With an anticipated enrollment of 440 patients, it will be the world’s largest trial ever completed with an iPSC-derived cell therapeutic.
It’s official. Australian stem cell company Cynata Therapeutics is preparing to test its iPSC-derived cell therapeutic, CYP 004, in a Phase 3 clinical trial enrolling up to 440 patients. Led by the University of Sydney and funded by the Australian Government National Health and Medical Research Council (NHMRC), the historic trial will assess whether Cynata’s cell therapeutic, CYP-004, has the capacity to improve patient outcomes in osteoarthritis (OA).
CYP-004 is an allogeneic, iPSC-derived mesenchymal stem cell (MSC) product derived using Cynata’s proprietary Cymerus™ technology. The clinical trial is set to achieve two world-firsts:
- It will be the world’s first clinical trial involving an iPSC-derived cell therapeutic product to enter Phase 3.
- It will be the largest trial ever completed with an iPSC-derived cell therapeutic, with an anticipated enrollment of 440 patients.
Cynata’s iPSC-Derived MSCs
Cynata’s Cymerus™ technology utilizes iPSCs originating from an adult donor as the starting material for generating mesenchymoangioblasts (MCAs). It then differentiates these cells into mesenchymal stem cells (MSCs). Because Cynata is using iPSCs as its starting material for manufacturing therapeutic MSCs, it has the potential to solve a bottleneck in the therapeutic use of MSCs.
For the first time in history, Cynata’s is able to manufacture MSCs in unlimited quantities, in uniform batches, from a single donor, and at low cost – a powerful combination of advantages. This makes the company’s Cymerus™ technology a needed solution to traditional stem cell manufacturing challenges.
CYP-004 is one of several iPSC-derived cell therapeutics that Cynata Therapeutics has under development. Its other iPSC-derived cell therapetucis include CYP-001 for the prevention and treatment of GvHD in humans, CYP-002 for critical limb ischemia (CLI) and CYP-004 for osteoarthritis.
As stated by Cynata Therapeutics:
“The Phase 3 clinical trial of CYP-004 has now been formally approved by the University of Sydney Human Research Ethics Committee (HREC). The Company originally described this as a Phase 2 clinical trial, but it has since been determined that a Phase 3 trial will be performed, and that is reflected in the final HREC-approved version of the protocol.
Furthermore, the Company is pleased to announce that agreement has been reached on an expedited regulatory pathway for this trial. The Company previously announced (8 May 2020) that the trial sponsor, the University of Sydney, intended to utilise the Clinical Trial Exemption (CTX) clinical trial approval route for this trial. However, after further consultation between the Company, the University of Sydney and the Therapeutic Goods Administration (TGA), the TGA has advised that the trial can be conducted under the Clinical Trial Notification (CTN) scheme subject to Cynata supplying appropriate Good Manufacturing Compliance (GMP) compliance documentation to the trial sponsor.”
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To learn more about the expanding market for iPSCs, view the “Global Induced Pluripotent Stem Cell (iPS Cell) Industry Report – Market Size, Trends, and Forecasts.”
