Approval of Off-the-Shelf Stem Cell Therapy to Treat Epidermolysis Bullosa Sought in Japan, JCR Pharmaceuticals Says

JCR Pharmaceuticals is asking regulators in Japan to extend the use of Temcell HS Injection to epidermolysis bullosa (EB) patients in that country as a wound healing treatment.

Temcell HS is a mesenchymal stem cell therapy that approved to treat acute graft versus host disease (aGVHD), a severe complication of allogeneic (donor-derived) bone marrow transplants, in Japan in 2015. In aGVHD, the donor’s immune cells react against the patient’s own tissues.

Traditionally found in bone marrow, mesenchymal stem cells are adult stem cells that can generate several types of cells.

According to a press release, Temcell was given EB patients via subcutaneously injection in an investigator-initiated clinical trial at Osaka University Hospital and showed “promising results.” Japanese regulators, based on these findings, designated the therapy an “orphan treatment” to offer incentives to advance its development for EB, a rare disease.

Mesoblast, the company for which JCR is licensed to act on its behalf in Japan, announced the regulatory application filing. The company specializes in mesenchymal lineage adult stem cells products, engineered to be “off-the-shelf” and readily available, Mesoblast states on its website. All are allogeneic, and cells from one donor may be used in various recipients without the need for matching, it adds.

JCR Pharmaceuticals is also planning to file for a label extension for an intravenously administered version of this cell product in Japan.

A Phase 3 clinical trial (NCT02336230) of one of its intravenously-delivered mesenchymal stem cell therapy, known as MSC-100-IV or remestemcel-L, tested the product in 55 children with steroid-refractory aGVHD. Mesoblast reports that the trial met its primary goal, that of more patients — 69% — achieving an overall response to the therapy at 28 days post-treatment compared to a protocol-defined historical control rate of 45%. Survival was  87% at 100 days post-treatment among responders.

Based on these reported results, Mesoblast plans to ask the U.S. Food and Drug Administration (FDA) to approve remestemcel-L as a treatment for acute graft-versus-host disease, it said in the release.

Epidermolysis bullosa is a group of hereditary disorders characterized by severe blistering of the skin and mucous membranes, and can also affect organs. Available treatments largely aim to prevent blisters or manage the wounds caused by this disease.

According to DEBRA International, a nonprofit advocating on behalf of the EB patients and their families and support research efforts, about 25,000 people have EB in the United States.

Under the agreement between the two companies, Mesoblast will have access to clinical data generated by JCR, so that Mesoblast employees can further develop and commercialize its investigational mesenchymal stem cell product remestemcel-L outside of Japan, as a possible treatment for EB and other diseases or conditions.

Like Temcell HS, remestemcel-L consists of cultured, cryopreserved mesenchymal stem cells derived from the bone marrow of healthy donors.