Australian cell therapy company Mesoblast joins forces with Germany's Grünenthal to tackle low back pain
While the FDA reviews a rolling application for its experimental stem cell therapy for acute graft versus host disease (GVHD), Australia’s Mesoblast has tied up Germany’s pain specialist Grünenthal to develop a treatment for chronic low back pain caused by degenerative disc disease.
Under the deal, Grünenthal will gain exclusive commercialization rights to the drug, MPC-06-ID, for Europe and Latin America. Mesoblast, which is testing the drug in a US late-stage study that is expected to readout next year, is getting $150 million in upfront and milestone payments, in addition to potential royalties. Cumulative milestone payments could surpass $1 billion, the companies said on Monday.
A single injection of the drug, which is being developed for use in patients who have not benefited from existing options, resulted in over a three-fold increase, versus placebo, in successfully achieving a composite goal in a 100-patient mid-stage study. The endpoint consisted of 50% improvement in low back pain and 15 point improvement in function at both 12 and 24 months with no treatment or surgical interventions at the treated level through 24 months. Overall, 37% of patients treated with MPC-06-ID compared with 10% in the control group met the composite endpoint over two years, Mesoblast said.
In the United States, the FDA has amped up its scrutiny of stem cell clinics promising wonder cures without a shred of evidence. Mesoblast is putting its technology — which is based on mesenchymal lineage adult stem cells (rare cells found in the vicinity of blood vessels that promote tissue repair and modulate immune responses) — through the clinic to test its viability across a variety of conditions such as GVHD, heart failure, pain, rheumatoid arthritis and Crohn’s disease. The company’s off-the-shelf GVHD cell therapy, Temcell, has been approved in Japan (and is licensed by partner JCR Pharmaceuticals in the region).
MPC-06-ID is designed as an intra-discal injection — engineered to degenerate intervertebral discs to allow the mesenchymal lineage adult stem cells (MLCs) to secrete biomolecules involved in enhanced migration and proliferation of intervertebral disc progenitor cells, in a bid to strengthen the load-bearing function of the disc by improving its stability and enhancing its water content, while also reducing inflammation and pain.
Grünenthal and Mesoblast have agreed on an overall development plan for MPC-06-ID to meet European regulatory requirements, and the two partners will accordingly work on a late-stage design for Europe. The results of the European and US trials are expected to support regulatory approvals in both regions, the companies said.
Among key characteristics of Mesoblast’s technology is that the company’s products are fertile for significant expansion in culture, Mesoblast chief executive Silviu Itescu noted. “These properties facilitate their use as allogeneic, or ‘off-the-shelf,’ therapeutics with well-defined release criteria and batch-to-batch reproducibility that meet stringent regulatory requirements.”
These are some of the challenges faced by the first generation of cell therapies which are autologous CAR-T therapeutics — Novartis’ $NVS Kymriah and Gilead’s $GILD Yescarta which secured FDA approval in 2017. These products require the manufacturers to first isolate cells from the patient, manipulate them in the lab by adding chimeric antigen receptors to direct T cells to snuff out cancer cells and then re-infuse them back into the patient. This process requires enormous manufacturing heft — a challenge that has triggered relatively muted sales for Kymriah, with Novartis struggling to meet demand.