BioDiem to trial blindness treatment

Melbourne-based biopharma BioDiem (ASX:BDM) has received regulatory approval to commence a phase I/II clinical trial for its peptide BDM-E in patients with diabetic macula oedema, a leading cause of blindness.

BDM-E is a potential treatment for the two major causes of blindness -- Diabetic Retinopathy (DR) and Age-related Macular Degeneration (AMD). It is a small synthetic peptide which in vitro studies show stimulates growth of new retinal pigmented epithelial cells and inhibits the growth of choroidal endothelial cells associated with unwanted neovascularisation.

"The phase I/II clinical trial is essentially a proof of concept trial, designed to show efficacy as well as safety of BDM-E administered by subcutaneous injections, in comparison with placebo, in patients with clinically relevant diabetic macular oedema." said BioDiem CEO Tom Williams. "It will test the ability of BDM-E to reduce the swelling that's typical of diabetic retinopathy in the back of the eye, which often leads to significant vision loss."

The trial, which includes 192 patients, will be conducted at eight centres in St Petersburg and Moscow in Russia. The trial protocol has designed by Assoc Prof Mark Gillies, at Sydney University, who also conducted the in vitro studies.

"He advised us to conduct the phase I/II trials in Russia because it would take a year to recruit that number of patients in Australia, but is likely to take 3-4 months in St Petersburg and Moscow," Williams said.

"We take an international approach and look all over the world to find the best people who are able to do the job in a cost effective and efficient manner. In this case, where clinical trial material must be made to GMP standards, the raw material is being synthesised in California and the final clinical trial ampoules made in Germany."

Gillies said there was an urgent need for new safe and effective treatments for diabetic retinopathy and other retinal diseases, such as age-related macular degeneration.

"Nearly all patients who have type 1 diabetes for 20 years or more will have evidence of diabetic retinopathy," he said. "Up to 21 per cent of people with type 2 diabetes have retinopathy when they are first diagnosed with diabetes, and most will eventually develop some degree of retinopathy. Diabetes is responsible for 8 per cent of legal blindness, making it the leading cause of new cases of blindness in adults 20-74 years of age. In the United States, each year, 12,000 to 24,000 people lose their sight because of diabetes."

Diabetic retinopathy is a term used for the abnormalities of the small blood vessels of the retina caused by diabetes, such as weakening of blood vessel walls or leakage from blood vessels.

The trial will be managed by a Swiss contract research organisation specialising in running clinical eye studies to best international standards. The first patients are expected to be treated in January 2006 and the results should be available by late 2006. The study end-points will be changes in macular oedema measured by ocular coherence tomography and secondly changes in visual acuity measured over a three-month observation period. The trial will be a placebo controlled, randomised double-blinded Phase I/II trial designed to establish proof of concept. The study will be conducted in compliance with ICH Good Clinical Practice (GCP) guidelines as required by the world's leading regulatory authorities, including the FDA, EMEA and TGA.

"We are likely to do several more phase II studies in the future before moving onto phase III trials," Williams said. "Currently our business model is to out-license the product to a large pharmaceutical company before the phase III trial."

Only two drug treatments are currently available for AMD, but these are effective in only a small percentage of patients. Nonetheless, sales of up to US$1 billion are projected by 2008. There is currently no approved drug treatment for DR. Intra-ocular steroids have been used with some success, but their use is limited by major side effects such as cataract and glaucoma. A safe and effective drug treatment for DR would be expected to have major commercial potential, especially if it did not have to be given by intraocular injection.

"Forty per cent of eye disease occurs in the back of the eye. At the moment only five per cent of treatments address the back of the eye," said Williams.