Kymriah demonstrates consistent efficacy and safety outcomes with real-world experience in children and young adults with r/r ALL.- Novartis

Novartis announced results from two analyses of real-world experience with Kymriah (tisagenlecleucel), the only CAR-T cell therapy approved in two distinct indications . These analyses are from a readout of a 15-year post-marketing study that add to and complement the rigor of the Kymriah pivotal trials with evidence of the Kymriah real-world experience in expanded groups of patients. When Kymriah was used in the real-world setting, efficacy and safety were consistent when compared to the pivotal trials, including the 24-month analysis of JULIET in adults with r/r diffuse large B cell lymphoma (DLBCL) and ELIANA in children and young adults with r/r B-cell acute lymphoblastic leukemia (ALL). The real-world experience data were presented at the 61st American Society of Hematology (ASH) annual meeting.

Real-world experience with Kymriah in children and young adults with r/r ALL : Efficacy outcomes were similar and safety outcomes appear to be more favorable in the real world setting compared to the ELIANA pivotal trial. Among 146 children and young adult patients with r/r ALL treated in the real world setting for whom three or more months of post-infusion outcomes were available, CR was 85% as compared to 82% in the ELIANA trial (n=79). Median follow-up in the real-world analysis was 6 months. In this analysis (safety set, N=154), the rate of grade 3 or higher CRS and neurologic events were 14% and 8%, respectively, as compared to 48% and 13% in the ELIANA clinical trial. The real-world analysis used the grading scales ASTCT for CRS and ICANs for neurologic events, whereas the ELIANA trial used the Penn Grading Scale for CRS and MedDRA SMQ for neurologic events..

�It is exciting to see how oncologists are using Kymriah and how patients are responding to it in routine clinical practice,� said Stephan A. Grupp, MD, PhD, Director of the Cancer Immunotherapy Program and Section Chief of Cell Therapy and Transplant at Children's Hospital of Philadelphia, and a Professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. �We are seeing broader efficacy data that replicate what we saw in the pivotal trial, and the collection of these data is ensuring that we are getting a clear view of adverse events when administering Kymriah.�

These data on the use of Kymriah in r/r pediatric ALL in the real-world setting were presented in a poster presentation at the ASH annual meeting (Abstract #2619; Sunday, December 8, 6:00 � 8:00 PM EST).

The collection of this real-world experience data was made possible by a collaboration between the CIBMTR (Center for International Blood and Marrow Transplant Research � the research collaboration between the National Marrow Donor Program /Be The Match and the Medical College of Wisconsin) and Novartis, developed to capture long-term follow-up of recipients of Kymriah who agree to participate in the registry. For patients whose cell viability was below 80%, product is provided through an established EAP program and long-term follow-up is captured through the CIBMTR. Globally, 90% patients who have been prescribed Kymriah have received the final manufactured product, either commercially, or when out of commercial specification..