8 January 2009 - Ark Therapeutics Group plc (“Ark” or the “Company”) today announces that it has successfully completed the work requested by the US Food and Drug Administration (FDA) to qualify a potency test for Trinam®. As a result, the Company has now been notified by the FDA that it can commence patient recruitment into its Trinam® Phase III trial. In June 2008, Ark reported that its application for Special Protocol Assessment (SPA) for Trinam® had been successful and that, in parallel, the updated Investigational New Drug (IND) application for the trial had also been reviewed and approved by the FDA. The IND was subject to a requirement for Ark to qualify a product release test relating to potency prior to treating patients. That condition has now been met.
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 biodegradable local delivery device (EG001). Trinam® has already been granted Orphan Drug Status in the USA and Europe and Ark now also intends to apply to the FDA for Fast Track Designation and to submit a rolling Biologic Licence Application (BLA) for sale and marketing approval in the US in due course. 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.
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 end point and overall patency and a number of other important pre-defined clinical endpoints will also be measured. Safety will be assessed by an independent Data and Safety Monitoring Board (DSMB) against a pre-specified set of stopping rules defined during the SPA. The DSMB will also undertake a ‘sample sizing’ analysis after 150 patients have been recruited 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.
Dr David Eckland, R & D Director of Ark, commented: “Qualifying and validating batch release assays in advanced biologicals is pioneering work and is part of the evolving process of developing and improving the quality controls for these breakthrough drugs as they move nearer to market. The work requires considerable scientific capabilities and again our Finnish unit has demonstrated its capabilities in developing this potency assay.”
Nigel Parker, CEO of Ark, added: “Following finalisation of the test work and acceptance of the assay by the FDA, we are delighted now to be commencing patient recruitment in the Phase III trial for Trinam®. Maintenance of graft access for kidney dialysis patients is vital for their survival , making this an area of high clinical need for which we are hopeful that this pioneering gene-based medicine can provide a solution. With our other lead product, Cerepro®, now filed for regulatory approval in Europe, Ark’s portfolio continues to make strong progress towards the market.”
