BioWorld International Correspondent
LONDON - Ark Therapeutics plc cleared another hurdle in its bid to become the first to launch a gene therapy product in a regulated market, when the independent data and safety committee unanimously recommended that the Phase III trial of Cerepro continue to completion without modification.
The review, based on the first 130 glioma patients to enter the trial, shows that Ark's GMP-approved manufacturing facility is delivering a reliable and predictable product, said CEO Nigel Parker.
"This is a really, really important announcement for us," he told BioWorld International. "We have cleared the last of a series of obstacles that analysts said could not be overcome. Gene therapy is now poised to take off - it will be like the opening of the market for monoclonal antibodies."
The Phase III trial has a target of 250 patients and now has recruited 160. Half are receiving standard care and half standard care plus Cerepro. The product uses an adenovirus to deliver the gene for the enzyme thymidine kinase. That converts a prodrug into a form that is toxic to tumor cells but has no effect on normal brain cells.
Cerepro became the first gene therapy product to move into a full regulatory review (outside China) in 2005 when the European regulator EMEA accepted it for the "exceptional approval" route on the basis of two previous Phase II trials. EMEA's review is due to complete in March 2007.
"Obviously EMEA would have been interested if there were any safety implications from the [safety committee] review, but there aren't," Parker said. In terms of outcomes, the Phase III is still blinded and is yet to add to the submitted data from the Phase II trials in which Cerepro increased life expectancy by an average of 7.5 months over the average eight month survival for patients receiving standard care.
Armed with the conclusions of the data and safety committee, London-based Ark now will meet with the FDA to discuss its requirements for registering Cerepro in the U.S. "The FDA is familiar with where we are, as Cerepro has U.S orphan drug status," said Parker. "We now want to establish what we need to do to get approval."