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Last update 17 Apr 2025

Rexlemestrocel-L

Last update 17 Apr 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Mesenchymal stem cell therapy

Synonyms

Allogeneic mesenchymal precursor cells Mesoblast, Allogeneic STRO3 immunoselected bone marrowderived mesenchymal precursor cells (MPCs) Mesoblast, Mesenchymal stem cell therapy Mesoblast

+ [3]

Target-

Action-

Mechanism

Cell replacements

Therapeutic Areas

Nervous System DiseasesSkin and Musculoskeletal DiseasesOther Diseases

Active Indication

Intervertebral Disc DegenerationLow Back Pain

Inactive Indication

Acute myocardial infarctionAnterior Cruciate Ligament InjuriesChronic heart failure+ [11]

Originator Organization

Institute of Medical & Veterinary Science

Active Organization

Mesoblast Ltd.

Inactive Organization

Teva Pharmaceutical Industries Ltd.

Mesoblast, Inc.

Drug Highest PhasePhase 3

First Approval Date-

RegulationOrphan Drug (United States), Rare Pediatric Disease (United States), Regenerative Medicine Advanced Therapy (United States)

Clinical Trials associated with Rexlemestrocel-L

NCT06325566

/ RecruitingPhase 3

A Prospective, Multicenter, Randomized, Double-blind, Controlled Study to Evaluate the Efficacy and Safety of a Single Injection of Rexlemestrocel-L Combined With HA in Subjects With Moderate to Severe Chronic Low Back Pain

The primary purpose of this study is to evaluate the efficacy of rexlemestrocel-L+HA compared to control in reducing low back pain at 12 months post-treatment and safety of a single injection of rexlemestrocel-L+HA injected into a lumbar intervertebral disc compared to control through 12 months post-treatment.

Start Date03 Jul 2024

Sponsor / Collaborator

Mesoblast Ltd.

NCT03079401

/ Active, not recruitingPhase 1/2IIT

Mesoblast Stem Cell Therapy for Patients With Single Ventricle and Borderline Left Ventricle

Patients under the age of 5, with a diagnosis of hypoplastic left heart syndrome (HLHS), unbalanced atrioventricular canal (uAVC), or borderline left heart who are undergoing staged LV recruitment following bidirectional Glenn (BDG) or undergoing BDG with plans for LV recruitment will be considered for enrollment in this study. Those patients enrolled in the study will be randomized to either the experimental arm or control arm of the study. Those patients randomized to the experimental arm will receive mesenchymal precursor cells (MPCs) injected directly into the LV endocardium during their LV recruitment or BDG procedure. Those patients randomized to the control arm will receive normal standard of care during their procedure with no injection of MPCs.
It is believed that injection of MPCs will help improve the chances of those patients with single ventricle or borderline left ventricle being converted to biventricular circulation which could improve quality of life and longevity over palliation.

Start Date27 Nov 2017

Sponsor / Collaborator

The Children's Hospital Corp.

NCT02412735

/ CompletedPhase 3

A Prospective, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of a Single Injection of Rexlemestrocel-L Alone or Combined With Hyaluronic Acid (HA) in Subjects With Chronic Low Back Pain

This is a prospective, multicenter, randomized, double-blind, placebo-controlled Phase 3 study designed to evaluate the safety and efficacy of Mesoblast's rexlemestrocel-L alone or combined with hyaluronic acid (HA) in participants with chronic low back pain (> 6 months) associated with moderate radiographic degenerative changes of a disc.

Start Date06 Mar 2015

Sponsor / Collaborator

Mesoblast Ltd.

[+1]

100 Clinical Results associated with Rexlemestrocel-L

100 Translational Medicine associated with Rexlemestrocel-L

100 Patents (Medical) associated with Rexlemestrocel-L

Literatures (Medical) associated with Rexlemestrocel-L

01 Oct 2016·EBioMedicineQ1 · MEDICINE

Allogeneic Mesenchymal Precursor Cells (MPC) in Diabetic Nephropathy: A Randomized, Placebo-controlled, Dose Escalation Study

Q1 · MEDICINE

ArticleOA

Author: Segal, Karen R ; Fraser, Ian R ; Kerr, Peter G ; Packham, David K

BACKGROUND:

Diabetic nephropathy is the most common cause of end stage renal failure. We assessed the safety, tolerability, and explored therapeutic effects of adult allogeneic bone-marrow derived mesenchymal precursor cells (MPC) in patients with moderate to severe diabetic nephropathy.

METHODS:

Multicenter, randomized, double-blind, dose-escalating, sequential, placebo-controlled trial assessing a single intravenous (IV) infusion of allogeneic MPC (United States adopted name: rexlemestrocel-L) 150×10⁶ (n=10), 300×10⁶ (n=10) or placebo (n=10) in adults with diabetic nephropathy with an estimated glomerular filtration rate (eGFR) 20-50ml/min/1.73m². Thirty patients at three Australian centers were enrolled between July 2013 and June 2014 and randomized 2:1, in two sequential dose cohorts, to receive rexlemestrocel-L or placebo. Study duration was 60weeks. Primary endpoint was safety and tolerability. Primary exploratory efficacy endpoint was change from baseline in eGFR and directly measured GFR by ⁹⁹Tc-DTPA plasma clearance (mGFR) at 12weeks post-infusion. The trial was registered on ClinicalTrials.gov (NCT01843387).

FINDINGS:

All patients completed the study and were included in analyses applied to the intention to treat population. There were no acute adverse events (AEs) associated with infusion and no treatment-related AEs or serious AEs were deemed treatment-related by investigators. No patients developed persistent donor specific anti-HLA antibodies. Relative to placebo, a single IV rexlemestrocel-L infusion showed trends of stabilizing or improving eGFR and mGFR at week 12. The adjusted least squares mean (LSM±SE) differences from placebo in changes from baseline at 12weeks in the rexlemestrocel-L groups were 4.4±2.16 and 1.6±2.15ml/min/1.73m² for eGFR and 4.1±2.75 and 3.9±2.75 for mGFR for the 150×10⁶ and 300×10⁶ cell groups, respectively.

INTERPRETATION:

This study demonstrates the safety of rexlemestrocel-L in diabetic nephropathy with suggestive effects on renal function to be confirmed in larger, appropriately powered trials.

EUROPEAN JOURNAL OF HEART FAILURE

Mesenchymal precursor cells reduce mortality and major morbidity in ischaemic heart failure with inflammation: DREAM‐HF

Article

Author: Rose, Eric ; James, Christopher W. ; Perin, Emerson C. ; Greenberg, Barry ; Rutman, Olga ; Itescu, Silviu ; Henry, Timothy D. ; Hayes, Jack ; Skali, Hicham ; Borow, Kenneth M. ; Jenkins, Margaret

Aims:

Progressive heart failure with reduced ejection fraction (HFrEF) is adversely affected by alterations in the myocardial balance between bone marrow‐derived pro‐inflammatory cardiac macrophages and embryo‐derived reparative cardiac resident macrophages. Mesenchymal precursor cells (MPCs) may restore this balance and improve clinical outcomes when inflammation is present. The purpose was to (i) identify risk factors for cardiovascular death (CVD) in control patients with HFrEF in the DREAM‐HF trial, and (ii) determine if MPCs improve major clinical outcomes (CVD, myocardial infarction [MI], stroke) in high‐risk patients with ischaemic HFrEF and inflammation.

Methods and results:

Cause‐specific regression analyses were used to identify CVD risk factors in DREAM‐HF control patients. Aalen–Johansen cumulative incidence curves were used to examine CVD, 2‐point major adverse cardiovascular events (MACE) (MI or stroke), and 3‐point MACE (CVD or MI or stroke) by treatment group in ischaemic vs non‐ischaemic HFrEF and in patients with or without baseline inflammation. In control DREAM‐HF patients, factors portending the greatest risk for CVD were inflammation (baseline plasma high‐sensitivity C‐reactive protein ≥2 mg/L; p = 0.003) and ischaemic HFrEF aetiology (p = 0.097), with increased CVD risk of 61% and 38%, respectively. Over 30‐month mean follow‐up, MPCs reduced 2‐point and 3‐point MACE by 88% (p = 0.005) and 52% (p = 0.018), respectively, in patients with ischaemic HFrEF and inflammation compared to controls.

Conclusion:

Ischaemic aetiology and inflammation were identified as major risk factors for MACE in control DREAM‐HF patients. A single intramyocardial MPC administration produced the most significant, sustained reduction in 2‐point and 3‐point MACE in patients with ischaemic HFrEF and inflammation.

News (Medical) associated with Rexlemestrocel-L

22 Jan 2025

·www.prnewswire.com

Chronic Heart Failure Market to Observe Stunning Growth at a CAGR of 10% by 2034 Owing to Strong Uptake of Entresto and approved SGLT2 Inhibitors along with Emergence of New Class of Therapies | DelveInsight

Several leading classes of therapies are exploring diverse mechanisms of action to treat CHF, including SGLT2 inhibitors, cardiac myosin activators, myeloperoxidase inhibitors, mineralocorticoid receptor antagonists, GLP-1 receptor agonists, cell therapies, and more. This growing focus is expected to drive the chronic heart failure market forward. In summary, the existing blockbuster therapies such as Entresto, recently approved SGLT2 inhibitors, and Soluble guanylate cyclase (sGC) stimulators, along with emerging cell therapies, peptides, monoclonal antibodies, and small molecules, together have the potential to drive the existing market along with securing a significant market share upon introduction of new entrants to the CHF treatment landscape. LAS VEGAS, Jan. 22, 2025 /PRNewswire/ -- DelveInsight's Chronic Heart Failure Market Insights report includes a comprehensive understanding of current treatment practices, chronic heart failure emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Chronic Heart Failure Market Report According to DelveInsight's analysis, the market size for chronic heart failure was found to be approximately USD 5.5 billion in the US in 2023. Among the 7MM, the US contributed the largest market share of the total CHF market size and will continue to experience a steep rise in the market size throughout the forecast period. As per DelveInsight's estimate, the total diagnosed prevalent cases of CHF in the 7MM were approximately 20 million in 2023. Heart failure with preserved ejection fraction (HFpEF) contributed to most of the cases. Within class-specific diagnosed prevalence of Heart failure, NYHA class II and class III contribute the maximum. Established chronic heart failure companies with approved therapies in the market are Novartis, Otsuka, Boehringer Ingelheim/Eli Lilly, Bayer/Merck, Amgen/Servier, scPharmaceuticals, and Lexicon Pharmaceuticals/Viatris. In addition, Daiichi Sankyo and American Regent are targeting adult patients with heart failure who are iron deficient. Among all the approved therapies, Novartis and Otsuka's Entresto, approved since 2015 in the US, is still the leading therapy generating more than USD 4 billion in the 7MM in 2023. Among, Eli Lilly's Jardiance and AstraZeneca's Farxiga, both SGLT2 inhibitors, Farxiga is the leading prescribed SGLT2 inhibitor globally by volume Leading chronic heart failure companies developing emerging therapies, such as Cytokinetics, Bayer, Eli Lilly, BioCardia, Mesoblast, Tenax Therapeutics, Novo Nordisk, AstraZeneca, Boehringer Ingelheim, Bristol Myers Squibb, Intra-Cellular Therapies, Cardurion Pharmaceuticals, Rivus Pharmaceuticals, and others are developing novel chronic heart failure drugs that can be available in the chronic heart failure market in the coming years. The promising chronic heart failure therapies in the pipeline include Omecamtiv Mecarbil, KERENDIA (finerenone), Tirzepatide (LY3298176), CardiAMP Cell Therapy, Ziltivekimab (NN6018), REVASCOR (rexlemestrocel-L), Levosimendan (TNX-103), Semaglutide, Balcinrenone (AZD9977) + dapagliflozin, Mitiperstat (AZD4831), Vicadrostat (BI 690517) + Empagliflozin, Cimlanod (CXL-1427/BMS-986231), Lenrispodun (ITI - 214), CRD-740, HU6, and others. In October 2024, Viatris entered into an exclusive licensing agreement with Lexicon Pharmaceuticals for INPEFA (sotagliflozin) in all markets outside of the US and EU. In September 2024, Bayer presented the late-breaking data of Phase III FINE-HEART findings of KERENDIA (finerenone) during a Hot Line session at the European Society of Cardiology (ESC) Congress 2024. In May 2024, Eli Lilly and Company committed an additional USD 5.3 billion manufacturing investment in the company's newest Indiana site to boost API production for tirzepatide and pipeline medicines. AstraZeneca expects the data of the Phase III BalanceD-HF trial of balcinrenone (AZD9977) + dapagliflozin for heart failure in 2025. Discover which therapies are expected to grab the major chronic heart failure market share @ Chronic Heart Failure Market Report Chronic Heart Failure Overview Chronic heart failure is a long-term condition in which the heart's ability to pump blood efficiently is reduced, leading to inadequate circulation of oxygen and nutrients to meet the body's needs. The primary causes of CHF include coronary artery disease, high blood pressure, diabetes, cardiomyopathy, valvular heart disease, and previous heart attacks. Lifestyle factors like obesity, smoking, excessive alcohol consumption, and a sedentary lifestyle can also contribute to its development. The symptoms of CHF vary in severity and may include persistent fatigue, shortness of breath, swelling in the legs, ankles, or abdomen, rapid or irregular heartbeat, and difficulty exercising. Advanced cases can lead to severe fluid retention, wheezing, and an inability to carry out daily activities. Diagnosis typically involves a comprehensive medical history review and a physical examination, followed by tests like an ECG to assess heart rhythm, echocardiography to evaluate heart structure and function, blood tests, chest X-rays, and stress tests. Early diagnosis and management are crucial to improving quality of life and preventing complications. Chronic Heart Failure Epidemiology Segmentation The chronic heart failure epidemiology section provides insights into the historical and current chronic heart failure patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The chronic heart failure market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Diagnosed Prevalent Cases of Heart Failure Gender-specific Cases of Heart Failure Ejection Fraction-specific Cases of Heart Failure New York Heart Association (NYHA) Class-specific Cases of Heart Failure Type-specific Cases of Heart Failure Age-specific Cases of Heart Failure Chronic Heart Failure Treatment Market Current treatments for heart failure primarily rely on angiotensin-converting enzyme (ACE) inhibitors, angiotensin receptor blockers (ARBs), beta-blockers, and diuretics. Additional therapies, such as aldosterone antagonists, amiodarone, antiplatelets, anticoagulants, calcium channel blockers, and nitrates, are also utilized in managing the condition. Most treatment regimens involve a combination of therapies, with beta-blockers being among the most commonly prescribed. Among the approved treatments, Novartis' ENTRESTO has emerged as a leading drug in the congestive heart failure market. Initially slow to gain traction, ENTRESTO has become one of Novartis' key growth drivers, with revenue expected to increase in the coming years. Following its approval for use in patients with preserved ejection fraction, ENTRESTO's market presence has grown significantly. However, ENTRESTO faces growing competition. The entry of new drugs, including SGLT2 inhibitors (such as FARXIGA and JARDIANCE) and VERQUVO, poses a challenge to ENTRESTO's dominance. Additionally, Novartis is set to lose ENTRESTO's exclusivity in the United States in 2025, raising the risk of generic competition. The company is actively engaged in several patent lawsuits to protect its position. SGLT2 inhibitors have also gained a foothold in the CHF market. Although FARXIGA and JARDIANCE are established drugs, they are relatively new in this therapeutic area. The outlook for SGLT2 inhibitors in heart failure is promising, but AstraZeneca and Lilly/Boehringer will have limited time to solidify their positions, as FARXIGA's US patent expires in 2025 and JARDIANCE's in 2028. The recently approved SGLT2 inhibitor sotagliflozin may face challenges in capturing market share from these established treatments. To know more about chronic heart failure treatment guidelines, visit @ Chronic Heart Failure Management Chronic Heart Failure Pipeline Therapies and Key Companies Omecamtiv Mecarbil: Cytokinetics KERENDIA (finerenone): Bayer Tirzepatide (LY3298176): Eli Lilly and Company CardiAMP Cell Therapy: BioCardia Ziltivekimab (NN6018): Novo Nordisk REVASCOR (rexlemestrocel-L): Mesoblast Levosimendan (TNX-103): Tenax Therapeutics Semaglutide: Novo Nordisk Balcinrenone (AZD9977) + dapagliflozin: AstraZeneca Mitiperstat (AZD4831): AstraZeneca Vicadrostat (BI 690517) + Empagliflozin: Boehringer Ingelheim Cimlanod (CXL-1427/BMS-986231): Bristol Myers Squibb Lenrispodun (ITI - 214): Intra-Cellular Therapies CRD-740: Cardurion Pharmaceuticals HU6: Rivus Pharmaceuticals Discover more about chronic heart failure drugs in development @ Chronic Heart Failure Clinical Trials Chronic Heart Failure Market Dynamics The chronic heart failure market dynamics are expected to change in the coming years. An increasing prevalence of cardiovascular diseases, fueled by aging populations and lifestyle-related risk factors such as obesity, hypertension, and diabetes, has significantly raised the demand for effective CHF treatments. Advances in medical technology, including innovative drug therapies, cardiac devices, and regenerative medicine, are expanding treatment options and improving patient outcomes. Growing awareness about heart health and early diagnosis, supported by government and non-government initiatives, is also boosting market expansion. Additionally, the rising focus on personalized medicine and the development of therapies targeting the underlying molecular mechanisms of CHF are creating lucrative opportunities. The increasing healthcare expenditure and the availability of reimbursement policies in developed and emerging economies further bolster the market's growth trajectory. Furthermore, potential therapies are being investigated for the treatment of chronic heart failure, and it is safe to predict that the treatment space will significantly impact the chronic heart failure market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the chronic heart failure market in the 7MM. However several factors may impede the growth of the chronic heart failure market. A key challenge is the underdiagnosis and delayed recognition of CHF due to overlapping symptoms with other conditions and the lack of widespread access to advanced diagnostic tools in many regions. Economic constraints, particularly in low- and middle-income countries, limit patient access to costly therapies, including advanced drugs and devices. Additionally, adherence to treatment regimens remains low due to the complexity of management protocols and the long-term nature of care, leading to suboptimal outcomes. Regulatory hurdles and lengthy approval processes for novel therapies further impede the introduction of innovative treatments. Finally, a limited understanding among general practitioners about newer guidelines and therapies reduces the referral rates to specialists, creating gaps in patient care. Addressing these barriers requires concerted efforts in education, affordability, and healthcare infrastructure. Scope of the Chronic Heart Failure Market Report Therapeutic Assessment: Chronic Heart Failure current marketed and emerging therapies Chronic Heart Failure Market Dynamics: Key Market Forecast Assumptions of Emerging Chronic Heart Failure Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Chronic Heart Failure Market Access and Reimbursement Download the report to understand which factors are driving chronic heart failure market trends @ Chronic Heart Failure Market Trends Table of Contents Related Reports Chronic Heart Failure Epidemiology Forecast Chronic Heart Failure Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology as well as the chronic heart failure epidemiology trends. Chronic Heart Failure Pipeline Chronic Heart Failure Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key CHF companies, including Zensun (Shanghai) Sci & Tech, Cytokinetics, Mesoblast, Shanghai Hongyitang Biopharmaceutical Technology, Tasly Pharmaceuticals, Ionis Pharmaceuticals, Berlin Cures, CardioCell, Help Therapeutics, Eli Lilly and Company, Chong Kun Dang Pharmaceutical, Antlia Biosciences, Cardiol Therapeutics, among others. Acute Coronary Syndrome Market Acute Coronary Syndrome Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key ACS companies, including Novartis, Boehringer Idorsia Pharmaceuticals, Viatris, Recardio, AstraZeneca, Faraday Pharmaceuticals, DalCor Pharmaceuticals, Roche, Jiangsu Vcare PharmaTech, CeleCor Therapeutics, Novo Nordisk, Bristol Myers Squibb, Johnson & Johnson Innovative Medicine, CellProthera, BioCardia, Kancera, Amgen, Arrowhead Pharmaceuticals, Abcentra , among others. Acute Coronary Syndrome Pipeline Acute Coronary Syndrome Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acute coronary syndrome companies, including Janssen Research & Development, LLC, DalCor Pharmaceuticals, Hyloris Pharmaceuticals, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 3Drug ApprovalLicense out/in

19 Dec 2024

·pipelinereview.com

Mesoblast’s RYONCIL® is the First U.S. FDA-Approved Mesenchymal Stromal Cell (MSC) Therapy

NEW YORK, NY, USA I December 18, 2024 I Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today 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. Transplant physician Dr Joanne Kurtzberg, the Jerome Harris Distinguished Professor of Pediatrics and Professor of Pathology, and Director, Marcus Center for Cellular Cures at Duke University Medical Center (DUMC), said: “Steroid-refractory acute graft versus host disease is a devastating condition with an extremely poor prognosis. From today we are able to offer RYONCIL, the first FDA-approved treatment which will be life saving for so many children and will have a great impact on their families.” 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. 1-5 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 . 6 “We are very pleased that the FDA has granted approval of RYONCIL ® and are proud of the company’s commitment to the GVHD community in bringing this important new treatment to children and families with no other acceptable options,” said Dr. Silviu Itescu, Chief Executive of Mesoblast. “With RYONCIL approval by FDA, Mesoblast has demonstrated the ability to bring the first MSC product to market. We will continue to work closely with FDA to obtain approval of our other late-stage products, including REVASCOR ® for cardiovascular diseases and rexlemestrocel-L for inflammatory pain indications, as well as expanding the indications for RYONCIL in both children and adults with inflammatory conditions.” RYONCIL will be available in the United States at transplant centers and other treating hospitals. Please see the full Prescribing Information at www.ryoncil.com . The FDA’s approval press release is available here . What is RYONCIL (remestemcel-L) RYONCIL is an allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy indicated for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months of age and older. The recommended dosage of RYONCIL is 2 × 10 6 MSC /kg body weight per intravenous infusion given twice per week for 4 consecutive weeks. Response is assessed 28 ± 2 days after the first dose and further treatment administered as appropriate. Important Safety Information Contraindications: Known hypersensitivity to dimethyl sulfoxide (DMSO) or porcine and bovine proteins. Adverse reactions: Serious adverse reactions included pyrexia (9%), respiratory failure (9%), pneumatosis intestinalis (7%) and staphylococcal bacteremia infection (<5%). Adverse reactions of Grade 3 occurring in ≥10% of patients were viral infectious disorders (15%), bacterial infectious disorders (19%), and infections pathogen unspecified (15%). No grade 4 or 5 adverse reactions occurred in the study. Eight patients had discontinuation of RYONCIL treatment due to the following: acute infusion reactions (n=3), hypotension (n=1), gastroenteritis (n=1), and death (n=3). You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . You may also report side effects to Mesoblast at toll-free phone #1-844-889-MESO (6376) Please see the RYONCIL full Prescribing Information for additional Important Safety Information. 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. 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 SOURCE: Mesoblast

Cell TherapyPhase 3Clinical ResultDrug ApprovalImmunotherapy

19 Dec 2024

·www.pharmaceutical-technology.com

FDA approves Mesoblast’s Ryoncil for SR-aGvHD treatment

The therapy is also under development for SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Credit: PowerUp/Shutterstock. Mesoblast has gained US Food and Drug Administration (FDA) approval for its mesenchymal stromal cell (MSC) therapy, Ryoncil (remestemcel-L), to treat steroid-refractory acute graft versus host disease (SR-aGvHD) in paediatric patients aged a minimum of two months and up to adolescents and teenagers. It is the only MSC therapy approved in the US for any indication. Ryoncil is part of a development programme for inflammatory diseases such as SR-aGvHD in adults, and biologic-resistant inflammatory bowel disease. Other therapies, such as rexlemestrocel-L, are being developed for conditions such as chronic lower back pain and heart failure. The company has established commercial partnerships for these therapies in China, Japan and Europe. Mesoblast chief executive Dr Silviu Itescu stated: “We are very pleased that the FDA has granted approval of Ryoncil and proud of the company’s commitment to the GVHD community in bringing this important new treatment to children and families with no other acceptable options. “With RYONCIL’s approval by the FDA, Mesoblast has demonstrated the ability to bring the first MSC product to market. We will continue to work closely with the FDA to obtain approval of our other late-stage products, including REVASCOR for cardiovascular diseases and rexlemestrocel-L for inflammatory pain indications, as well as expanding the indications for RYONCIL in both children and adults with inflammatory conditions.” The mesenchymal lineage cell therapy technology platform of the company is designed to respond to severe inflammation with the release of anti-inflammatory factors. These factors counter and modulate multiple effector immune system arms, leading to a reduction in the damaging inflammatory process. In the US, the therapy will be available at hospitals and transplant centres. 10,000 individuals in the US undergo an allogeneic bone marrow transplant annually, of whom 1,500 are paediatric patients. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

Cell TherapyDrug ApprovalImmunotherapyClinical StudyAccelerated Approval

100 Deals associated with Rexlemestrocel-L

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Intervertebral Disc Degeneration	Phase 3	United States	Mesoblast Ltd.	06 Mar 2015
Intervertebral Disc Degeneration	Phase 3	Australia	Mesoblast Ltd.	06 Mar 2015
Low Back Pain	Phase 3	United States	Mesoblast Ltd.	06 Mar 2015
Low Back Pain	Phase 3	Australia	Mesoblast Ltd.	06 Mar 2015
Chronic heart failure	Phase 3	United States	Mesoblast, Inc.	14 Feb 2014
Chronic heart failure	Phase 3	Canada	Mesoblast, Inc.	14 Feb 2014
Heart Failure	Phase 3	United States	Teva Pharmaceutical Industries Ltd.	21 Jan 2014
Diabetic Nephropathies	Phase 2	Australia	Mesoblast Ltd.	01 Jul 2013
Rheumatoid Arthritis	Phase 2	United States	Mesoblast Ltd.	26 Apr 2013
ST Elevation Myocardial Infarction	Phase 2	-	Mesoblast, Inc.	11 Mar 2013

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


Literature Manual	Not Applicable	Heart Failure	-	Mesenchymal precursor cells	vevmevbdya(lyhchjiavt): Difference (%) = -88, P-Value = 0.005 View more	Positive	26 Nov 2024
				Comparator
NCT03079401 (Literature) Manual	Phase 1/2	Hypoplastic Left Heart Syndrome	19	rexlemestrocel-L	chqpbiadxl(pellnkqzmy) = Baseline PRA levels were high, with no difference between the groups at 12 months. xoehqeoggy (bskevfzzvc )	Positive	01 Oct 2023
				No Intervention
NCT02032004 (DREAM HF-1, GlobeNewswire) Manual	Phase 3	Heart Failure	565	Rexlemestrocel-L	hbfygblwtd(tjsuqrbruu): HR = 1.17 (95% CI, 0.81 - 1.69), P-Value = 0.406 View more	Negative	27 Feb 2023
				Sham Comparator
NCT02412735 (MSB-DR003, CTgov)	Phase 3	Intervertebral Disc Degeneration \| Low Back Pain	404	Rexlemestrocel-L (Rexlemestrocel-L)	mtoclbwcmz(wvcthjvoxf) = uclxyyakyi alcnvsihqw (uxalhbcpil, 0.038) View more	-	18 Jan 2022
				Rexlemestrocel-L + HA Mixture (Rexlemestrocel-L + HA)	mtoclbwcmz(wvcthjvoxf) = yipidlbris alcnvsihqw (uxalhbcpil, 0.043) View more
NCT01781390 (AMICI, CTgov)	Phase 2	ST Elevation Myocardial Infarction \| Acute myocardial infarction	106	Placebo (Placebo)	nfgsihzdlo = ubtenvhrpa rkyyalqhkp (xigqzuxucm, wobzcprivo - xwwncoqlcb) View more	-	18 Jan 2022
				Mesenchymal Precursor Cells (MPC) 12.5 M (Mesenchymal Precursor Cells (MPC) 12.5 M)	nfgsihzdlo = jojnsvjkgm rkyyalqhkp (xigqzuxucm, msmqordakq - meincmukaz) View more
NCT02032004 (DREAM-HF, GlobeNewswire) Manual	Phase 3	Heart Failure	537	rexlemestrocel-L	ujlznqudem(xsjjjtpcsp) = iqhxpztntd xadldsixov (wwxpgoxthp ) View more	Positive	14 Dec 2020
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