First Patient Treated in Phase 2 GvHD Trial

06 March 2024 | Wednesday | News

Cynata Therapeutics' innovative approach using induced pluripotent stem cell-derived mesenchymal stromal cells receives Orphan Drug Designation, signaling a new horizon in combating acute steroid-resistant conditions.
Image Source : Public Domain

Image Source : Public Domain

Cynata Therapeutics Limited (ASX: "CYP", "Cynata", or the "Company"), a clinical-stage biotechnology company specialising in cell therapeutics, confirms that the first patient has been enrolled and treated in its Phase 2 clinical trial of CYP-001 in high-risk acute graft versus host disease (aGvHD). 

CYP-001 is Cynata's Cymerus™ off-the-shelf iPSC[1]-derived MSC[2] product candidate for intravenous infusion, which previously generated very encouraging safety and efficacy results in a Phase 1 clinical trial in steroid-resistant aGvHD.[3] The US FDA has cleared an Investigational New Drug (IND) application for CYP-001 and granted the product Orphan Drug Designation[4] for the treatment of aGvHD.

aGvHD is a potentially life-threatening complication of bone marrow transplants or similar procedures. It arises when immune cells in the transplant (the graft) attack the recipient's tissues (the host) as "foreign". In this trial, CYP-001 is being investigated as a potential immune modulating treatment for aGvHD.

This global Phase 2 trial aims to enrol approximately 60 patients with high-risk aGvHD, who will be randomised to receive either steroids plus CYP-001, or steroids plus placebo. Additional details on the trial may be found at using identifier NCT05643638. The trial has been approved to commence in Australia, the USA and Turkey, and numerous clinical centres across those jurisdictions are now open for recruitment. The first patient was enrolled in the USA.

Dr Kilian Kelly, Cynata's Chief Executive Officer, said:
"The treatment of the first patient in this Phase 2 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."


[1] iPSC = induced pluripotent stem cell

[2] MSC = mesenchymal stem (or stromal) cell

[3] Bloor AJC, et al. Production, safety and efficacy of iPSC-derived mesenchymal stromal cells in acute steroid-resistant graft versus host disease: a phase I, multicenter, open-label, dose-escalation study. Nat Med. 2020;26(11):1720-1725.

[4] Orphan Drug Designation qualifies Cynata for incentives including extended marketing exclusivity, tax credits and fee waivers.


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