11 January 2007 - Ark Therapeutics Group plc (“Ark”) today announces that it has held a positive “end of Phase II” meeting with the US Food and Drug Administration (“FDA”) regarding Trinam®, its novel gene therapy to prevent blood vessels blocking in kidney dialysis patients who have undergone vascular access graft surgery.
Key points to emerge from the meeting were that the FDA has agreed that the data from the Phase II trial, reported by Ark in August 2006, are sufficient to allow progression to Phase III and a single Phase III trial will be acceptable for the basis of a marketing approval. Furthermore, the FDA has offered Special Protocol Assessment (SPA) for the single pivotal Phase III study for Trinam®. The SPA procedure allows Ark to work directly with the FDA to ensure the design of the trial, the definitive clinical objectives and data analyses are optimised to support regulatory approval.
The Phase III study is being planned as a multi-centre, randomised, controlled trial of up to 250 patients 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® 4x1010 viral particles in addition to standard care or standard care alone at the time of surgical placement of a synthetic PTFE graft for vascular access. The primary endpoint of the trial will be the time to graft failure.
Ark reported the preliminary results of the ongoing, open-label, standard-care controlled Phase II trial in August, with the new data from the trial showing that the access grafts of low dose patients remained functional for dialysis on average over five times longer (17.8 months) than control patients in the trial (3.3 months). At that time, in the high dose group, recruited after the low dose group, all patients with successful graft implants had open grafts with patency averaging 8 months. For the primary end point of safety, no quantifiable systemic distribution of Trinam® was found in either of the high or low dose groups and the product is well tolerated. No serious side effects were exhibited other than those consistent with the nature of the operation and condition.
As part of the overall Trinam® programme, Ark also announces today that, after consultation with the FDA, it intends to undertake a small pre-clinical study on Trinam®, investigating biodistribution in an “end-to-side” procedure for surgical placement of the graft. If the results of this trial are in line with expectations, it will allow the Phase III trial to include this procedure alongside the “end-to-end” placement procedure. Pending SPA agreement, the Phase III study is expected to commence around mid-2007 and to last for approximately 18 months.
Commenting on today’s announcement, Dr Nigel Parker, Chief Executive of Ark, said:
“The FDA’s positive response to the next stage of Trinam®’s development, particularly its offer of Special Protocol Assessment, confirms our belief in the future of this product. We have very encouraging Phase II data on the clinical effectiveness of Trinam® and believe that Trinam® may have a valuable role to play in the treatment of kidney failure patients where the problem of vascular access blocking is identified in the US Healthy People 2010 Framework as one of the key medical issues to be resolved. This outcome is in line with our budgeted plan and we look forward to giving a further update after receiving Special Protocol Assessment and to the commencement of the Phase III study.”
For further information:
Ark Therapeutics Group plc Tel: + 44 (0)20 7388 7722 Dr Nigel Parker, CEO Martyn Williams, CFO
Financial Dynamics Tel: +44 (0)20 7831 3113 David Yates Anna Keeble
