Rexlemestrocel-L
- Generic Name
- Rexlemestrocel-L
- Drug Type
- Biotech
- CAS Number
- 2172954-10-6
- Unique Ingredient Identifier
- 4LS0IJZ59V
- Background
Rexlemestrocel-L is an investigational human bone marrow-derived allogeneic mesenchymal precursor cell (MPC) product. It is being investigated in various cardiac conditions, such as chronic heart failure and acute myocardial infarction.
Myocardial Infarction Therapeutics Pipeline Shows Promise with Over 45 Companies Involved
• The myocardial infarction therapeutics pipeline is robust, featuring over 45 companies developing more than 50 potential drugs.
• Key companies like Boehringer Ingelheim and Idorsia Pharmaceuticals are advancing novel therapies to improve outcomes post-myocardial infarction.
• Several promising therapies, including Selatogrel and FDY-5301, are in Phase III trials, showing potential for significant advancements.
• Recent clinical trial activities, such as the commencement of the PERFECT study and completion of enrollment in the Iocyte AMI-3 trial, highlight ongoing research efforts.
FDA Approves Ryoncil, First Mesenchymal Stromal Cell Therapy for Pediatric Steroid-Refractory Acute Graft-versus-Host Disease
• The FDA has approved Ryoncil (remestemcel-L-rknd) as the first mesenchymal stromal cell (MSC) therapy for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients.
• Ryoncil's approval was based on a Phase III trial demonstrating a 70% overall response rate in children with SR-aGVHD after 28 days of treatment.
• This allogeneic, bone marrow-derived MSC therapy offers a new treatment option for children with SR-aGVHD who do not respond to steroid treatment.
• Ryoncil is administered intravenously and should be monitored for infusion reactions; common side effects include infections, fever, hemorrhage, and abdominal pain.
Revascor Shows Promise in DREAM-HF Trial for Ischemic Heart Failure
• Mesoblast's Revascor demonstrated improved survival and reduced major morbidity in high-risk ischemic heart failure patients with inflammation.
• The DREAM-HF trial highlighted Revascor's potential to reduce cardiovascular mortality and major adverse cardiovascular events.
• A single intramyocardial injection of Revascor led to a sustained reduction in cardiovascular mortality in high-risk heart failure patients.
• Revascor also showed a reduction in the risk of heart attacks, strokes, and recurrent hospitalizations in heart failure patients.
FDA Approves Ryoncil, First Mesenchymal Stromal Cell Therapy for Pediatric SR-aGVHD
• The FDA has approved Ryoncil (remestemcel-L) as the first mesenchymal stromal cell (MSC) therapy in the United States.
• Ryoncil is indicated for steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients aged 2 months and older.
• Clinical trials showed a 70% overall response rate by Day 28 in children with SR-aGvHD treated with Ryoncil.
• Ryoncil offers a new treatment option for a life-threatening condition with limited alternatives, improving outcomes for affected children.
Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025
• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD.
• EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration.
• Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option.
• Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.
Mesoblast Anticipates FDA Decision on Ryoncil for Pediatric SR-aGvHD by January 2025
• Mesoblast anticipates an FDA decision on Ryoncil (remestemcel-L) by January 7, 2025, for treating steroid-refractory acute graft versus host disease (SR-aGvHD) in children under 12.
• The company has secured manufacturing and distribution channels for Ryoncil and completed a Pre-License Inspection by the FDA with no significant regulatory issues.
• Revascor (rexlemestrocel-L) received Rare Pediatric Disease and Orphan-Drug Designation for treating paediatric congenital heart disease, potentially leading to FDA incentives.
• Mesoblast reported reduced operating cash outflows and a solid cash balance, supported by financing facilities contingent on Ryoncil's approval, alongside cost-control measures.
Mesoblast Awaits FDA Decision on Ryoncil for Pediatric SR-aGvHD
• Mesoblast anticipates FDA decision on Ryoncil (remestemcel-L) by January 7, 2025, for treating steroid-refractory acute graft versus host disease (SR-aGvHD) in children.
• The company has secured manufacturing and distribution channels for Ryoncil, completing an FDA Pre-License Inspection with no significant regulatory issues.
• Revascor (rexlemestrocel-L) received Rare Pediatric Disease and Orphan-Drug Designation for paediatric congenital heart disease, advancing other clinical programs.
• Mesoblast reduced net operating cash outflows by 26% year-over-year, with US$51.1m in cash reserves and access to US$60m in financing upon Ryoncil's approval.
Mesoblast Anticipates FDA Decision on Ryoncil for Pediatric SR-aGVHD by January 7, 2025
• Mesoblast's Biologics License Application (BLA) for Ryoncil (remestemcel-L) in children with steroid-refractory acute graft versus host disease (SR-aGVHD) is under FDA review, with a PDUFA date of January 7, 2025.
• Strategic financing is in place, including a potential US$50 million convertible note agreement, to support the commercial launch of Ryoncil upon FDA approval.
• A Phase 3 trial has commenced for rexlemestrocel-L in chronic low back pain due to inflammatory degenerative disc disease, with FDA agreement on trial design and endpoints.
Mesoblast Anticipates FDA Decision on Ryoncil for Pediatric SR-aGVHD by January 7, 2025
• Mesoblast's Biologics License Application (BLA) for Ryoncil (remestemcel-L) in children with steroid-refractory acute graft versus host disease (SR-aGvHD) is under FDA review, with a decision expected by January 7, 2025.
• Strategic financing, including a potential US$50 million convertible note, is in place to support the commercial launch of RYONCIL upon FDA approval.
• A Phase 3 trial of rexlemestrocel-L for chronic low back pain due to inflammatory degenerative disc disease has commenced enrollment, following FDA agreement on trial design and endpoints.
Mesoblast's Ryoncil Nears Potential FDA Approval for Pediatric GvHD
• Mesoblast anticipates FDA approval for Ryoncil in early January, a stem cell therapy for pediatric steroid-refractory acute graft-versus-host disease (GvHD).
• Ryoncil's approval would mark the first FDA-approved allogeneic stem cell treatment derived from healthy human donor cells, offering an off-the-shelf solution.
• Mesoblast is advancing multiple Phase 3 programs, including rexlemestrocel-L for chronic low back pain, aiming to broaden its therapeutic applications.
• The company has secured $50 million to support the commercialization of Ryoncil, highlighting its strategic focus on addressing unmet needs in inflammatory diseases.
Mesoblast's Ryoncil Nears Potential FDA Approval for Pediatric Graft-versus-Host Disease
• Mesoblast's remestemcel-L (Ryoncil) has a PDUFA date of January 7, 2025, for treating steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients.
• If approved, Ryoncil would be the first treatment option for children under 12 with SR-aGVHD, addressing a critical unmet need in this vulnerable population.
• Industry analysts maintain a consensus Buy rating for Mesoblast, with an average price target of $11, representing a 56% upside potential.
FDA Grants RMAT Designation To Mesoblast's Revascor for Pediatric Hypoplastic Left Heart Syndrome
The U.S. Food and Drug Administration has granted Mesoblast Limited's Revascor (rexlemestrocel-L) the Regenerative Medicine Advanced Therapy (RMAT) designation for treating children with hypoplastic left heart syndrome (HLHS), following results from a randomized controlled trial. This designation highlights the therapy's potential in addressing this serious congenital heart condition.
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