Oxford Biomedica Regulatory News (OXB)

Oxford BioMedica notes the primary analysis results from the pivotal JULIET trial demonstrating that Kymriah™ (tisagenlecleucel) sustained complete responses at six months in adults with r/r DLBCL, a difficult-to-treat cancer

Oxford, UK - 11 December 2017: Oxford BioMedica plc ("Oxford BioMedica" or "the Group") (LSE:OXB), a leading gene and cell therapy group, today notes an announcement by Novartis on the updated results from the JULIET clinical trial demonstrating sustained responses with Kymriah™ (tisagenlecleucel) suspension for intravenous infusion, formerly CTL019, in adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL). Kymriah is a novel immunocellular therapy and a one-time treatment that uses a patient's own T cells to fight cancer.

The data from this pivotal trial, led by researchers from the University of Pennsylvania (Penn), show an overall response rate (ORR) of 53% (95% confidence interval [CI], 42% - 64%; p

Results from this study of Kymriah, the first-ever FDA-approved chimeric antigen receptor T cell (CAR-T) therapy, were included in the US and EU regulatory filings for Kymriah in r/r DLBCL and will be presented in an oral presentation at the 59th American Society of Hematology (ASH) annual meeting (Abstract #577; Monday, December 11, 7:00 AM EST)1.

Results of a cost-effectiveness analysis of Kymriah for the treatment of r/r B-cell ALL in the US will be presented in an oral presentation at the meeting (Abstract #609; Monday, December 11, 7:30 AM EST). The analysis showed that, based on the current US list price of $475,000, Kymriah is cost-effective compared to standard of care2.

In addition, results of another analysis to determine the potential societal value of Kymriah to patients with r/r ALL in the United Kingdom were presented in a poster presentation at the meeting (Abstract #1330; Saturday, December 9, 5:30 PM EST). Results show that therapies such as Kymriah have the potential to provide benefit to patients and society, particularly through gains in survival, contributing to productivity3.

In April 2017, the US Food and Drug Administration (FDA) granted Breakthrough Therapy designation to CTL019 based on data from the JULIET study. In October 2017, Novartis submitted an application to the FDA for CTL019 for the treatment of adult patients with r/r DLBCL who are ineligible for or relapse after ASCT. Novartis is also seeking approval from the European Medicines Agency (EMA) for CTL019 in pediatric and young adult patients with r/r B-cell ALL and adult patients with r/r DLBCL who are ineligible for or relapse after ASCT. Additional filings beyond the US and EU are anticipated in 2018.

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.  

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Notes for editors

About Kymriah

In August 2017, Kymriah became the first available chimeric antigen receptor T cell (CAR-T) therapy when it received FDA approval for children and young adults with B-cell acute lymphoblastic leukaemia (ALL) that is refractory or has relapsed at least twice. Kymriah is a novel immunocellular therapy and a one-time treatment that uses a patient's own T cells to fight cancer. Kymriah uses the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular expansion and persistence.

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.

References

1. Schuster S., et al. Primary Analysis of Juliet: A Global, Pivotal, Phase 2 Trial of CTL019 in Adult Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma. 59th American Society of Hematology Annual Meeting and Exposition. Abstract #577.

2. Hao Y., et al. Cost-Effectiveness Analysis of CTL019 for the Treatment of Pediatric and Young Adult Patients with Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia in the United States. 59th American Society of Hematology Annual Meeting and Exposition. Abstract #609.

3. Snider J., et al. The Economic Value of CTL019 Therapy for Pediatric Patients with Relapsed and Refractory Acute Lymphoblastic Leukemia in the United Kingdom. 59th American Society of Hematology Annual Meeting and Exposition. Abstract #1330.