FDA to Conduct Pre-Launch Inspection of Remestemcel-L Manufacturing Facility

Mesoblast Limited (ASX:MSB; Nasdaq:MESO), the global leader in allogeneic cellular medicines for inflammatory diseases, recently announced that the United States Food and Drug Administration (FDA) will be conducting a Pre-License Inspection (PLI) of its cell therapy manufacturing operations at Lonza Bioscience in Singapore. This inspection is part of the ongoing review of the BLA for remestemcel-L in the treatment of children with SR-aGVHD.

On March 7, 2023, the FDA welcomed the Company’s re-submission of the BLA for remestemcel-L with open arms, setting an ambitious target date of August 2, 2023 via the Prescription Drug User Fee Act (PDUFA). This marks an important step forward in the development of a potential new treatment for serious conditions.

Remestemcel-L, which has been granted both FDA Fast Track and Priority Review designations, offers hope for those suffering from severe forms of SR-aGVHD. For the past two decades, there have been no improvements in survival rates for children or adults with the most severe forms of the disease. For this reason, a therapy that can improve the dismal survival outcomes in children is desperately needed, especially since there are currently no approved treatments for children under 12.

We are delighted to announce that FDA has scheduled a pre-license inspection of our manufacturing operations. We are committed to collaborating with FDA to make remestemcel-L a potential therapy for children suffering from SR-aGVHD. We are eager to bring this life-saving treatment to those in need, and look forward to a successful review period.

About Steroid-Refractory Acute Graft Versus Host Disease

Every year, over 30,000 people worldwide undergo allogeneic bone marrow transplants (BMTs) for blood cancers, with about 20% of these patients being children. Unfortunately, these transplants come with a potentially deadly complication known as SR-aGVHD, which has a mortality rate as high as 90%. Even worse, there are currently no FDA-approved treatments for children under 12 who suffer from this condition. For those over 50% of BMT recipients who develop acute GVHD, the stakes are high and the costs of extended hospital stays can be significant.

About Remestemcel-L

Mesoblast’s pioneering product Remestemcel-L is an investigational therapy composed of culture-expanded mesenchymal stromal cells sourced from the bone marrow of an unrelated donor. Administered through a series of intravenous infusions, Remestemcel-L is thought to reduce the detrimental inflammatory processes of SR-aGVHD by reducing the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and encouraging the recruitment of natural anti-inflammatory cells to affected tissues.

The Oncologic Drugs Advisory Committee (ODAC) voted in overwhelming favor (9:1) that the clinical outcomes of 309 children with severe and refractory acute graft versus host disease (SR-aGVHD) who were treated with remestemcel-L presented in three separate trials supported the efficacy of this treatment. The impressive data demonstrated consistent treatment responses and survival rates, affirming remestemcel-L as a promising option for pediatric patients with SR-aGVHD.

Mesoblast’s Phase 3 trial recently reported remarkable results, with 67% of children with high-risk disease responding positively to remestemcel treatment within 28 days, and surviving after 180 days. This is in stark contrast to the 10% response and survival rate of the control group of 27 children treated with various biologics, including ruxolitinib, from the Mount Sinai Acute GvHD International Consortium (MAGIC) database.

This impressive outcome was enabled by the inclusion of additional clinical and biomarker data to the BLA resubmission, including data from a propensity-matched study based on the validated MAP biomarker score.

The Phase 3 trial conducted by the Center for International Blood and Marrow Transplant Research (CIBMTR) showcased impressive results for 51 evaluable patients with SR-aGVHD. Results from the 4-year survival study revealed that 63% of patients survived 1 year and 51% survived 2 years, despite having predominantly grade C/D disease (89%) with a dismal expected 2-year survival rate of 25-38% with best available therapy.1,8-9 These results demonstrate the durability of early day 180 survival benefits, showing promising results in the treatment of SR-aGVHD.

About Mesoblast

Mesoblast is revolutionizing the medical world by developing groundbreaking allogeneic cellular medicines to treat severe and life-threatening inflammatory conditions. Its unique mesenchymal lineage cell therapy technology platform has enabled the creation of a diversified portfolio of late-stage product candidates that have the potential to reduce damaging inflammation by releasing powerful anti-inflammatory factors that counter and modulate the immune system. Mesoblast is paving the way for a new era of treatments for inflammatory diseases.

Mesoblast boasts an impressive global intellectual property portfolio, with patents secured in all major markets until 2041. Its advanced manufacturing processes create industrial-scale cellular medicines that can be cryopreserved and stored for long-term use. This means that patients around the world can access these treatments with consistent quality and precision, anytime, anywhere.

Mesoblast is leading the way in the development of innovative product candidates for a wide range of indications based on its advanced remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. From steroid refractory acute graft versus host disease and biologic-resistant inflammatory bowel disease to acute respiratory distress syndrome and advanced chronic heart failure, these products are helping to revolutionize the way patients are treated.

Furthermore, Mesoblast has already established commercial partnerships in Europe and China for certain Phase 3 assets, with two products being commercialized in Japan and Europe by its licensees.

Mesoblast is a global player with locations in Australia, the United States and Singapore and is publicly traded on both the Australian Securities Exchange (MSB) and the Nasdaq (MESO). Its presence in multiple countries and exchanges makes it an attractive opportunity for investors looking to take advantage of global markets.

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