Celyad has reported a complete morphological leukemia-free status (MFLS) response in acute myeloid leukemia (AML) in the NKR CAR T-cell THINK study. Spontaneous remission in refractory/relapsed AML is extremely rare, so this is a significant result. Importantly, the response was achieved with no toxic preconditioning. CYAD-01 has shown limited toxicities to date. The clinical strategy has been updated to focus on AML and colorectal cancer. Additionally, with the approvals of Yescarta (Gilead) at a price of $373k and Kymirah (Novartis) at $475k, we have increased our expected price for NRK CAR T-cell therapy to $200k, formerly $150k. The revised strategy and price assumption change moves the indicative value to $122 per ADR, formerly $61 per ADR.
Celyad found one AML patient given 3x108 NKR CAR T-cells had a complete MLFS response. In effect, it was a near CRi; that is, a complete response with high but not normal blood cell levels. Treatment with NKR CAR T-cells has enabled him to move to stem cell transplant. Median survival of relapsed and refractory AML patients is typically less than four months (source Celyad, see AML treatment).
With a near complete response in AML and two stable disease cases at low dose in colorectal cancer, Celyad has decided to focus its development efforts on these two indications. The current THINK study in AML, MM and five solid cancers, including colorectal, will continue to find the optimal dose but with preferential recruitment of AML and colorectal patients. Celyad also intends to evaluate combination therapies. As the first such study, the SHRINK trial of CYAD-01 is ready to start recruitment in metastatic CRC in combination with FOLFOX chemotherapy.
Our previous approach to valuing the Celyad NKR CAR T-cells portfolio was to treat AML and MM as defined indications with a probability of 20% and to take a weighted average of the five solid tumor indications. We have now focused the valuation on AML at 25% probability and colorectal at 20% (adjusted from 10% in August). The C-Cure a cardiac indication is still seeking a partner so is now given a nominal value of $12m, formerly $191m. Rebasing the valuation to January 2018 gives a new indicative value of €$1,236m, formerly $616m (indicating $122 per ADR (formerly $61)). Management states that Celyad has cash to fund it through the first half of 2019. Additional cash might enable a broader and faster development of the clinical program given its promising current outlook.