New CYP-001 GvHD Clinical Data in Nature Medicine

Cynata Therapeutics Limited, an Australian clinical-stage biotechnology firm, has announced the publication of two-year follow-up data from its Phase 1 clinical trial of CYP-001 in patients with steroid-resistant acute graft versus host disease (SR-aGvHD). This significant development was documented in the esteemed journal Nature Medicine.

CYP-001, Cynata's proprietary Cymerus™ product, is an off-the-shelf mesenchymal stem cell (MSC) therapeutic derived from induced pluripotent stem cells (iPSCs). It is administered intravenously and is explored as a potential treatment for immune modulation. SR-aGvHD is a severe complication that arises when immune cells from a donor's transplant attack the recipient’s body. It is particularly challenging to treat when patients do not respond to corticosteroids, the initial standard treatment.

The recently published paper highlights the sustained positive outcomes observed at the two-year mark. Among the 15 patients treated in the trial, the overall survival rate was impressive at 60%, with no reported serious adverse events related to the treatment. This outcome is notably better compared to historical data and other treatments. For instance, an Phase 3 trial supporting the approval of ruxolitinib reported 18-month survival rates of only 38% for the ruxolitinib group and 36% for the control group using the best available treatment. Historical data show that the prognosis for SR-aGvHD patients is generally poor, often with two-year survival rates below 20%.

The primary evaluation results of the Phase 1 trial, published in Nature Medicine in 2020, were promising. They reported Complete Response and Overall Response rates at Day 100 of 53% and 87%, respectively. The sustained positive outcomes at the two-year follow-up build upon these initial findings.

Professor John Rasko, the international coordinating Principal Investigator for the trial, emphasized the importance of this study in the broader context of cellular therapy. He pointed out that this was the first completed clinical trial globally involving iPSC-derived cells. The promising overall survival outcomes and lack of safety concerns are encouraging signs for the future of Cymerus MSCs. In addition to further trials for aGvHD, Professor Rasko anticipates the application of iPSC-derived cells in diverse human diseases and regenerative medicine.

Cynata Therapeutics, listed on the Australian Securities Exchange (ASX: CYP), is focused on developing therapies through its proprietary Cymerus™ platform. This platform utilizes iPSCs and mesenchymoangioblast (MCA) precursor cells to produce therapeutic products, such as MSCs, at a commercial scale without the limitations associated with multiple donors.

The company's lead product candidate, CYP-001, has shown positive safety and efficacy data for SR-aGvHD in its Phase 1 trial and is currently undergoing a Phase 2 clinical trial in the United States. Beyond SR-aGvHD, Cynata is also exploring the utility of its Cymerus technology in treating a variety of conditions, including osteoarthritis, diabetic foot ulcers, renal transplant, critical limb ischaemia, idiopathic pulmonary fibrosis, asthma, heart attack, sepsis, acute respiratory distress syndrome (ARDS), and cytokine release syndrome.

Cynata Therapeutics continues to encourage its investors to use paperless communications by registering with its designated registry service provider, Automic Group.