OXFORD BIOMEDICA PUBLISHES PRECLINICAL EFFICACY RESULTS WITH ITS PRODUCT CANDIDATE SMN1-G FOR SPINAL MUSCULAR ATROPHY

Oxford, UK – 16 December 2004: Oxford BioMedica (LSE: OXB), a leading gene therapy company, is pleased to announce the publication of promising preclinical results with its product candidate, SMN1-G, for the treatment of spinal muscular atrophy (SMA), in the Journal of Clinical Investigation. SMA is a fatal genetic disease that affects the spinal cord and brain stem. To date there are no effective drug treatments for SMA. The preclinical results published in the Journal of Clinical Investigation (Volume: 114, No: 12, pp: 1726-1731), suggest that SMN1-G may have potential in the treatment of this genetic disease. 

The preclinical studies were supported by FightSMA, a US charitable organisation. In these studies, mice with a defective SMN gene were given intramuscular injections with either SMN1-G or a control. The mice treated with SMN1-G showed a statistically significant improvement in survival together with improved motor neuron survival. 

SMA is one of the most common genetic diseases leading to death in childhood. The disease, characterised by degeneration of motor neurons in the spinal cord and brain stem leading to muscle weakness, is caused by mutations or deletion of the SMN1 gene. SMN1-G is Oxford BioMedica’s gene-based therapeutic which utilises the Company’s LentiVector gene delivery system carrying a corrected SMN1 gene. The Company anticipates progressing SMN1-G into clinical development during 2006. 

Martha Slay, President of FightSMA, said, “For the first time ever, researchers have replaced a missing gene in a mouse model of SMA, a disease that kills more infants than any other genetic disorder.” 

Oxford BioMedica’s CEO, Professor Alan Kingsman, said, “We are very encouraged by our preclinical data with SMN1-G and its potential to reverse the effects of this fatal disease. We believe that there is a significant commercial opportunity in the development of a safe and effective treatment for this unmet medical need.” 

-Ends- 

For further information, please contact: 

Oxford BioMedica plc:

Professor Alan Kingsman, Chief Executive

Tel: +44 (0)1865 783 000

 City/Financial Enquiries:

Lisa Baderoon/ Mark Court/ Mary-Jane Johnson Buchanan Communications

Tel: +44 (0)20 7466 5000

 Scientific/Trade Press Enquiries:

Katja Stout/ Ashley Lilly

Northbank Communications

Tel: +44 (0)20 7886 8150

 FightSMA:

Martha Slay, President

Tel: +1 804 515 0080

A copy of the publication and this release can be found by visiting the Oxford BioMedica website: www.oxfordbiomedica.co.uk/news2004.htm  

Notes to editors 

1. Oxford BioMedica

Oxford BioMedica (LSE: OXB) is a biopharmaceutical company specialising in the development of novel gene-based therapeutics with a focus on the areas of oncology and neurotherapy. The Company was established in 1995 as a spin out from Oxford University, and is listed on the London Stock Exchange.  

In addition to its technical expertise in gene delivery, Oxford BioMedica has in-house clinical, regulatory and manufacturing know-how. The development pipeline includes two novel anti-cancer products in clinical trials; two neurotherapy products in advanced preclinical development for Parkinson’s disease and retinopathy; and three further preclinical neurotherapy products. The Company is underpinned by an extensive preclinical and research portfolio and over 80 patent families, which represents one of the broadest patent estates in the field. 

The Company has a staff of approx. 65 split between its main facilities in Oxford and its wholly owned subsidiary, BioMedica Inc, in San Diego, California. Oxford BioMedica has corporate collaborations with Wyeth, Intervet, Merck & Co, Amersham, Viragen and Kiadis.  

Further information is available at http://www.oxfordbiomedica.co.uk/ 

 2. SMN1-G for spinal muscular atrophy

Spinal muscular atrophy (SMA) refers to a group of diseases, which affect the motor neurons of the spinal cord and brain stem. These critically important cells are responsible for supplying electrical and chemical messages to muscle cells. Without the proper input from the motor neurons, muscle cells cannot function properly. The muscle cells will, therefore, become much smaller (atrophy) and will produce symptoms of muscle weakness. It is caused by mutations or deletion of the SMN-1 gene, leading to depletion in SMN protein levels.  

SMA is the most common genetically determined cause of infant death. It is estimated that SMA occurs in between one-in-6,000 and one-in-20,000 births. Based on the clinical severity and age at onset, the childhood SMAs have been divided into three types. Type I SMA is the most severe, with onset occurring by 6 months of age and death by 2 years. There are currently no approved treatments for this disorder. Oxford BioMedica estimates the market opportunity for an effective treatment of SMA to be approximately $50 million. 

Oxford BioMedica is developing a novel gene therapy candidate, SMN1-G, for the treatment of SMA. The product is designed to replace the defective SMN-1 gene using the Company’s proprietary LentiVector gene delivery system carrying the SMN-1 gene. The development of SMN1-G is being supported by the US charitable organisation, FightSMA. The Company expects to enter clinical development with SMN1-G in 2006. 

The article from the Journal of Clinical Investigation (Volume: 114, No: 12, pp: 1726-1731), describing the preclinical efficacy data with SMN1-G in a mouse model of the disease, is available at http://www.oxfordbiomedica.co.uk/news2004.htm 

3. FightSMA

FightSMA is a non-profit corporation, founded in Richmond, VA, in 1991 as "Andrew's Buddies", to accelerate research for a treatment or a cure for spinal muscular atrophy, the number-one inherited cause of death of children under two. 

Further information is available at http://www.fightsma.com/