Oxford Biomedica Stock

Oxford BioMedica plc, a biopharmaceutical company, operates in the gene and cell therapy biotechnology sector.

The company has a primary position in lentiviral vector and cell therapy research, development and production. Gene and cell therapy is the treatment of disease by the delivery of therapeutic DNA into a patient’s cells. Gene and cell therapy has the potential to transform medicine, providing long term and potentially curative treatment options for a range of diseases.

The company expects various therapies, primarily ex vivo cell therapy treatments, such as Novartis’s CTL019 that the company is involved with. The company has built its LentiVector platform of technologies and capabilities with which the company designs, develops, and produces gene and cell-based medicines for the company and its partners.

The company has product-related partnerships with Novartis, Immune Design, and Orchard Therapeutics; licensed products and technology rights to Sanofi and GSK; and a R&D collaboration with Green Cross LabCell. And the company has its own proprietary pipeline of gene and cell therapy products addressing neurodegenerative and ocular diseases, and a range of cancers, for which there are either no treatments or where therapy remains inadequate.

LentiVector Platform

The company’s LentiVector platform technology is an advanced lentiviral vector based gene delivery system, which is designed to overcome the safety and delivery problems associated with earlier generations of vector systems. The technology could stably deliver genes into cells with approximately 100% efficiency and could integrate genes into non-dividing cells, including neurons in the brain and retinal cells in the eye. In such cell types, studies suggest that gene expression could be maintained indefinitely. The LentiVector platform technology also has a larger capacity than most other vector systems and could accommodate multiple therapeutic genes.

In-House Products

OXB-101/OXB-102: Parkinson’s Disease

OXB-102 is a gene-based treatment for Parkinson’s disease, a progressive movement disorder caused by the degeneration of dopamine producing nerve cells in the brain. OXB-102 uses the company’s LentiVector platform technology to deliver the genes for three enzymes that are required for the synthesis of dopamine. The product is administered locally to the region of the brain called the striatum, converting cells into a replacement dopamine factory within the brain, thus replacing the patient’s own lost source of the neurotransmitter.

OXB-101 was the first generation version of this product and OXB-102 is the second generation. OXB-101 (ProSavin) showed encouraging efficacy and four-year duration in a Phase I/II clinical study, which provides great encouragement for OXB-102.

OXB-102 has delivered compelling proof-of-concept results in a ‘gold standard’ model of Parkinson’s disease indicating potency 5-10 times greater than OXB-101, and the company has completed manufacturing of GMP clinical study material in preparation for the next stage of development.

OXB-201: ‘Wet’ Age-Related Macular Degeneration (AMD)

OXB-201 is a gene-based treatment for neovascular ‘wet’ AMD and diabetic retinopathy (DR). OXB-201 intends to preserve and improve the vision of patients through anti-angiogenesis, blocking the formation of new blood vessels. The product uses the company’s LentiVector platform technology to deliver two anti-angiogenic genes, endostatin and angiostatin, directly to the retina.

In 2016, data was presented at the Association for Research in Vision and Ophthalmology (ARVO) conference demonstrating that lentiviral vector gene expression measured in the eyes of patients treated in the Phase I/II study continued without significant decline for approximately four years.

OXB-202: Corneal Graft Rejection

OXB-202 is a gene-based treatment for the prevention of corneal graft rejection. Corneal grafting arises from a need to remove and replace pathology arising in the cornea causing ‘clouding’. Although one of the successfully transplanted tissues, various grafts are rejected due to vascularisation.

OXB-202 uses the company’s LentiVector platform technology to deliver endostatin and angiostatin ex vivo to donor corneas prior to transplant to block vascularisation and to prevent graft rejection. The company has been working on appropriate regulatory approval for the initiation of a Phase I/II clinical study in the U.K.

5T4 Tumour Antigen

The 5T4 tumour antigen is a protein found on various common types of solid cancer. It is a target for novel anti-cancer interventions given its restricted expression in normal tissues and its high prevalence on the surface of both primary and metastatic cancerous cells. The 5T4 tumour antigen was identified through research into the similarities between the development of the placenta during pregnancy and the progression of cancer. 5T4 is produced by both cancerous cells and also by placental and foeta cells, suggesting that the process of immunological escape in pregnancy and cancer is based on similar mechanisms.

OXB-302 (CAR-T5T4): Cancer

OXB-302 intends to destroy cancerous cells expressing the 5T4 tumour antigen. It uses the company’s LentiVector platform and 5T4 antigen to target cancer cells expressing 5T4 tumour antigen expressed on the surface of various solid tumours and some haematological malignancies.

OXB-302 has completed pre-clinical studies, which have demonstrated proof of concept in both in vitro and in vivo industry-standard models.

OXB-301 (MVA-5T4): Cancer

OXB-301 is a therapeutic vaccine that stimulates the immune system to destroy cancerous cells expressing the 5T4 tumour antigen, which is present on various solid tumours. The product is based on an attenuated modified vaccinia virus Ankara (MVA), engineered to deliver the 5T4 antigen. Vaccinia viruses are commonly used as delivery systems for the development of antigen-specific vaccines.

In February 2017, the clinical investigators leading the open-label Phase I/II clinical trial in 53 patients with advanced colorectal cancer (TaCTiCC) presented a poster at the American Society of Clinical Oncology and Society for Immunotherapy of Cancer (ASCO-SITC) symposium.

Partnership Products

Sanofi (SAR 422459): Stargardt Disease

SAR 422459 is a gene-based therapy for the treatment of Stargardt disease. The disease is caused by a mutation of the ABCR gene, which leads to the degeneration of photoreceptors in the retina and vision loss. SAR 422459 uses the company’s LentiVector platform technology to deliver a corrected version of the ABCR gene. A single administration of the product directly to the retina could provide long-term or potentially permanent correction. SAR 422459 has continued to make progress in their Phase I/II clinical studies.

Sanofi (SAR 421869): Usher Syndrome Type 1B

SAR 421869 is a gene-based therapy for the treatment of Usher syndrome 1B. The disease is caused by a mutation of the gene encoding myosin VIIA (MY07A), which leads to progressive retinitis pigmentosa combined with a congenital hearing defect. SAR 421869 intends to address vision loss due to retinitis pigmentosa by using the company’s LentiVector platform technology to deliver a corrected version of the MYO7A gene. A single administration of the product could provide long-term or potentially permanent correction. SAR 421869 has continued to make progress in their Phase I/II clinical studies.

Novartis (CTL019)

In 2016, Novartis announced the results of the ELIANA study, which evaluated the efficacy and safety of CTL019 in relapsed/refractory (r/r) paediatric and young adult patients with B-cell acute lymphoblastic leukaemia (ALL). Novartis has indicated their intention to submit CTL019 for approval by the U.S. regulatory authority in early 2017.

Immune Design (LV305)

In 2016, Immune Design expanded its collaboration with Oxford BioMedica to include a license for the use of lentiviral vector-based products in the in vivo treatment and prevention of cancer. Immune Design is progressing gene therapy products targeting NY-ESO-1 expressing tumours with clinical development underway in various cancers, including soft tissue sarcoma.

Orchard Therapeutics (ADA-SCID, MPS III A)

The company signed a new collaboration with Orchard Therapeutics focusing on the development of transformative gene therapies for patients with serious life threatening orphan diseases. The company would initially develop and supply lentiviral vectors for two of Orchard Therapeutics’ stem cell based treatments, targeting primary immune deficiency and metabolic disorders.

Discovery and Pre-Clinical

The company would continue to invest in the identification and early-stage development of novel gene and cell therapy products based on the LentiVector gene delivery platform. This approach is designed to provide an ongoing pipeline of next generation product candidates while also building new intellectual property to maintain the company’s leadership position in the gene and cell therapy field.

The company is investing in various product concepts, including the new NK cell research and development collaboration with Green Cross LabCell. Where appropriate, the company would also consider in-licensing suitable targets and technologies.

Segments

Partnering: This segment includes the bioprocessing and process development activities for third parties.

R&D: This segment includes the costs of the company’s proprietary R&D activities in product and technology developments.

Significant Events

In November 2020, Oxford Biomedica plc announced a discovery collaboration with PhoreMost Limited to develop CAR-T cell therapies.

History

Oxford BioMedica plc was founded in 1995.