Australian clinical-stage stem cell and regenerative medicine company Cynata Therapeutics (ASX: CYP) has received confirmation from its partner Leiden University Medical Centre (LUMC) that the first patient has been treated with CYP-001 in the Phase 1/2 Nereid kidney transplant clinical trial.
CYP-001 is Cynata’s Cymerus iPSC1-derived MSC2 product candidate for intravenous use. The trial is being funded and managed by LUMC, under the supervision of Prof Ton Rabelink and Dr Siebe Spijker, while Cynata will supply CYP-001 for use in the trial. Importantly, Cynata will retain full commercial rights for this indication.
The primary objective of the trial is to study the safety and efficacy of CYP-001 – to determine if administering MSCs after kidney transplantation allows patients to take a reduced amount of the immunosuppressant drug tacrolimus, which is used to prevent the rejection of transplanted organs. Although tacrolimus is effective at preventing rejection of transplanted kidneys, it is associated with significant toxicity, including increased risk of serious infections, cancer, diabetes, and kidney damage.
Consequently, there is an urgent need to identify and evaluate safer methods of preventing organ transplant rejection. If it can be demonstrated that Cymerus MSC-based therapy allows patients to stop taking tacrolimus, or reduce tacrolimus dosage, this would be expected to reduce the level of toxicity that they experience. Close to one hundred thousand kidney transplants are performed worldwide every year.
The trial will seek to recruit a total of up to 16 patients who have undergone a kidney transplant. The first six patients will receive either one (n=3) or two (n=3) infusions of CYP-001, in addition to standard treatment. Subject to favourable safety review of the initial cohorts, a further ten patients will receive two infusions of CYP-001, followed by tacrolimus dose reduction.
Prof Rabelink and colleagues have previously published encouraging data from a clinical trial in which MSCs derived from bone marrow were used in a similar way. They found that early tacrolimus withdrawal with MSC therapy was safe, without increased rejection, and concluded that this is a potentially useful approach after kidney transplantation.
“This trial will help build the growing body of data on the use of Cymerus iPSC-derived MSCs in a wide range of clinical indications. There are clear parallels between use of CYP-001 in kidney transplant recipients, and use of CYP-001 in graft versus host disease, which led to very promising safety and efficacy outcomes in a completed Phase 1 clinical trial,” Dr Kilian Kelly, Cynata’s CEO and Managing Director, said.
“We look forward to continuing to work with Prof Rabelink, Dr Spijker and the wider team at LUMC on this important project.”
