Cynata Begins Phase 2 Trial for CYP-001 in High-Risk aGvHD

Photo:Representative Image

Cynata Therapeutics Limited, a clinical-stage biotechnology company specializing 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 iPSC1-derived MSC2 product candidate for intravenous infusion, which previously generated very encouraging safety and efficacy results in a phase 1 clinical trial in steroid-resistant aGvHD.

The US FDA has cleared an Investigational New Drug (IND) application for CYP-001 and granted the product Orphan Drug Designation4 to treat 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”. CYP-001 is being investigated in this trial as a potential immune-modulating treatment for aGvHD.

This global phase 2 trial aims to enroll approximately 60 patients with high-risk aGvHD, who will be randomized to receive either steroids plus CYP-001 or steroids plus placebo. The trial has been approved to commence in Australia, the USA, and Turkey, and numerous clinical centers 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 in the clinical development journey of CYP-001 for aGVHD. We are still opening more clinics and expect to launch our first product in the second half of 2025, with enrollment completed by the end of this year. Cymerus, a regenerative medicine company, focuses on developing treatments based on its technology.