12 April 2016

The Cell and Gene Therapy Catapult, the UK organisation dedicated to the growth of the UK cell and gene therapy industry by bridging the gap between scientific research and commercialisation, alongside UCL Business PLC (UCLB) and Imperial Innovations, today announces a positive interim review in the phase I/II trial conducted by Catapult Therapy TCR using a T cell therapy to target acute myeloid leukaemia (AML).

The review has been conducted by the Data Safety Monitoring Board (DSMB), an independent panel of specialists in the field. The reviewed data shows that in the first cohort comprising 3 patients treated, there was a good safety profile with no serious adverse events (SAEs) related to the WT1-TCR T cell therapy. Importantly, the therapy also met the protocol specified requirements for cell persistence, showing that the WT1 targeted T cells can survive and expand in patients.

Passing this first interim review has enabled the clinical trial to enter its second phase. Dosing has now successfully started in the second cohort of patients in the trial which allows patients to receive a higher dose of the gene modified WT1 TCR T cells.

The therapy uses TCR gene-modified T cells to target WT1-overexpressing cells. It involves modification by gene therapy of the patient’s own T cells, so that they may recognise and destroy WT1-expressing cells when infused back into the body. It shows potential in disorders such as acute myeloid leukaemia and myelodysplastic syndrome and has been ranked by the US National Cancer Institute (NCI) as the number 1 target for cancer immunotherapy.

The trial is operated by Catapult Therapy TCR Limited, a company formed by the Cell and Gene Therapy Catapult with UCLB and Imperial Innovations to develop the T cell therapy, and is supported by the NIHR Biomedical Research Centre (BRC) at University College London Hospitals, with manufacturing supported by the NIHR BRC Great Ormond Street Hospital (GOSH). Pre-clinical development of the TCR gene therapy programme was supported by Bloodwise, previously Leukaemia and Lymphoma Research. In August 2015 the Cell and Gene Therapy Catapult appointed Cellular Therapeutics Limited (CTL), as a manufacturer for the clinical trial.

Developing TCR gene-modified T cells to target WT1-overexpressing cells continues to show promise as an immunotherapy for a range of cancers including the trials that we have underway in acute myeloid leukaemia and myelodysplastic syndrome,” said Keith Thompson, CEO, the Cell and Gene Therapy Catapult. “The progress of the WT1 TCR clinical trials supported by the positive DSMB review, is encouraging and we look forward to expanding these trials across the UK and Europe. Further results are expected during 2017.

We are delighted with the progress of this clinical trial in acute myeloid leukaemia,” said Professor Emma Morris, UCL Institute of Immunity and Transplantation, Chief Investigator and co-inventor as well as Director of the Infection, Immunity and Inflammation Programme at the National Institute for Health Research University College London Hospitals Biomedical Research Centre. “This is an important area of unmet medical need and we are progressing with the next stages of recruitment now ongoing in the second cohort of the AML trial.

We are pleased to note the progress of this innovative therapy,” said Tony Hickson, Managing Director of Technology Transfer at Imperial Innovations. “Catapult Therapy TCR is a great example of collaboration between UK universities and technology transfer organisations and we look forward to the results of forthcoming trials.

About the Cell and Gene Therapy Catapult
The Cell and Gene Therapy Catapult was established in 2012 as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 100 employees focusing on cell and gene therapy technologies, we work with our partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. We offer leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. We aim to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. Regenerative medicine is one of the UK government’s “eight great technologies” that support UK science strengths and business capabilities.  The Cell and Gene Therapy Catapult works with Innovate UK.

For more information go to ct.catapult.org.uk or visit www.gov.uk/innovate-uk

About UCLB
UCL Business PLC (UCLB) is a leading technology transfer company that supports and commercialises research and innovations arising from UCL, one of the UK’s top research-led universities. UCLB has a successful track record and a strong reputation for identifying and protecting promising new technologies and innovations from UCL academics. UCLB has a strong track record in commercialising medical technologies and provides technology transfer services to UCL’s associated hospitals; University College London Hospitals, Moorfields Eye Hospital, Great Ormond Street Hospital for Children and the Royal Free London Hospital. It invests directly in development projects to maximise the potential of the research and manages the commercialisation process of technologies from laboratory to market.

For further information, please visit www.uclb.com – Twitter @UCL_Business

About Imperial Innovations
Imperial Innovations Group plc (“Innovations”) creates, builds and invests in pioneering technology companies and licensing opportunities developed from outstanding scientific research from the ‘Golden Triangle’, the geographical region broadly bounded by London, Cambridge and Oxford. This area has an unrivalled cluster of outstanding academic research and technology businesses, and is home to four of the world’s top 10 universities, as well as leading research institutions, the cream of the UK’s science and technology businesses and many of its leading investors.

Innovations supports scientists and entrepreneurs in the commercialisation of their ideas, through the licensing of intellectual property, by leading the formation of new companies, by recruiting high-calibre management teams and by providing investment and encouraging co-investment. Innovations remains an active investor over the life of its portfolio companies, with the majority of Innovations’ investment going into businesses in which it is already a shareholder.

Since becoming a public company in 2006, Innovations has raised more than £430 million of equity from investors, which has enabled it to invest in some of the most exciting spin-outs to come out of UK academic research. In addition, the Group has agreed £80.0 million in loan facilities from the European Investment Bank (EIB).

Between Innovations’ admission to AIM (August 2006) and 31 July 2015, Innovations had invested a total of £236.8 million across its portfolio companies, which had raised collectively investment of £1.3 billion.

About the National Institute for Health Research (NIHR)
The National Institute for Health Research (NIHR) is funded by the Department of Health to improve the health and wealth of the nation through research. The NIHR is the research arm of the NHS. Since its establishment in April 2006, the NIHR has transformed research in the NHS. It has increased the volume of applied health research for the benefit of patients and the public, driven faster translation of basic science discoveries into tangible benefits for patients and the economy, and developed and supported the people who conduct and contribute to applied health research. The NIHR plays a key role in the Government’s strategy for economic growth, attracting investment by the life-sciences industries through its world-class infrastructure for health research. Together, the NIHR people, programmes, centres of excellence and systems represent the most integrated health research system in the world.

For further information, visit the NIHR website: www.nihr.ac.uk