Ark Therapeutics Group plc (’Ark’ or the ‘Company’) announces that the first patient has been enrolled into the US Phase III study for Trinam®. Trinam® is Ark’s novel gene-based medicine to prevent blood vessels blocking in kidney dialysis patients who have undergone vascular access graft surgery. The product is an adenovirus-mediated VEGF D gene delivered with a novel biodegradeable local delivery device (EG001).

The Phase III study is a US multi-centre, randomised, controlled trial, in which the efficacy and safety of Trinam® will be investigated in patients with end-stage renal disease (ESRD) requiring vascular access for haemodialysis. Patients with ESRD will be randomised to receive either Trinam® in addition to standard care or standard care alone at the time of surgical placement of a synthetic PTFE graft for vascular access. Primary Unassisted Patency (time to any first intervention) will be the primary regulatory endpoint. Overall patency and a number of other important pre-defined clinical endpoints will also be measured.

The safety of the trial will be assessed by an independent Data and Safety Monitoring Board (DSMB) against a pre-specified set of stopping rules defined during the Special Protocol Assessment (SPA). The DSMB will also undertake a blinded ’sizing’ analysis after 150 patients have been enrolled to determine the final trial size. This type of adaptive design assists groundbreaking drugs to ensure robust efficacy data are available to satisfy regulatory requirements as approval standards evolve.

Results from a Phase II open-label, non-randomised, standard-care controlled trial of Trinam®, reported in March 2007, indicated that the access grafts of patients given Trinam® remained functional for dialysis, on average, up to three times longer than in untreated controls. Trinam® was well tolerated with no quantifiable systemic distribution of the product found and no serious side effects were exhibited other than those consistent with the nature of the operation and underlying condition.

Trinam® was awarded Fast Track Status by the FDA earlier this month and has been granted Orphan Drug Status in both the US and Europe. US regulatory review for the product comes under the responsibility of the Centre for Biologics Evaluation and Research (CBER), the specialist biologics division of the FDA.

Dr David Eckland, Research and Development Director of Ark, commented: “We are very pleased to commence enrolment into the Phase III study for Trinam® which follows the Fast Track Status gained from the FDA earlier this month. There is a significant unmet therapeutic need in this indication and today’s news brings us a step closer to gaining approval for a product which we believe will transform the prognosis for many patients suffering from kidney failure.”

Dr Nigel Parker, CEO of Ark, added: “First patient enrolment is a significant milestone for this very important gene-based medicine and reflects the good progress we continue to make at Ark. Our whole portfolio is growing in strength and we look forward to announcing details of Trinam®’s continued progress in due course.”

Trinam®

Trinam® is a combination of a vascular endothelial growth factor gene in an adenoviral vector (Ad-VEGF-D) and Ark’s biodegradable local delivery collagen collar device (EG001). At the end of the access graft surgery procedure, the collar is fitted around the outside of the vein/graft join. The Ad-VEGF-D solution, which reduces the likelihood of blood clots and intimal hyperplasia, is then injected into the space between the wall of the collar and the blood vessel. This unique method of administration of the gene localises its delivery to the target tissue site, maximising efficacy, avoiding systemic distribution and thus minimising the potential for side effects.

Source
Ark Therapeutics Group plc