The first successful organ donation occurred in 1954 where a living donor gave a kidney to his identical twin. Since then, organ donation has become a routine practice with over 25,000 transplants conducted per year in US hospitals. Transplantation is far from simple, with an immense amount of preparation and aftercare required for recipients.

A common side effect of transplantation is acute graft versus host disease (aGvHD) where the new donor cells attack the recipient’s healthy cells. Acute GvHD is diagnosed within the first 100 days following transplant and can cause a wide range of symptoms such as skin rashes, gastrointestinal upset and eye irritation. The complication is usually treated with steroids and immunosuppressants, however these can have varying effects. For patients with steroid resistant GvHD choices are limited, resulting in mortality rates of over 90%. 

Clinical stage stem cell and regenerative medicine company, Cynata Therapeutics (ASX: CYP) is addressing aGvHD with their lead asset, CYP-001. The drug was proven to be safe and well tolerated in patients with steroid-resistant aGvHD in a recent Phase 1 trial. Now, the Company is preparing for a Phase 2 trial to investigate the efficacy and safety of the drug in combination with corticosteroids, and alone. 

60 patients who have had a recent stem cell transplant and subsequently diagnosed with the disease will be eligible for the trial. 100 days after the first dose, each patient will be evaluated, with longer-term follow ups scheduled for up to two years post dosing.

The trial has received regulatory clearance from the US FDA as well as Investigational New Drug (IND) status. A well recognised acronym in the medical research world, an IND is key for Cynata to drive value and strengthen ongoing commercial partnership opportunities. 

“Clearance of our IND application confirms that the FDA is satisfied with both the clinical and preclinical data as well as the manufacturing and quality control data on our product that we submitted in support of this application,” said Cynata Chief Operations Officer, Dr Kilian Kelly. 

“This achievement represents a hugely important milestone in the development of CYP-001 and our Cymerus platform. We had previously completed several positive interactions with the FDA, as well as securing Orphan Drug Designation for CYP-001 in 2018. However, the planned Phase 2 aGvHD trial will be our first clinical trial conducted in the US, which is the largest healthcare market worldwide. We look forward to commencing this Phase 2 study, which aims to build on the very encouraging Phase 1 clinical trial results.” 

The trial is expected to begin enrolling patients later this year, with results expected by early 2024.

Other assets from the Company are in trials for osteoarthritis (Phase 3) as well as respiratory failure and diabetic foot ulcers. 

The Company rounded out the March quarter with over $25 million in the bank, seeing them well funded for the trials to come.