UK MHRA approves Cynata Therapeutics for GvHD Clinical Trial

cynata-logoCynata Therapeutics, the Australian stem cell and regenerative medicine firm, has obtained approval from the UK MHRA to conduct its Phase 1 clinical trial of CYP-001 in patients with steroid-resistant graft-versus-host disease (GvHD).

CYP-001 is Cynata’s lead Cymerus mesenchymal stem cell MSC product.

Cynata plans to conduct the Phase 1 clinical trial, entitled “An Open-Label Phase 1 Study to Investigate the Safety and Efficacy of CYP-001 for the Treatment of Adults With Steroid-Resistant Acute Graft Versus Host Disease”, at a number of leading clinical centres in UK and Australia. Additional centres in other jurisdictions will be considered at a later date.

The trial aims to recruit around 16 participants who have undergone a bone marrow transplant or similar procedure, and were subsequently diagnosed with steroid-resistant Grade II-IV acute GvHD.

Cynata believes this will be the world’s first clinical trial involving a therapeutic product derived from allogeneic induced pluripotent stem cells (iPSCs).

Pluripotent stem cells are the most versatile cells, having the ability to reproduce themselves indefinitely, and have the ability to differentiate into any other type of cell in the body. iPSCs have very similar characteristics to embryonic stem cells, but with none of the ethical controversies associated as they are derived from adult cells as opposed to embryos.

There has been enormous interest globally in the development of iPSC-derived therapies for a number of years. Meaning, Cynata’s trial represents a major milestone in the field of regenerative medicine. Results from the clinical trial will provide further insight into the use of Cynata’s proprietary technology for the potential treatment of various diseases.

Cynata vice president of product development Kilian Kelly said: “We are delighted that the MHRA has approved our clinical trial. Not only does this enable us to start providing our highly promising therapy to patients with a particularly devastating disease, it also provides clear validation of our manufacturing process and preclinical development program, from one of the most highly regarded regulatory authorities worldwide.”


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