Cynata unveils promising results in treating graft versus host disease with stem cells

Cynata Therapeutics ASX CYP trial stem cells treating graft versus host disease

Cynata Therapeutics (ASX: CYP) has unveiled “excellent” data from its “world first” clinical trial using its stem cell product CYP-001 in treating steroid resistant graft versus host disease.

After a patient has received a bone marrow transplant, graft versus host disease can present and is evident when the donated marrow or blood stem cells attack the recipient’s body. This can often be fatal.

Cohort A in the “world first” trial was enrolled in mid-November last year to study the impact of Cynata’s Cymerus mesenchymal stem cell CYP-001 treatment on patients who’ve had a bone marrow transplant that led to graft versus host disease.

Eight participants were enrolled in the study with the overall survival rate for patients at day 100 sitting at 87.5% – equating to seven out of eight patients remaining alive.

One patient in the study had passed from complications relating to pneumonia and the death was not considered to be connected to the treatment.

Despite the fatality, all patients showed improvement after receiving CYP-001, which was defined as lessening in the disease’s severity and symptoms.

Additionally, half of the patients achieving a complete response to CYP-001, which was classified as the absence of disease or symptoms.

According to Cynata, the study was deemed safe with zero adverse effects or events noted.

“We are pleased and excited by the data generated at this point,” Cynata vice president of product development Dr Kilian Kelly said.

“Steroid-resistant graft versus host disease is a horrific disease, which causes debilitating symptoms and a very high rate of mortality, so we are delighted to see such positive outcomes among participants of cohort A of this ground-breaking trial,” Dr Kelly added.

The cohort was evaluated for 100 days and each participant received dosages considered at the lower end of the effective range. The patients were administered an infusion on the first day and a second one a week later.

Next clinical study

In the upcoming cohort B study, the dosage will be increased to test the safety and efficacy of CYP-001 at higher levels.

This second cohort will also involve eight participants with people currently being recruited across the United Kingdom and Australia.

Cynata claims the global market for treating graft versus host disease will hit US$500 million by 2021.

“These results, when combined with our data across other target areas, demonstrate the potential of our Cymerus platform,” Cynata chief executive officer Dr Ross Macdonald commented.

Shares in Cynata had risen more than 7% to A$1.16 by mid-afternoon trade.