This edition presents leading US and EU key opinion leader (KOL) views on recent developments in the CAR-T therapy space. Topics covered include expert opinions on the FDA approvals of Novartis’ Kymriah (tisagenlecleucel-T; CTL019) and Gilead/Kite’s Yescarta (axicabtagene ciloleucel; KTE-C19) for the treatment of patients with paediatric acute lymphoblastic leukaemia (ALL) and adult diffuse large B-cell lymphoma (DLBCL) respectively. KOLs discuss the three-month data reported in the DLBCL setting for Juno Therapeutics’ and Celgene’s JCAR017 in the TRANSCEND study and also offer their candid insights on allogeneic CAR-T therapies such as Cellectis and Servier/Pfizer’sUniversal Chimeric Antigen Receptor T-cells (UCARTs) and the recent progress made in diseases such as multiple myeloma and acute myeloid leukaemia (AML).
Business Questions:
- According to the KOLs, how are Kymriah and Yescarta faring on the US market so far?
- What are the experts’ initial views on the cost of the two CAR-T therapies?
- In terms of distribution and centre capacity, how do the KOLs view the roll-out of the two CAR-T therapies? Are there any major setbacks to successful roll-out?
- What challenges lie ahead in terms of infrastructural and production capacity?
- Do the experts have any remaining concerns about the manufacturing processes for CAR-T?
- What challenges face the two CAR-T therapies if and when they are approved in Europe?
- What do KOLs think CAR-T companies can do to help in terms of further training, support and education?
- Are KOLs impressed with the three-month data for Juno and Celgene’s JCAR017 in the TRANSCEND study?
- How does JCAR017 match up to Kymriah and Yescarta so far in terms of efficacy and safety?
- Do the benefits of a defined composition CAR-T product outweigh the potential disadvantages?
- How do the KOLs view allogeneic CAR-T cells, in particular Cellectis/Servier/Pfizer’s UCART programme?
- What are KOLs’ main concerns about ‘off-the-shelf’ allogeneic CAR-T approaches?
- How do KOLs view the safety issues with UCART123 in patients with AML? Where does the opportunity for CAR-T exist in AML?
- With Bluebird Bio reporting early data for anti-BCMA CAR-T cells in multiple myeloma, how do the experts view the recent progress made with this novel CAR-T approach?
