Ark Holds Positive End of Phase II Meeting with FDA on Trinam(R) Gene Therapy
Phase II data accepted and FDA offers Special Protocol Assessment for single
pivotal Phase III study
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(R), 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(R).
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(R) 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(R) 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(R) 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(R) programme, Ark also announces today that, after
consultation with the FDA, it intends to undertake a small pre-clinical study on
Trinam(R), 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(R)'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(R) and believe that Trinam(R) 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.' |
