In This Article:
Two-year overall survival rate in patients with steroid-resistant acute GvHD was 60%
MELBOURNE, Australia, May 23, 2024 /PRNewswire/ -- Cynata Therapeutics Limited (ASX: "CYP", "Cynata", or the "Company"), a clinical-stage biotechnology company specialising in cell therapeutics, is pleased to announce the publication of two-year follow-up data of CYP-001 in patients with steroid-resistant acute graft versus host disease (SR-aGvHD) in the prestigious peer-reviewed journal Nature Medicine.
CYP-001 is Cynata's Cymerus™ off-the-shelf iPSC[1]-derived MSC[2] product for intravenous infusion, which is being investigated as a potential immune modulating treatment.
Graft versus host disease (GvHD) is a potentially life-threatening complication in recipients of bone marrow transplantation or similar procedures. It arises when immune cells in the transplant (the graft) attack the recipient's tissues (the host) as "foreign". SR-aGvHD occurs when there is no response to corticosteroid treatment, which is the standard first-line treatment for aGvHD.
The newly published paper reports sustained outcomes at the two-year follow-up in the Phase 1 clinical trial of CYP-001 in patients with SR-aGvHD. Key results include a two-year overall survival rate of 60% (9/15 patients), with no treatment-related serious adverse events or safety concerns identified. This survival rate compares very favourably to previously reported outcomes in SR-aGvHD. For example, in the Phase 3 study that supported approval of the drug ruxolitinib, the 18-month overall survival rates were only 38% in the ruxolitinib group and 36% in the "best available treatment" control group (survival at two years was not evaluable).[3] Historically the prognosis in patients with SR‑aGvHD has been very poor, with two-year overall survival rates below 20%.[4]
The details of the paper are as follows:
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Kelly K, Bloor AJC, Griffin JE, Radia R, Yeung DT, and Rasko JEJ. Two-year safety outcomes of iPSC-derived mesenchymal stromal cells in acute steroid-resistant graft versus host disease. Nat Med, 2024, https://www.nature.com/articles/s41591-024-02990-z
The two-year follow-up results build on the highly encouraging primary evaluation results at Day 100, which included Complete Response and Overall Response rates of 53% and 87%, respectively. The original paper summarising the primary evaluation results was published in Nature Medicine in 2020.[5]
Professor John Rasko, AO,[6] the international coordinating Principal Investigator for the trial, said:
"The publication of a second paper on this trial in such a high-impact journal is a testament to the importance of this project to the wider field of cellular therapy. This was the first ever completed clinical trial worldwide involving iPSC-derived cells of any kind. The overall survival outcomes and safety data in this group of patients are very promising, and we look forward to the continued development of Cymerus MSCs, in particular the ongoing global Phase 2 trial in aGvHD. [7] More broadly, our demonstration of the safe clinical use of iPSC-derived cells anticipates their application in diverse human diseases and regenerative medicine."