Mesoblast, JCR Expand the Use of Mesenchymal Stem Cells for Treating Newborns with HIE

Mesoblast announced it has expanded its partnership with JCR Pharmaceuticals in Japan to include the use of mesenchymal stem cells (MSCs) for treating newborns who have neonatal hypoxic ischemic encephalopathy (HIE), a condition in which an unborn baby experiences shortage of oxygen supply to the brain during gestation.

This condition may lead to severe neurological impairments, seizures, developmental and motor delays, cerebral palsy, or even death.

Studies in animal models of HIE have shown that transplanting MSCs might be a viable option to prevent neurological and behavioral changes associated with HIE. MSCs are a type of adult stem cells that can be isolated from almost all tissues in the body and are able to give rise to different cell types.

TEMCELL HS, is a line of mesenchymal stem cells based on Mesoblast’s proprietary mesenchymal adult stem cell technology. It has been licensed to its partner, JCR Pharmaceuticals, for the treatment of children and adults with acute graft versus host disease (aGVHD, a serious complication in which the transplanted cells attack the host’s body), in Japan.

Under the terms of the partnership agreement, Mesoblast will receive royalties for each TEMCELL HS sale in Japan for all its licensed indications.

In March 2019, JCR Pharmaceuticals submitted an application seeking an extension of the marketing approval of TEMCELL HS to include an indication for wound healing among patients with epidermolysis bullosa (EB) in Japan.

Now the company has expanded the license agreement to be able to use TEMCELL HS for treating newborns with HIE in Japan. JCR Pharma is now planning to launch a clinical trial assessing the effects of TEMCELL HS in newborns with HIE to extend the product label for that indication. The trial is expected to start in July 2019.

Under the terms of the agreement, Mesoblast may use all safety and efficacy data generated by its partner to support the development and marketing of remestemcel-L in the U.S. Remestemcel-L is one of its lead mesenchymal stem cell product candidates for treatment of aGVHD, wound healing in EB, and now newborns with HIE.

“We are pleased with the strategy by our partner to expand TEMCELL marketing approval for indications beyond aGVHD. This supports our own strategic growth plans for our MSC product candidate remestemcel-L beyond aGVHD in children, including other pediatric indications such as HIE and adult conditions such as aGVHD, chronic GVHD, biologic-refractory Crohn’s disease, and osteoarthritis,” Silviu Itescu, CEO of Mesoblast, said in a press release.

Statistics estimate that around 6,000 babies with moderate-to-severe neonatal hypoxic ischemic encephalopathy are born each year in the U.S. It is possible these children may benefit from remestemcel-L, should its indication be expanded to include the condition.

In the meantime, Mesoblast has already submitted a Biologics License Application (BLA) to the U.S Food and Drug Administration (FDA) seeking the marketing approval of remestemcel-L for treating aGVHD in children.