Oxford BioMedica, UK Cystic Fibrosis Gene Therapy Consortium, Boehringer Ingelheim and Imperial Innovations Form Partnership to Develop a Novel Gene Therapy Treatment for Cystic Fibrosis

Oxford, UK – 6 August 2018: Oxford BioMedica plc (LSE:Oxford Biomedica) (“Oxford BioMedica” or “the Group”), a leading gene and cell therapy group, today announces that it has entered into a process development collaboration agreement with the UK Cystic Fibrosis Gene Therapy Consortium (GTC) and Imperial Innovations to develop a long-term therapy for patients with cystic fibrosis (CF). Concurrently with this, a separate option and license agreement has been signed between Oxford BioMedica and Boehringer Ingelheim.

The collaboration will use a novel, replication deficient lentiviral vector, in an inhaled formulation, to selectively introduce a CFTR gene into the relevant target cell. This approach has demonstrated high gene transfer efficiency, and offers the possibility of repeated administration to maintain a therapeutic effect. In addition, the approach has the potential to address all of the more than 2,000 different, known gene mutations across patients equally well, and thus offer a disease-modifying treatment option for all patients.

Under the terms of the process development collaboration agreement, Oxford BioMedica will be responsible for process and analytical development, scale up of manufacture of the candidate and generation of material for toxicology studies. There is scope within the terms for the parties to establish a clinical supply agreement in the future. The collaboration may also include an evaluation of Oxford BioMedica’s Transgene Repression in vector Production (TRiP) System, and the development of stable producer cell lines for large scale production of the lentiviral vector.

In order to treat CF patients with a gene therapy, there is a requirement for a large volume of lentiviral vectors. Oxford BioMedica can produce large quantities of lentiviral vectors using its highly efficient GMP-compliant manufacturing process in bioreactors.

Under a separate option and license agreement between Oxford BioMedica and Boehringer Ingelheim, Oxford BioMedica has granted Boehringer Ingelheim an option to licence the exclusive global rights over Oxford BioMedica’s lentiviral vector technology to manufacture, register and commercialise this lentiviral vector-based gene therapy for the treatment of CF. The financial terms for the option and license agreement with Boehringer Ingelheim are undisclosed.

This collaboration brings together the UK’s leading CF academic and clinical groups within the GTC, who have already pioneered clinical development of other gene therapy programmes for CF and are recognised leaders in this important disease with high unmet medical need. Boehringer Ingelheim is a global, research-driven pharmaceutical company with a strong franchise in respiratory medicine and a depth of experience in developing novel pharmaceutical treatments.

John Dawson, Chief Executive Officer of Oxford BioMedica, said: “This novel three-way partnership brings together an unparalleled combination of clinical, scientific, manufacturing and commercial skills in an effort to develop new treatments and make a major contribution to the lives of patients affected by cystic fibrosis. The GTC has been working determinedly for over 15 years to get to this exciting point of forming a partnership with a global pharmaceutical company with respiratory expertise, Boehringer Ingelheim.

“Our contribution to this partnership reaffirms our leading position in the development and manufacture of lentiviral vector gene therapy products at large scale. We look forward to working with our new academic and industry partners.”

Commenting on the announcement, Deborah Gill, Professor of Gene Medicine at the Department of Medicine of Oxford University, said: “The UK CF Gene Therapy Consortium has, for the last 17 years, vigorously sought to establish whether gene therapy can become a clinically viable option for patients with CF. We have developed a novel viral vector-based product which is currently funded by the Health Innovation Challenge Fund (a partnership between the Wellcome Trust and the Department of Health and Social Care) and the Cystic Fibrosis Trust. It is with great pleasure that we now join forces with two world-class organisations. Boehringer Ingelheim will provide their multinational industry expertise, including a proven track record in the respiratory field, to drive the product towards the clinic, whilst Oxford BioMedica is the acknowledged leader in the field of lentiviral manufacturing. The GTC believes that this partnership provides CF patients with the optimal chance to establish gene therapy as routine clinical practice. We would like to take this opportunity to warmly thank all of our fundraisers who have supported us over many years.”

Commenting on the announcement, Clive R. Wood, Ph.D., Senior Corporate Vice President, Discovery Research at Boehringer Ingelheim,from Boehringer Ingelheim, said: “Through this collaboration, we are joining forces with some of the top talents in this disease space to propel treatment advances forward. Bringing together our existing expertise as a leader for nearly a century in the discovery and development of therapies that have advanced patient care in respiratory diseases with the gene therapy knowledge of our partners, we aim to unlock unprecedented opportunities for patients with this devastating disease, who are desperately waiting for better treatment options.”

Andrew Tingey, Director of Healthcare Licensing at Imperial Innovations, said: “The UK Cystic Fibrosis Gene Therapy Consortium shows the power of world-class academic groups collaborating to develop advanced potential therapies. We were delighted to have been chosen as the technology transfer partner for the GTC and have worked closely with them during the development of this potential new gene therapy, securing the intellectual property necessary to drive forward its commercial development and to support the collaboration with Boehringer Ingelheim and Oxford BioMedica. The combination of expertise and resources realised by this deal will give the project a unique opportunity to develop an advanced therapy that could significantly impact the lives of thousands of people living with cystic fibrosis, and we are delighted to have played the lead role in securing this partnership.”

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The information contained within this announcement is deemed by the Company to constitute inside information as stipulated under the Market Abuse Regulation (EU) No. 596/2014. Upon the publication of this announcement via the Regulatory Information Service, this inside information is now considered to be in the public domain.

Notes to editors

About Cystic Fibrosis
Cystic fibrosis (CF) is characterised by abnormal ion transport in the apical membrane of epithelial cells. This leads to altered mucus accumulation in the gastrointestinal and, reproductive tracts, liver, pancreas and most severely in the airways. Sweat glands produce excessively salty secretions, the pancreas is damaged, 10-15 % of neonates suffer from severe intestinal blockage termed meconium ileus and thick mucus secretions build up in the lungs leading to respiratory infections and lung damage. Current therapies include antibiotics, pancreatic enzyme supplements, products to partially correct ion channel function, high fat diets, physiotherapy and heart-lung transplants. The continued development of conventional therapies has greatly improved both the quality of life and the mean life expectancy of people with CF which has reached around 40 years. However, there is still no treatment for the underlying defect causing the disease and individuals with the disease suffer from a high therapeutic burden and a severely compromised quality of life. While treatments do exist, the disease continues to be fatal for most patients as existing treatments and treatments under development can only slow the progression of disease and patients will still ultimately succumb to lung failure.

About Oxford BioMedica
Oxford BioMedica (LSE:Oxford Biomedica) 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, Bioverativ, Sanofi, Axovant, Orchard Therapeutics, GC LabCell 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 320 people. Further information is available at www.oxfordbiomedica.co.uk

About the UK Cystic Fibrosis Gene Therapy Consortium
The UK Cystic Fibrosis Gene Therapy Consortium (GTC) comprises Imperial College London and the Universities of Oxford and Edinburgh. In 2001, the groups joined together as one organisation to share expertise and funding with a single common goal of making gene therapy for CF patients a clinical reality. The GTC has, to date, undertaken six Phase 1/2a proof-of-concept studies and the first Phase 2b gene therapy trial for CF which met its primary outcome. In parallel, it has developed the novel lentiviral vector which is the subject of the current tripartite agreement.

Further details and commentary can be found at www.cfgenetherapy.org.uk

About Boehringer Ingelheim
Improving the health and quality of life of patients is the goal of the research-driven pharmaceutical company Boehringer Ingelheim. The focus in doing so is on diseases for which no satisfactory treatment option exists to date. The company therefore concentrates on developing innovative therapies that can improve patients’ lives. In animal health, Boehringer Ingelheim stands for advanced prevention.

Family-owned since it was established in 1885, Boehringer Ingelheim is one of the pharmaceutical industry’s top 20 companies. Some 50,000 employees create value through innovation daily for the three business areas human pharmaceuticals, animal health and biopharmaceuticals. In 2017, Boehringer Ingelheim achieved net sales of nearly 18.1 billion euros. R&D expenditure, exceeding three billion euros, corresponded to 17.0 per cent of net sales.

As a family-owned company, Boehringer Ingelheim plans in generations and focuses on long-term success. The company therefore aims at organic growth from its own resources with simultaneous openness to partnerships and strategic alliances in research. In everything it does, Boehringer Ingelheim naturally adopts responsibility towards mankind and the environment.

More information about Boehringer Ingelheim can be found on www.boehringer-ingelheim.com or in our annual report: https://annualreport.boehringer-ingelheim.com.

About Imperial Innovations
Imperial Innovations is the Technology Transfer Office for Imperial College London, one of the UK’s leading universities. With a focus on societal and economic impact, Innovations develops and commercialises technologies developed by Imperial academics via intellectual property protection, technology licensing, and spinout formation & incubation. Combining deep understanding of science & technology with commercial acumen and a wide professional network, Innovations is able to gain insight into a technology’s potential and identify the best route to market. More information can be found at www.imperialinnovations.co.uk

About the Health Innovation Challenge Fund
The Health Innovation Challenge Fund is a parallel funding partnership between the Wellcome Trust and the Department of Health to stimulate the creation of innovative healthcare products, technologies and interventions and to facilitate their development for the benefit of patients in the NHS and beyond.