REVASCOR is an allogeneic preparation of immunoselected and culture-expanded mesenchymal precursor cells (MPC) and is being developed as an immunomodulatory therapy to address the high degree of inflammation in the heart and in the circulation that is present across the spectrum of heart failure and reduced ejection fraction (H FrEF) patients, from New York Heart Association (NYHA) class II through end-stage CHF, in order to reduce the high rate of major cardiac events and complications. This investigational therapy has been trialled in two large placebo-controlled randomized studies in patients with CHF, a 565-patient trial in NYHA class II/III HFrEF patients and a 159-patient trial in end-stage HFrEF patients implanted with a left ventricular assist device ("LVAD"). Rexlemestrocel-L has US Food and Drug Administration (FDA) Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations for patients with end-stage HFrEF implanted with an LVAD. Chronic heart failure (CHF) is characterized by poor heart function resulting in insufficient blood flow to the body's vital organs and extremities. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide. Mesoblast is developing product candidates for distinct indications based on its remestemcel-L andrexlemestrocel-L allogeneic stromal cell technology platforms. Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid refractory acute graft versus host disease, biologic-resistant inflammatory bowel disease, and acute respiratory distress syndrome. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast's preclinical and clinical studies, and Mesoblast's research and development programs; Mesoblast's ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast's ability To advance its manufacturing capabilities; the timing or likelihood of patients with LVAD's ability to advance its manufacturing capabilities. Annuncio • Jan 19
United States Food & Drug Administration Grants Mesoblast Rare Pediatric Disease Designation for Revascor®? (Rexlemestrocel-L) in Children with Congenital Heart Disease Mesoblast Limited announced that the United States Food and Drug Administration (FDA) has granted its allogeneic cell therapy Revascor® (rexlemestrocel-L) a Rare Pediatric Disease (RPD) Designation following submission of results from the randomized controlled trial in children with hypoplastic left heart syndrome (HLHS), a potentially life threatening congenital heart condition. RPD Designation is granted by the FDA for certain serious or life-threatening diseases which primarily affect children. On FDA approval of a Biologics Licensing Application (BLA) for REVASCOR for the treatment of HLHS, Mesoblast may be eligible to receive a Priority Review Voucher (PRV) that can be redeemed for any subsequent marketing application or may be sold or transferred to a third party. Results from a blinded, randomized, placebo-controlled prospective trial of REVASCOR conducted in the United States in children with HLHS were published in the December 2023 issue of the peer reviewed The Journal of Thoracic and Cardiovascular Surgery Open (JTCVS Open). In the HLHS trial conducted in 19 children, a single intramyocardial administration of REVASCOR at the time of staged surgery resulted in the desired outcome of significantly larger increases in left ventricular (LV) end-systolic and end-diastolic volumes over 12 months compared with controls as measured by 3D echocardiography, (p=0.009 & p=0.020 respectively). These changes are indicative of clinically important growth of the small left ventricle, facilitating the ability to have a successful surgical correction, known as full biventricular (BiV) conversion, which allows for a normal two ventricle circulation with the surgically repaired left ventricle taking over circulatory supportto the body. Without full BiV conversion the right heart chamber is under excessive strain with increased risk of heart failure and death. Annuncio • Oct 30
Mesoblast Limited, Annual General Meeting, Nov 28, 2023 Mesoblast Limited, Annual General Meeting, Nov 28, 2023, at 12:00 AUS Eastern Standard Time. Location: MinterEllison Collins Arch, Level 20 447 Collins Street Melbourne Victoria 3000 Melbourne Australia Agenda: To discuss Receipt and Consideration of Financial Statements and Reports; to discuss Adoption of the Remuneration Report; to discuss Amendment to the Constitution; to discuss Re-election of Directors; to discuss Approval of Proposed Issue of Options to Chief Executive Officer, Dr. Silviu Itescu; to discuss Approval of Proposed Issue of Options to Chief Medical Officer, Dr. Eric Rose; to discuss Approval of Proposed Issue of Options to Non-Executive Directors; to discuss Approval of Proposed Issue of Options to Director, Dr. Philip Krause; and to discuss Ratification of Issue of Securities to Institutional Investors. Annuncio • Sep 27
Jane Bell AM Appointed Chair of Mesoblast Audit and Risk Committee Mesoblast Limited announced that independent Director Jane Bell AM has been appointed Chair of the Mesoblast Board Audit and Risk Committee. Ms Bell joined the Board in August 2022, and is a banking and finance lawyer with 30 years of corporate finance expertise focussing on international investment transactions in the United States, Canada, Australia and the United Kingdom, including funds management, mergers, acquisitions, and divestments. The Board thanked retiring Director and Chair of Audit and Risk Committee Mr. Michael Spooner for his many years of dedicated service and contributions, and wished him well in his future endeavours. Ms Bell is currently Chair of the Audit and Risk Committee of publicly-listed biotechnology company Amplia Therapeutics, and serves as Chair of the Audit Committee and Deputy Chair of Monash Health, Australia’s largest and most diverse public health service delivering more than 3.46 million episodes of care across an extensive network of hospitals, rehabilitation, aged care, community health and mental health facilities. From 2014 until 2021 she was a director of U Ethical, Australia’s first ethical funds manager with over $1.2B of funds under management, and a member of both its Audit and Investment Committees. She has been a former Chair of Melbourne Health as well as of Advisory Groups for the Royal Australian and New Zealand College of Obstetricians and Melbourne Genomics Health Alliance, and has been a director of Hudson Institute of Medical Research and Chair of its Intellectual Property and Commercialization Committee. Ms Bell holds a Master of Laws from King’s College (London), Bachelor of Laws University of Melbourne, and Bachelor of Economics Monash University. In 2023 Ms Bell was appointed a Member of the Order of Australia (AM) for her significant service to governance in the medical research, healthcare and not-for-profit sectors. Annuncio • Sep 22
Mesoblast Limited Provides an Update on the Path to Approval for Its Lead-Product Candidate Remestemcel-L in the Treatment of Pediatric and Adult Steroid-Refractory Acute Graft Versus Host Disease Mesoblast Limited provided an update on the path to approval for its lead-product candidate remestemcel-L in the treatment of pediatric and adult steroid-refractory acute graft versus host disease (SR-aGVHD), following a Type A meeting held with the United States Food and Drug Administration (FDA). FDA clarified that the key remaining issue for pediatric approval is providing further evidence that the potency assay will assure the consistent efficacy of commercial product. The key outcomes of the meeting and Mesoblast next steps are: Mesoblast summarised the large body of existing clinical data with the improved RYONCIL® product version of remestemcel-L, manufactured after 2008 using the current process inspected by FDA, for potential approval in children. Mesoblast presented clinical data indicating that RYONCIL was associated with much higher survival of children with SR-aGVHD in the Expanded Access Protocol (EAP) compared with the earlier Prochymal version manufactured before 2008. Mesoblast presented consistently high survival in patients treated in RYONCIL, in children with SR-aGVHD in the EAP between 2008-2015, in the Phase 3 trial MSB-GVHD001 between 2015-2018, and in the Emergency Investigational New Drug (EIND) protocol from 2015-2023. FDA noted that the lack of a suitable potency assay for the RYONCIL product used during the Phase 3 trial MSB-GVHD001 for the pediatric acute GVHD indication has prevented the trial from being considered an adequate study for the purpose of demonstrating substantial evidence of effectiveness required for a marketing approval. Mesoblast intends to generate in the coming months new potency assay data for RYONCIL showing that the product used during pediatric Phase 3 trial MSB-GVHD001 was standardized as to its identity, strength, quality, purity, and dosage form to give significance to the results of the investigation as described in the Code of Federal Regulations for adequate and well-controlled studies (21 CFR 314.126), and that commercial batches made for the pediatric indication will meet the same standard. Mesoblast believes these additional data will provide a link between the RYONCIL product that was used in the Phase 3 trial MSB-GVHD001 for the pediatric indication to the RYONCIL product which will be used in a future trial for the adult indication. To address the adult indication, Mesoblast proposed an externally controlled single-arm registration trial design in adults and children over age 12 with SR-aGVHD who have failed both steroids and a second line agent, such as ruxolitinib, to be underpinned by a suitable potency assay. Mesoblast intends to perform this trial in partnership with investigators at the Blood and Marrow Clinical Trials Network (BM CTN), a body responsible for approximately 80% of all US transplants, with the costs expected to be covered by previously announced reductions in operational spend. FDA indicated its willingness to consider Mesoblast’s proposed registrational trial design in adults, subject to agreement on the suitability of the potency assay for the product to be used in adults. Mesoblast expects to receive the formal minutes of the Type A meeting within three weeks. Annuncio • Aug 04
Mesoblast Limited Receives Complete Response from U.S. Food and Drug Administration for Biologics License Application for Steroid-Refractory Acute Graft Versus Host Disease in Children Mesoblast Limited announced that the US Food and Drug Administration (FDA) has provided a complete response to its Biologics License Application (BLA) resubmission for remestemcel-L for the treatment of pediatric steroid-refractory acute graft versus host disease (SR-aGVHD) and requires more data to support marketing approval. To obtain the data required, Mesoblast will conduct a targeted, controlled study in the highest-risk adults with the greatest mortality. This adult study is in line with its overall commercial strategy, which envisioned a sequenced progression from pediatric to adult SR-aGVHD indications. Adults comprise 80% of the SR-aGVHD market. Mesoblast intends to enroll adult patients at highest mortality risk with SR-aGVHD where existing therapy has not improved outcomes and 90-day survival remains as low as 20-30%. Mesoblast has generated pilot data through its emergency IND program in adults showing a survival benefit with remestemcel-L in this target population. In line with its overall commercial strategy to expand into the adult SR-aGVHD indication, Mesoblast has already been working with leading investigators at various US centers of excellence to establish the adult follow-on study protocol, potentially utilizing established clinical trials networks. The company will seek alignment with FDA on the trial design for the adult study at a Type A meeting within 45 days. Prior to the resubmission, FDA guided Mesoblast to resolve outstanding chemistry, manufacturing and controls (CMC) issues before initiating any additional clinical trial. FDA completed the Pre-License Inspection (PLI) of the manufacturing facility, did not issue any Form 483, and found no objectionable conditions. In addition, FDA acknowledged in the resubmission review that changes implemented appear to improve assay performance relative to the original version of the assay used in the pediatric Phase 3 trial. Mesoblast has successfully met the pre-specified primary endpoint, prospectively agreed with FDA, of a single-arm Phase 3 trial in 54 children with SR-aGvHD. While the Oncologic Drugs Advisory Committee of FDA in August 2020 voted 9:1 in favor of remestemcel-L’s efficacy in a pediatric patient population, in September 2020 FDA recommended further steps be undertaken to obtain approval. The BLA resubmission of January 2023 included long-term follow-up data from the Phase 3 trial by the Center for International Blood and Marrow Transplant Research (CIBMTR) showing 50% survival through more than 4 years of follow-up for remestemcel-L treated patients in the Phase 3 trial for whom less than 20% survival at two years was expected based on disease severity. The resubmission also included a post-hoc propensity matched study showing 6 month survival was 67% with remestemcel-L vs 10% with other unapproved therapies in highest-risk patients as identified using the Mount Sinai Acute GVHD International Consortium (MAGIC). These pediatric data provide further support for use of remestemcel-L in the proposed study in high-risk adults with SR-aGVHD. Annuncio • Jun 05
Mesoblast Limited Appoints Dr. Philip R. Krause, Member of the Board of Directors, to Formal Strategic Advisory Role Mesoblast Limited announced that it has appointed Dr. Philip R. Krause, a member of the Board of Directors, to a formal strategic advisory role. Dr. Krause was formerly Deputy Director of the Office of Vaccines Research and Review at the US Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) and is an internationally recognized expert on development and regulatory aspects of biologic products. Dr. Krause will advise the company on its regulatory strategies related to broadening out the remestemcel-L platform, including the ground-breaking treatment for steroid-refractory acute graft versus host disease (SR-aGVHD), inflammatory bowel disease and inflammatory lung disease, and the second-generation platform rexlemestrocel-L, including the Phase 3 product candidates for discogenic chronic lower back pain and inflammatory heart failure. Annuncio • Feb 17
Remestemcel-L Clinical and Potency Studies Presented At Premier Meeting of Transplantation Research and Therapy Mesoblast Limited announced that two studies on the remestemcel-L development program for the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD) were selected by peerreview to be presented at the 2023 Tandem Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and the Center for Blood and Marrow Transplant Research (CIBMTR), taking place February 15-19 in Orlando, Florida. The data from both studies formed key components of Mesoblast's recent resubmission of its remestemcel-L Biologics License Application (BLA) to U.S. Food and Drug Administration (FDA) for children with SR-aGVHD. Survival outcomes have not improved over the past two decades for the most severe forms of SR-aGVHD, a life-threatening complication of an allogeneic bone marrow transplant following treatment for blood cancers and other conditions.(1-) (3) The lack of any approved treatments for children under 12 means that there is an urgent need for a therapy that improves the dismal survival outcomes. If remestemcel-L receives FDA approval, it will be the first allogeneic "off-the-shelf" cellular medicine to be approved in the United States, and the first therapy for children under 12 years old with SR-aGVHD. Annuncio • Feb 09
Mesoblast Limited Announces FDA Grants Regenerative Medicine Advanced Therapy Designation for Rexlemestrocel-L in Chronic Low Back Pain Mesoblast Limited announced that the United States Food and Drug Administration’s Office of Tissues and Advanced Therapies has granted Regenerative Medicine Advanced Therapy designation for rexlemestrocel-L in the treatment of chronic low back pain associated with disc degeneration, in combination with hyaluronic acid as delivery agent for injection into the lumbar disc. RMAT designations aim to expedite the development of regenerative medicine therapies intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition where preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for the disease or condition. An RMAT designation for rexlemestrocel-L provides all the benefits of Breakthrough and Fast Track designations, including rolling review and eligibility for priority review on filing of a Biologics License Application (BLA). There is a significant need for a safe, effective, and durable opioid-sparing treatment in patients with CLBP associated with degenerative disc disease. Data from the completed 404-patient randomized, blinded placebo-controlled Phase 3 trial of rexlemestrocel-L combined with HA formed the basis for FDA’s determination that Mesoblast’s allogeneic cell therapy has the potential to address unmet medical needs for patients suffering from CLBP due to disc degeneration. Results from the trial showed that: A single injection of rexlemestrocel-L+HA into the lumbar disc resulted in significant reduction in pain compared with saline control at 12 and 24 months across all subjects (n=404): The reduction in low back pain observed in all subjects was substantially enhanced in the pre-specified population with CLBP of shorter duration than the median of 68 months for the study (n=194). Significant improvement in function and quality of life measures were observed in subjects with a shorter duration of CLBP at baseline. Pain reduction through 36 months was seen in the subset of patients using opioids at baseline (n=168) with the rexlemestrocel-L+HA group having substantially greater reduction at all time points compared with saline controls. Among patients on opioids at baseline, despite instructions to maintain existing therapies throughout the trial, at 36 months 28% who received rexlemestrocel-L + HA were not taking an opioid compared with 8% of saline treated controls. Annuncio • Feb 01
Mesoblast Limited Resubmits Biologic License Application to FDA for Remestemcel-L in Children With Steroid-Refractory Acute Graft Versus Host Disease (Sr-aGVHD) Mesoblast Limited announced it has resubmitted to the U.S. Food and Drug Administration (FDA) its Biologics License Application (BLA) for approval of remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD). Survival outcomes have not improved over the past two decades for the most severe forms of SR-aGVHD, a life-threatening complication of an allogeneic bone marrow transplant following treatment for blood cancers and other conditions.1-3 The lack of any approved treatments for children under 12 means that there is an urgent need for a therapy that improves the dismal survival outcomes. If remestemcel-L receives FDA approval, it will be the first allogeneic “off-the-shelf” cellular medicine to be approved in the United States, and the first therapy for children under 12 years old with SR-aGVHD. The resubmission contains substantial new information as required by FDA in the Complete Response Letter (CRL) received in September 2020 to the BLA for remestemcel-L. Mesoblast has maintained an active dialogue with FDA since receiving the CRL and in October 2022 provided a high-level synopsis of the substantial new information under its Investigational New Drug (IND) application for remestemcel-L. FDA granted remestemcel-L Fast Track designation, a process to facilitate the development and expedited review of therapies for serious conditions that fill unmet medical needs, and Priority Review designation, which is given to drugs that treat a serious condition and provide a significant improvement in safety or effectiveness over existing treatments. The BLA resubmission will have a review period up to six months from filing upon acceptance by FDA. Mesoblast has responded to the CRL and the further guidance it has received from the FDA and has generated and provided new data and analyses in the resubmission which company believe provide substantial evidence of remestemcel-L's effectiveness in pediatric SR-aGVHD. Specifically, the resubmission contains the following: new long-term survival data of children enrolled in the Phase 3 trial showing durability of treatment effect through at least four years, new data showing remestemcel-L’s treatment benefit in high-risk disease activity and on survival in propensity-matched studies of children in the Phase 3 trial and controls stratified by validated biomarkers for high-risk disease, new analyses of data obtained prospectively showing that the validated potency assay which was in place and used to release product for the 54-patient Phase 3 clinical trial measures a key product attribute which reflects the primary mechanism of action of remestemcel-L in children with SR-aGVHD, correlates with the product’s in vivo bioactivity, and predicts overall survival outcomes, new analyses of data obtained prospectively relating manufacturing changes during product development prior to Phase 3 to progressive increases in potency and to improved survival outcomes in larger studies of remestemcel-L under expanded access in children with SR-aGVHD, new data showing that the validated potency assay has low variability and can adequately demonstrate manufacturing consistency and reproducibility, and establishment of a new specification for release of commercial product based on extensive clinical data which provides assurance that future batches of remestemcel-L will have attributes supportive of expected survival outcomes.