Cynata’s CYP-001 Stem Cell Therapy Meets Expectations In Phase 1 Graft-Versus-Host Disease Trial

CYP-001 phase I trial results. Source: Cynata Therapeutics Limited.
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Cynata Therapeutics Limited is an Australian clinical-stage stem cell and regenerative medicine company founded in 2003 that is developing a therapeutic stem cell platform technology, Cymerus™, originating from the University of Wisconsin-Madison, a world leader in stem cell research..

CYP-001, Cynata’s lead Cymerus™ mesenchymal stem cell (MSC) product candidate, met all clinical endpoints in a Phase 1 trial for the treatment of steroid-resistant acute graft-versus-host disease (GvHD).

The proprietary Cymerus™ technology addresses a critical shortcoming in existing methods of production of mesenchymal stem cells (MSCs) for therapeutic use, which is the ability to achieve economic manufacture at commercial scale. Cymerus™ utilises induced pluripotent stem cells (iPSCs) to produce a particular type of MSC precursor, called a mesenchymoangioblast (MCA).

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Cymerus™ provides a source of MSCs that is independent of donor limitations and an “off-the-shelf” stem cell platform for therapeutic product use, with a pharmaceutical product business model and economies of scale. This has the potential to create a new standard in the emergent arena of stem cell therapeutics, and provides both a unique differentiator and an important competitive position.

Graft-versus-host disease (GvHD) is a complication that can occur after a bone marrow transplant or similar procedure, when the donor’s immune cells (from the “graft”) attack the recipient of the transplant (the “host”). The only approved treatment for GvHD is corticosteroid therapy, which is typically only effective in about 50 percent of patients. When GvHD fails to improve or worsens despite steroid treatment, patients are described as having steroid-resistant GvHD. The prognosis for these patients is poor, with mortality rates in excess of 90 percent.

“Cynata is the only company with the ability to produce allogeneic MSC therapies at commercial scale without the limitation of multiple donors. The positive data support the potential of Cymerus to generate ‘off-the-shelf’ stem cell therapies for multiple diseases providing significant opportunities for monetisation and commercial partnerships.  The completion of this trial marks a major achievement not only for Cynata but also as a world-first for iPSC-derived therapeutic products.” – Dr Ross Macdonald, Cynata’s Chief Executive Officer