Cynata Therapeutics Limited (ASX:CYP) welcomes approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) to proceed with its Phase 2 clinical trial of the Cymerus™ mesenchymal stem cell (MSC) product CYP-002 in patients with critical limb ischaemia (CLI).
CLI is an advanced stage of peripheral artery disease (PAD), a narrowing of arteries in the limbs which is a major risk factor for cardiovascular events such as heart attack.
Cynata chief operating officer Dr Kilian Kelly said approval of the Phase 2 clinical trial was a major step for treatment of the condition.
He said: “The approval of this Phase 2 clinical trial by the MHRA is a major advancement for the development of Cymerus MSCs.
“We look forward to working with our contract research organisation and clinical investigators to evaluate CYP-002 in this devastating condition, for which there remains an enormous unmet need.
“The approval also confirms the successful implementation of the company’s strategy to use the positive Phase 1 trial of Cymerus MSCs in graft versus host disease to support direct progression from preclinical to Phase 2 clinical studies for CLI and multiple other indications.”
Cynata anticipates conducting the clinical trial at multiple centres in the UK and Australia.
CLI prevalence increasing
The risk of PAD is increased in patients with diabetes, obesity, high blood pressure and in people who smoke and can often result in amputation of the affected limb.
Outcomes in CLI patients are typically poor and with CLI prevalence estimated to be approximately 2 million patients in the United States alone and likely to rise, as such, the value of the global CLI treatment market has been forecasted to reach US$5.4 billion by 2025.
Cynata is working to develop better treatment options to meet the growing commercial need.
Economic manufacture of products
Cymerus overcomes the challenges of other production methods by using induced pluripotent stem cells (iPSCs) and a precursor cell known as mesenchymoangioblast (MCA) to achieve economic manufacture of cell therapy products, including mesenchymal stem cells (MSCs), at commercial scale without the limitation of multiple donors.
In the Phase 1 trial, Cynata’s CYP-001 product met all clinical endpoints and demonstrated positive safety and efficacy data for the treatment of steroid-resistant acute graft-versus-host disease (GvHD).
The company plans to advance its Cymerus™ MSCs into Phase 2 trials for GvHD, critical limb ischemia and osteoarthritis.
The technology has also proved efficient in preclinical models of asthma, diabetic wounds, heart attack and cytokine release syndrome - a life-threatening condition stemming from cancer immunotherapy.