Product Candidates Overview

We have applied our proprietary cell technology platform, which is based on mesenchymal lineage adult stem cells (MLCs), to build what we believe is the most advanced cell-based product portfolio in the industry. This technology has the potential to address multiple conditions with significant unmet medical needs.

Understanding and capitalizing on the mechanisms of action of our proprietary MLCs has enabled us to develop MLC-based products that target specific disease states. These mechanisms of action demonstrated in preclinical models include induction of tissue repair, new blood vessel network formation, and immunomodulation.

Our ‘off-the-shelf’ products are allogenic - meaning stem cells from a single donor can be used different recipients without the need for matching.

The Japanese Government has approved TEMCELL® HS. Inj., a product based on our proprietary mesenchymal lineage adult stem cell technology licensed to JCR Pharmaceuticals Co. Ltd for the treatment of graft versus host disease. TEMCELL is the first allogeneic cell-based product to receive full regulatory approval in Japan.

We expect to have the first industrially manufactured allogeneic mesenchymal lineage cell-based product launched in the United States. The Phase 3 trial using our product candidate MSC-100-IV in children with steroid-refractory acute graft versus host disease successfully met the primary endpoint of Day 28 overall response in February 2018.  Based on interactions with the FDA, Mesoblast believes that successful results from the completed Phase 3 trial through Day 100, together with Day 180 safety and quality of life parameters in these patients, may provide sufficient clinical evidence for filing for MSC-100-IV in the United States under an accelerated approval pathway.

Two other lead product candidates, MPC-150-IM and MPC-06-ID, are in late-stage development in Phase 3 programs targeting, respectively, advanced chronic heart failure and chronic low back pain due to degenerative disc disease. These are major diseases with high unmet medical needs.

Additionally, we have a strong emerging pipeline of product candidates targeting a number of inflammatory/immune-mediated conditions.

 

Portfolio of Advanced Candidates: