Phase 2 GvHD Trial Begins with First Patient Treatment

Cynata Therapeutics, an Australian biotechnology firm, has made significant strides in its Phase 2 clinical trial for CYP-001, a treatment for high-risk acute graft versus host disease (aGvHD). The company has enrolled and administered the treatment to its first patient, marking a crucial step in the clinical development of CYP-001. This product is derived from induced pluripotent stem cells (iPSCs) and is designed for intravenous infusion.

Previously, CYP-001 showed promising safety and efficacy in a Phase 1 trial for steroid-resistant aGvHD. The US FDA has approved the Investigational New Drug (IND) application for CYP-001 and has granted it Orphan Drug Designation, which offers incentives such as extended marketing exclusivity, tax credits, and fee waivers.

aGvHD is a severe complication that can occur after bone marrow transplants, where the transplanted immune cells attack the recipient's tissues. The ongoing global Phase 2 trial plans to include around 60 patients with high-risk aGvHD. These patients will be randomly assigned to receive either a combination of steroids and CYP-001 or steroids and a placebo. The trial, which can be further explored using the identifier NCT05643638 on clinicaltrials.gov, has received approval to begin in Australia, the USA, and Turkey, with several clinical centers now open for patient recruitment. Notably, the first patient to be enrolled was in the United States.

Cynata's CEO, Dr. Kilian Kelly, expressed that the initiation of treatment for the first patient in the Phase 2 trial is a significant milestone. The company is in the process of opening more clinical centers and expects to complete patient enrollment by the end of the year, with the primary results expected to be released in the second half of 2025.

Cynata Therapeutics is dedicated to utilizing its proprietary Cymerus™ platform technology to develop stem cell and regenerative medicine therapies. The Cymerus™ technology leverages iPSCs and mesenchymoangioblasts (MCAs) to produce cell therapy products economically and at a commercial scale, overcoming the limitations posed by the need for multiple donors.

The company's lead candidate, CYP-001, has already demonstrated positive safety and efficacy data in treating steroid-resistant aGvHD in a Phase 1 trial. In addition to the ongoing Phase 2 trial for aGvHD, Cynata is also conducting trials for Cymerus products in osteoarthritis (Phase 3) and diabetic foot ulcers. Furthermore, trials for renal transplant are anticipated to start soon. The Cymerus technology has also shown potential in preclinical models for a range of diseases, including critical limb ischemia, idiopathic pulmonary fibrosis, asthma, heart attack, sepsis, acute respiratory distress syndrome, and cytokine release syndrome.

Cynata Therapeutics is encouraging its investors to adopt a paperless approach by registering with Automic Group, their designated registry service provider.