Pediatric Multisystem Inflammatory Disease, COVID-19 Related

NEW YORK, April 29, 2025 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided highlights of its recent activities for the third quarter ended March 31, 2025. “We were very pleased to have made Ryoncil® (remestemcel-L) commercially available to treat children with acute GVHD within one quarter of receiving FDA approval as the first mesenchymal stromal cell (MSC) therapy approved in the US for any indication,” said Dr. Silviu Itescu, CEO of Mesoblast. “With our strong cash position we are well placed to expand Ryoncil® indications to other serious and life-threatening pediatric inflammatory diseases, and to adults with acute GvHD.” FINANCIAL HIGHLIGHTS Net operating cash spend for the quarter was US$12.7 million.Cash on hand at the end of the quarter was US$182 million (A$290 million)1. OPERATIONAL HIGHLIGHTS Ryoncil® (remestemcel-L) U.S. Launch for Steroid-Refractory Acute Graft Versus Host Disease Ryoncil® became commercially available for purchase in the United States on March 28, 2025, with Federal Medicaid coverage, and to date 15 infusion kits have been purchased for patients to start or continue their treatment course.Ryoncil® infusion kits are purchased and distributed by Cencora to enable the efficient and secure delivery of cryopreserved product to U.S. treatment centers, either directly or via a specialty pharmacy option.To date, ten priority transplant centers have been fully onboarded, five of whom have enrolled patients through the MyMesoblast™ hub.Mesoblast anticipates onboarding an additional ten priority transplant centers in the current quarter.The full team of nine key account managers (KAMs) commenced activities in the last week of April. The KAMs will accelerate onboarding of the remaining 35 priority transplant centers, accounting for 80% of U.S. pediatric transplants, and will drive the business to provide on the ground engagement with healthcare providers and administrators.Mesoblast has continued to expand coverage for Ryoncil® to over 104 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 44 million lives.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. Revascor® (rexlemestrocel-L) for Chronic Heart Failure with Reduced Ejection Fraction (HFrEF) and Persistent Inflammation Mesoblast has a Type B meeting with FDA scheduled for this quarter 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. 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.In November 2024 a publication in the prestigious peer-reviewed European Journal of Heart Failure (EJHF) reported that a single intramyocardial injection of REVASCOR results in improved survival in high-risk NYHA Class II/III patients with ischemic heart failure and inflammation.2 This identifies the HFrEF population that is responsive to REVASCOR and will be the target of a confirmatory trial after accelerated approval, if received. Rexlemestrocel-L for Chronic Low Back Pain associated with Degenerative Disc Disease – Phase 3 Program The confirmatory Phase 3 trial of Mesoblast’s second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell product candidate rexlemestrocel-L in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration is actively enrolling and treating patients 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 RMAT for the treatment of chronic low back pain. Corporate During the period, Mesoblast successfully completed a global private placement primarily to existing major US, UK, and Australian shareholders raising A$260 million (US$161 million).Strengthened Board of Directors with appointment of Dr. Gregory George and Ms Lyn Cobley.Mesoblast was added to the S&P Dow Jones Indices’ S&P/ASX 200 Index effective March 6, 2025, on the Australian Stock Exchange (ASX). Other Fees to Non-Executive Directors were US$50,306, consulting payments to Non-Executive Directors were Nil and salary payments to full-time Executive Directors were US$223,092, detailed in Item 6 of the Appendix 4C cash flow report for the quarter.3 From August 2023 to July 2025, our Non-Executive Directors have voluntarily reduced cash payment of their fees by 50% and Executive Directors (our Chief Executive and Chief Medical Officers) reduced their base salaries by 30%, in lieu of accepting equity-based incentives. A copy of the Appendix 4C – Quarterly Cash Flow Report for the third quarter FY2025 is available on the investor page of the company’s website www.mesoblast.com. About Mesoblast Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. 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) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 months and older is the first FDA-approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com. 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. About Mesoblast intellectual property: 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 2041 in all major markets. About Mesoblast manufacturing: 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). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast References / Footnotes Translated at 1A$:0.6413US$ being the March 31, 2025 rate as reported by the Reserve Bank of Australia.Perin EC. Et al. Mesenchymal precursor cells reduce mortality and major morbidity in ischaemic heart failure with inflammation: DREAM-HF. Eur J Heart Fail 2024. https://doi.org/10.1002/ejhf.3522As required by ASX listing rule 4.7 and reported in Item 6 of the Appendix 4C, reported are the aggregated total payments to related parties being Executive Directors and Non-Executive Directors. Forward-Looking StatementsThis press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. 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, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s RYONCIL for pediatric SR-aGVHD and any other product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise. Release authorized by the Chief Executive. For more information, please contact: Corporate Communications / InvestorsPaul HughesT: +61 3 9639 6036 Media – Global Allison WorldwideEmma NealT: +1 603 545 4843E: emma.neal@allisonworldwide.com Media – AustraliaBlueDot MediaSteve DabkowskiT: +61 419 880 486E: steve@bluedot.net.au

NEW YORK--( BUSINESS WIRE )--PIF Partners announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its proprietary investigational therapeutic 101-PGC-005 (‘005) for the treatment of systemic juvenile idiopathic arthritis (sJIA) flares. ‘005 is a Type IA prodrug of dexamethasone that targets CD206 + macrophages. Currently, ‘005 is in Phase 3 clinical trials in 9 centers in India for the treatment of ARDS induced by COVID-19, registered under CTRI/2024/01/061531. “We are thrilled the FDA has recognized the potential of ‘005 to address the significant unmet medical needs of children suffering from sJIA,” said Alec Goldberg, CEO of PIF Partners. “This designation is a pivotal milestone that reinforces our commitment to developing innovative therapies for rare pediatric inflammatory diseases. By targeting activated macrophages with the most powerful anti-inflammatory compounds, we enhance efficacy and reduce toxicity. Critically, we have demonstrated no suppression of the HPA system, thus supplementing rather than replacing the natural anti-inflammatory response. We are eager to advance our clinical program and expand our research into other orphan diseases characterized by systemic inflammation.” The FDA awards RPDDs to promote the development of new drugs and biologics for serious or life-threatening diseases that primarily affect children under 18 years old and impact fewer than 200,000 people in the United States. This designation makes PIF Partners eligible to receive a Priority Review Voucher (PRV) upon approval of ‘005. A PRV can be redeemed to obtain priority review for a subsequent marketing application, accelerating the review process from the standard 10 months to six months. About sJIA sJIA is a rare and severe autoinflammatory disease affecting children under 16. Unlike other forms of juvenile arthritis, sJIA is characterized by systemic symptoms such as high spiking fevers, rash, enlargement of the liver and spleen, and inflammation of the lining of the heart and lungs (serositis), in addition to persistent joint inflammation. sJIA accounts for approximately 10-20% of all cases of juvenile idiopathic arthritis. The disease poses significant challenges, including chronic pain, growth retardation, joint damage, and potential life-threatening complications like macrophage activation syndrome (MAS). Current treatment options are limited and often involve long-term use of corticosteroids and immunosuppressive drugs, which can have serious side effects. There is a pressing need for new therapies that can effectively manage both the systemic and joint-related symptoms with improved safety profiles. About PIF Partners PIF Partners is a holding company with exclusive rights to ‘005 and other therapeutics using the underlying macrophage targeting platform. The company is seeking a partner to rapidly progress the development and commercialization of novel therapies for systemic juvenile idiopathic arthritis (sJIA) and other rare diseases characterized by systemic inflammation. Focused on addressing critical gaps in treatment, the company is advancing a pipeline of exclusively licensed therapeutic candidates targeting CD206 + macrophages. For more information, please visit pifpartners.com.

PHILADELPHIA, Oct. 22, 2024 /PRNewswire/ -- Researchers from Children's Hospital of Philadelphia (CHOP) demonstrated how rapid testing of cytokine levels can help distinguish different diseases with similar symptom profiles. Nearly 30% of clinicians changed how they managed disease in children after receiving cytokine level information, showing the potential for this testing to impact clinical practice. The findings were recently published in the Journal of Allergy and Clinical Immunology. Continue Reading Michele P. Lambert, MD, MSTR, Medical Director of the Special Coagulation Laboratory and attending physician in the Division of Hematology at Children's Hospital of Philadelphia During the COVID-19 pandemic, doctors began diagnosing patients with multisystem inflammatory syndrome in children (MIS-C), a condition that occurs weeks after infection and results from inflammation throughout the body. However, the initial symptoms – such as high fever and changes in blood pressure – are very similar to those of an acute, severe COVID-19 infection, so doctors needed to be able to distinguish between the two conditions, since each one requires a very different course of treatment. Distinguishing between MIS-C and COVID-19 is just one example of the value of serum cytokine panels. Cytokines are signaling proteins that regulate inflammation and coordinate immune responses. Researchers and clinical laboratories have worked hard to make sure that cytokine panels can be analyzed more quickly to inform the course of care – with most results returned after 5 to 7 days – yet understanding the value of cytokines in real time, within 24 hours, as biomarkers for different diseases is a relatively new concept. "We wanted to see what clinicians were able to do with this information if it was returned rapidly and whether results would change clinical decision making or muddy the waters and create confusion," said senior study author Michele P. Lambert, MD, Medical Director of the Special Coagulation Laboratory and an attending physician in the Division of Hematology at CHOP. "We wanted to see potential changes included adding additional antimicrobials or an immune suppressant in response to the results in the panel." The study found that the diagnosis was changed or informed in 18.8% of reviewed cases and management of the patient was changed or informed in 29.8% of cases. Even in the remaining 70.2% of cases where patient care did not change, the results provided important information confirming doctors were providing a correct course of treatment. Another key finding in the study was that specific disease states had clear cytokine patterns. For example, higher levels of interferon gamma (IFNg) and lower levels of interleukins 6 and 8 (IL-6 and IL-8) suggested a more robust inflammatory response, such as cytokine storm or familial hemophagocytic lymphohistiocystosis, a rare condition where the immune system attacks one's own body if left untreated. However, a reverse in those levels was more indicative of bacterial or viral sepsis. "The results of a cytokine panel could potentially save lives: supporting antibiotics or immune suppression, but only if we have the results fast enough," Lambert said. "The evolution of our response time and our understanding of the value of these biomarkers demonstrates how important it is for clinicians and clinical laboratories to collaborate to provide the best care possible for patients." Gallo et al, "Serum Cytokine Panels in Pediatric Clinical Practice." J Allergy Clin Immunol. Online September 18, 2024. DOI: 10.1016/j.jaci.2024.08.030 CONTACT: Amanda DiPaolo Bradley, [email protected] SOURCE Children's Hospital of Philadelphia WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED