Cynata Therapeutics Limited (ASX: CYP), an Australian clinical-stage biotechnology company specialising in cell therapeutics, has announced the disappointing primary evaluation results from its Phase 2 clinical trial of CYP-001. The company develops therapies based on its proprietary Cymerus™ therapeutic stem cell platform technology. The trial investigated CYP-001, an induced pluripotent stem cell (iPSC)-derived mesenchymal stem cell (MSC) product for intravenous use, in patients suffering from high-risk acute graft versus host disease (HR-aGvHD). Following the completion of the 100-day primary evaluation period, the company has made the decision to terminate the trial early due to the outcomes.
The key findings revealed no significant differences between the active group, which received steroids plus CYP-001, and the control group, which received steroids plus placebo. This lack of differentiation was observed across both primary and key secondary endpoints, including Day 28 Overall Response Rate (ORR) and Complete Response Rate (CRR), as well as Day 100 ORR, CRR, durable ORR, and overall survival rate. Encouragingly, no new safety concerns were identified, with a similar adverse event profile noted between both treatment groups. The Phase 2 trial enrolled 65 patients with HR-aGvHD across clinical centres in Australia, the USA, and Europe.
Dr Kilian Kelly, Cynata’s CEO and Managing Director, expressed profound disappointment with the outcome, stating that the results had fallen well short of expectations despite positive findings in the earlier Phase 1 clinical trial of CYP-001 in aGvHD. Cynata Therapeutics remains under voluntary suspension from the ASX as it awaits the results of its Phase 3 SCUlpTOR trial for CYP-004, targeting osteoarthritis of the knee. These results are anticipated to be announced later this week, with the company’s future course of action closely watched by investors.
