Australian biopharmaceutical company Cynata Therapeutics has enrolled and treated the first patient in its Phase II study of CYP-001, an investigational therapy for high-risk acute graft versus host disease (aGvHD), in the US.

CYP-001 is an off-the-shelf, induced pluripotent stem cell (iPSC) derived mesenchymal stem cell product candidate intended for intravenous infusion.

Cymerus' stem cell platform technology overcomes the challenges of other production approaches with the help of iPSCs and mesenchymoangioblast, a precursor cell.

The global Phase II trial will enrol around 60 patients with high-risk aGvHD, who will be randomised to receive either steroids in combination with CYP-001 or steroids with a placebo.

Cynata has received approval to begin the trial in Australia, the US and Turkey. Several clinical centres in these countries are now open for patient recruitment.

CYP-001 previously demonstrated ‘very encouraging' safety and efficacy results in a Phase I clinical trial for steroid-resistant aGvHD.

The US Food and Drug Administration (FDA) has approved an investigational new drug application for CYP-001, which has also received product orphan drug designation for the treatment of aGvHD.

aGvHD is a severe complication that can occur after bone marrow transplants, where the donor's immune cells attack the recipient's tissues.

CYP-001 is being investigated as a potential treatment to modulate the immune response in aGvHD.

Cynata CEO Dr Kilian Kelly said: “The treatment of the first patient in this Phase II trial marks a milestone moment in the clinical development journey of CYP‑001 for aGVHD.

“We are continuing to open additional clinical centres, and we anticipate completion of enrolment by the end of this calendar year, with primary results available in the second half of 2025.”

Cymerus is a clinical-stage stem cell and regenerative medicine company that focuses on developing therapies based on its proprietary stem cell platform technology.