Oxford Biomedica Regulatory News (OXB)

Oxford BioMedica notes that Axovant doses first patient in clinical study of AXO-Lenti-PD, a novel gene therapy for patients with Parkinson's disease

Oxford, UK - 25 October 2018: Oxford BioMedica plc (LSE:OXB) ("Oxford BioMedica" or "the Group"), a leading gene and cell therapy group, notes that its partner, Axovant Sciences (NASDAQ:AXON), a company developing innovative gene therapies for neurologic and neuromuscular diseases, has announced the successful dosing of the first patient in a clinical study of AXO-Lenti-PD, also known as OXB-102, an investigational gene therapy for patients with Parkinson's disease. AXO-Lenti-PD is a novel gene therapy that enables the expression of a set of three critical enzymes required for end-to-end dopamine synthesis in the brain. It was orginally developed by Oxford BioMedica and subsequently outlicensed to Axovant in June 2018.

AXO-Lenti-PD was administered to the first patient on the study at the Clinical Research Facility affiliated with the National Institute for Health Research (NIHR) and University College London Hospitals (UCLH). The patient experienced no complications related to the surgery or to administration of the vector and was discharged home as planned.

The ongoing clinical study of AXO-Lenti-PD will evaluate safety and tolerability, as well as collect efficacy data including standard measures of motor function in patients with Parkinson's disease. Initial data from the first cohort of patients in the clinical trial is expected in the first half of 2019. This study will build upon the long-term safety and efficacy results reported in the phase I/II trial of ProSavin®, which showed up to six-year durability of reductions in UPDRS Part III "OFF" scores in data shared last week at the Annual Congress of the European Society of Gene and Cell Therapy and recently published in Human Gene Therapy, Clinical Development.

Under the terms of the agreement with Axovant, Oxford BioMedica received a $30 million upfront payment (approximately £22 million) including $5 million as pre-payment for manufacturing activities related to OXB-102, now renamed AXO-Lenti-PD. Oxford BioMedica is also eligible to receive $55 million upon the achievement of specified development milestones and $757.5 million upon the achievement of specified regulatory and sales milestones, with 7% to 10% tiered royalties on net sales of AXO-Lenti-PD.

-Ends-

Notes to 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, Bioverativ, Sanofi, Axovant, Orchard Therapeutics, Boehringer Ingelheim/UK Cystic Fibrosis Gene Therapy Consortium/Imperial Innovations, 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 AXO-Lenti-PD

AXO-Lenti-PD, also known as OXB-102, is an investigational gene therapy for Parkinson's disease that delivers three genes in vivo via a lentiviral vector to encode a critical set of enzymes required for dopamine synthesis in the brain and is designed to provide patient benefit for many years following a single administration. AXO-Lenti-PD is a next-generation gene therapy designed to further increase endogenous dopamine production over the first-generation product, Prosavin, by modifying the payload configuration. Preclinical studies directly comparing AXO-Lenti-PD to ProSavin demonstrate increased AADC activity and dopamine productivity of the new vector configuration. Oxford BioMedica has successfully completed a phase I/II study for ProSavin®, which met its primary endpoint. The results, which were published in The Lancet in 2014, demonstrate favorable safety and tolerability and a statistically significant improvement from baseline of motor function as measured by the UPDRS Part III score at 6 and 12 months. This improvement has been observed to be sustained in patients for up to six years despite the progressively degenerative nature of Parkinson's disease. Initial data from the ongoing AXO-Lenti-PD clinical program is expected in the first half of 2019.

About Axovant Sciences

Axovant is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating diseases such as Parkinson's disease, oculopharyngeal muscular dystrophy, Duchenne muscular dystrophy, ALS, frontotemporal dementia, and other indications. For more information, visit www.axovant.com