Mesoblast is using its proprietary technology platform to develop and commercialize innovative allogeneic cellular medicines to treat complex diseases resistant to conventional standard of care and where inflammation plays a central role.
The Company’s portfolio of Phase 3 product candidates comprises RYONCIL™ (remestemcel-L) for steroid-refractory acute graft versus host disease (acute GVHD), remestemcel-L for the treatment of moderate to severe acute respiratory distress syndrome (ARDS) due to COVID-19 infection, REVASCOR® for advanced chronic heart failure and MPC-06-ID for chronic low back pain due to degenerative disc disease.
The United States Food and Drug Administration (FDA) has accepted for priority review Mesoblast’s Biologics License Application (BLA) to seek approval of RYONCIL for acute GVHD in children. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of September 30, 2020. If approved, Mesoblast will make RYONCIL immediately available in the United States.
Phase 3 Product Candidates
The Company also has a promising emerging pipeline of product candidates and next generation technologies.
Innovative technology platform enables scalable manufacturing
Mesoblast’s novel allogeneic product candidates are based on rare (approximately 1:100,000 in bone marrow) mesenchymal lineage cells that respond to tissue damage, secreting mediators that promote tissue repair and modulate immune responses.
Mesenchymal lineage cells are collected from the bone marrow of healthy adult donors and proprietary processes are utilized to expand them to a uniform, well characterized, and highly reproducible cell population. This enables manufacturing at industrial scale for commercial purposes. Another key feature of Mesoblast’s cells is they can be administered to patients without the need for donor–recipient matching or recipient immune suppression.
Proprietary media formulations, advances in development of 3D bioreactor technology and automation are intended to deliver step-changes improvement in product yield.
Robust Intellectual Property Estate
Mesoblast has an extensive patent portfolio comprising approximately 1,000 patents and patent applications with protection extending through 2040 in all major markets. This intellectual property portfolio covers composition of matter, manufacturing, and therapeutic applications of mesenchymal lineage cells. The Company believes this patent estate provides strong global protection in areas of its core commercial focus.
Evidence-based Science and Translational Medicine
Mesoblast’s approach to product development is to ensure rigorous scientific investigations are performed with well-characterized cell populations in order to understand mechanisms of action for each potential indication. Extensive preclinical translational studies guide clinical trials that are structured to meet stringent safety and efficacy criteria set by international regulatory agencies. All trials are conducted under the continuing review of independent Data Safety Monitoring Boards comprised of independent medical experts and statisticians. These safeguards are intended to ensure the integrity and reproducibility of results, and to ensure that outcomes observed are scientifically reliable.
Global Operations
Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO).