Please tell us about your clinical practice, background in plasma cell cancers; R/RMM and research in the space. How many patients with Multiple Myeloma do you treat,what is the first/second line of therapy? What % of patients do not respond well to first line?

How does a BCMA-targeted CAR T cell therapy work in increasing the persistence of CAR T cells; to enrich for T cells displaying memory-like phenotypes? What are Bluebird’s pipeline unique strengths/ particularities in the Multiple Myeloma space?

In its recent P1 data release, the company cites 10 of 12 (83%) patients in the 150 x 106 CAR+ T cells cohort demonstrated clinical response, of which 4 with a stringent complete response or complete response, and 6 with a very good partial response (VGPR). What is your opinion on these results?

Among the 10 confirmed responders, the median duration of response was 11.1 months (95% Confidence Interval (CI); 3.3 – not estimable) and none experienced disease progression. How important are those findings? How do they compare to the standard of care in R/RMM?

1. The company is to further recruit patients at the 450 x 106 CAR+ T cells dose cohort, where 4 of the 7 (57%) evaluable patients demonstrated clinical response; one with a sCR, two with a VGPR and one with a PR, with a median follow-up of 3.3 months (<1 – 6 months).
   
2. Can the worse results (despite increased dosage) in this cohort be explained by low enrollment nrs?

1. Even more, the 300 x 106 CAR+ T cells cohort, reported only 6/14 (43%) evaluable patients with demonstrated clinical response; 4 with a VGPR and 2 with a partial response (PR), with a median follow-up of four months (2 – 10 months). 
   
2. Based on this, what is your conclusion on the efficacy of the drug?
   
3. Is the good result on the low dosage cohort simply an outlier here?