Celyad SA (NASDAQ:CYAD): Data at ASH on 3 December confirmed that CYAD-01 T-cells can destroy acute myeloid leukaemia (AML) blast (cancer) cells. In eight reported AML patients, there was a 62% (5/8) response rate across three dose levels. As preconditioning was not used, this rate is impressive. Safety was excellent with only one dose-limiting toxicity event at a high dose. The good safety profile should now enable faster progress to determine the optimal dose schedule. Further data are promised in H119, probably mid-year. A study combining CYAD-01 with preconditioning is underway and may be crucial. The indicative value is still €1,090m (€89 per share) pending further data.
Lots of detail but no clear dose response in AML
In the reported open-label THINK study to date, 14 patients were dosed of whom eight were relapsed or refractory (r/r) cases, so small numbers to date. AML is a very aggressive and mutationally varied cancer affecting individuals usually from their late 60s. Three r/r AML patients showed complete remission (38%), but due to prior chemotherapies, none had a full haematological recovery. One, treated at the low dose (3x108) in November 2017, proceeded to a stem cell transplant; the best outcome to date. Long-term persistence of CYAD-01 cells is needed for disease control, but one course (three infusions) at a low dose is insufficient to control blasts for more than a few months at best. Multiple CYAD-01 courses are needed. Crucially, the NKG2D receptor used in CYAD-01 is not immunogenic so CYAD-01 can be repeatedly dosed. All patients showed high levels of the NKG2D ligands targeted by CYAD-01, especially the heterogeneous MICA and MICB ligands.
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