CAR-T Therapy in Haematological Malignancy: Update Bulletin [July 2017]

This edition presents US and EU key opinion leader (KOL) views on recent developments in the CAR-T therapy space. Topics covered include expert opinions on Novartis receiving a positive recommendation from the FDA’s Oncologic Drugs Advisory Committee (ODAC) for approval of its anti-CD19 CAR-T cell therapy CTL019 (tisagenlecleucel-T), in patients aged three to 25 years with relapsed/refractory B-cell acute lymphoblastic leukaemia (ALL). KOLs provide their insights on the case of cerebral oedema reported for Kite Pharma’s KTE-C19 (axicabtagene ciloleucel) in the ZUMA-1 study in May 2017. Following the release of interim results from the Phase II JULIET study with CTL019 in patients with diffuse-large B cell lymphoma (DLBCL), KOLs also compare CTL019 and KTE-C19 in the DLBCL setting.

Business Questions:

• How do KOLs view the FDA’s ODAC vote in favour of approval for Novartis’ CTL019 in patients with ALL?
• Based on the ODAC discussion, what long-term safety issues concern KOLs the most?
• Are KOLs concerned about the risk of insertional oncogenesis and replication competent lentivirus (RCL)?
• What long-term follow-up will be required for CTL019 and are KOLs satisfied with 15 years of patient monitoring?
• Are KOLs satisfied with Novartis’ proposed workflow to manage cytokine release syndrome (CRS)?
• How do KOLs view the prophylactic management of CRS with anti-IL-6 therapies?
• What manufacturing issues concern KOLs the most and how will they be addressed?
• Are KOLs concerned about batch-to-batch variability and purity of CAR-T therapies?
• What are KOLs’ initial opinions on Juno Therapeutics/Celgene’s fixed-dose composition CAR-T therapies?
• How do KOLs compare production turnaround times for CTL019 and KTE-C19 so far?
• What are KOLs’ opinions on the case of cerebral oedema reported for KTE-C19 in ZUMA-1 and are they concerned?
• Following the release of the JULIET study data, how do CTL019 and KTE-C19 compare so far in terms of efficacy in DLBCL?