CTL019 receives FDA approval for paediatric, young adult r/r B-cell ALL

29 August 2017

London, UK – 30 August 2017: Oxford BioMedica plc (“Oxford BioMedica” or “the Group”) (LSE:OXB), a leading gene and cell therapy group, today notes that the US Food and Drug Administration (FDA) has approved Novartis’ CAR-T cell therapy CTL019 (tisagenlecleucel, brand name KymriahTM) for the treatment of relapsed and refractory (r/r) paediatric and young adult patients with B-cell acute lymphoblastic leukaemia (ALL).

Oxford BioMedica is the sole manufacturer of the lentiviral vector that encodes CTL019. The Group signed an agreement with Novartis in July 2017 for the commercial and clinical supply of lentiviral vectors used to generate CTL019 and other undisclosed CAR-T products, for which Oxford BioMedica could potentially receive in excess of $100m from Novartis over the next three years.  As announced in October 2014, Oxford BioMedica will also receive undisclosed royalties on potential future sales of Novartis CAR-T products.

John Dawson, Chief Executive Officer of Oxford BioMedica, commented: “We are delighted that the FDA has now approved CTL019. This innovative immunocellular therapy will offer critically ill patients with an aggressive disease a new treatment option. Oxford BioMedica will supply the lentiviral vector encoding CTL019 and we look forward to continuing our collaboration with Novartis, both for commercial supply of CTL019, as well as bioprocessing and manufacturing for the clinical development of their other CAR-T products.”

– Ends –

Notes for editors:
About Oxford BioMedica

Oxford BioMedica (LSE:OXB) is a leading gene and cell therapy group focused on developing life changing treatments for serious diseases.  Oxford BioMedica and its subsidiaries (the “Group”) have built a sector leading lentiviral vector delivery platform (LentiVector®), which the Group leverages to develop in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology and CNS disorders. The Group has also entered into a number of partnerships, including with Novartis, Sanofi, GSK, Orchard Therapeutics and Immune Design, through which it has long-term economic interests in other potential gene and cell therapy products.  Oxford BioMedica is based across several locations in Oxfordshire, UK and employs more than 280 people. Further information is available at www.oxfordbiomedica.co.uk.

About CTL019 (tisagenlecleucel, brand name KymriahTM)
CTL019 was first developed by the University of Pennsylvania (Penn) and uses the 4-1BB costimulatory domain in its chimaeric antigen receptor to enhance cellular responses as well as persistence of CTL019 after it is infused into the patient, which may be associated with long-lasting remissions in patients. In 2012, Novartis and Penn entered into a global collaboration to further research, develop and commercialise CAR-T cell therapies, including CTL019, for the investigational treatment of cancers. In close collaboration with Novartis and Penn, Children’s Hospital of Philadelphia (CHOP) was the first institution to investigate CTL019 in the treatment of paediatric patients and led the single site trial.

The FDA approval is based on the Novartis-led ELIANA study (NCT02435849). Findings from a US multi-centre trial and a single site trial examining the safety and efficacy of CTL019 among paediatric and young adult patients with r/r B-cell ALL also supported the approval.