It's going to be a transformative year in 2026 for Cynata Therapeutics (CYP: $0.29)….well that's if things go well in at least one of its two clinical studies that will read out next year.
The most significant will be the Phase II study in GvHD with its allogeneic stem cell therapy. The company completed a Phase Ib study in 15 acute GvHD adult patients. The two-year survival achieved was 60% (nine patients). There were no serious adverse events, with 87% of patients improving by at least one grade in their disease classification (Overall Response Rate) and 53% achieving a complete response.
This compares with Jakafi (ruxolitinib) which was approved for aGvHD in 2019 in the US for patients over the age of 12. In a study involving 71 patients, 55% experienced infections, 51% had edemas, 49% a hemorrhage, and 32% incurred a bacterial infection. The ORR was 57% and the complete response rate was just 15%.
Earlier this month Cynata announced completion of recruitment into its Phase II GvHD study with 65 patients recruited. It is placebo controlled. The primary endpoint is the ORR at 28 days, with patients being followed out to 100 days. The study has taken just over two years to complete recruitment at 39 sites across Australia, the US and Europe. The company believes there are over 9,500 patients who could benefit from this treatment each year, with the only approved treatment being Jakafi.
Results from this study are due to be released in June next year.
Mesoblast achieved FDA approval for its allogeneic stem cell therapy, Ryoncil, in December last year in pediatric patients with steroid-refractory GvHD. Gross revenue for that product is currently tracking at US$120 million a year. Mesoblast is capitalized at $3.7 billion.
The second major study to readout next year will be in knee osteoarthritis. This is an externally run and funded trial, being conducted by Sydney University with an NHMRC grant. There were 321 patients who were recruited into the placebo-controlled trial. The final patient visit was completed last month, with results also expected in the second quarter of next year.
Results may also start to emerge from a third study, also being conducted externally (by the academic hospital LUMC in the Netherlands), in kidney transplant recipients. It's open label, with the aim being to reduce the reliance on the immune system rejection therapy tacrolimus, which has side effects and can be toxic to the kidneys.
Whilst the benefits of stem cell therapies has been widely investigated in smaller studies with stem cells derived using various approaches, what is lacking are larger studies with stem cells derived using reproducible therapies such as that being commercialized by Cynata.
Cynata is capitalized at $65 million with a proforma cash balance of $4.9 million at the end of September (including a tax rebate received since).
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