BioWorld International Correspondent

LONDON - At first glance the announcement that the European regulator EMEA was not giving approval to Ark Therapeutics Group plc's Cerepro gene therapy treatment for glioma looked like bad news.

But according to Nigel Parker, CEO, all the company's development and manufacturing people sported broad grins when they heard. "They are absolutely delighted. We always said we may need the next study in terms of efficacy, but the announcement from European Medicines Agency (EMEA) means that all the issues that have dogged gene therapy have gone away," Parker told BioWorld International.

The Cerepro filing has set the route for approval of gene therapies in Europe, and the product appears to have cleared all the technical hurdles, including chemistry and manufacturing controls, and preclinical and environmental requirements, Parker claimed. "The only outstanding objection is the weight of efficacy data. It looks to me as if this is the biggest advance in gene therapy for many years."

Cerepro, which has orphan drug status, was submitted on the basis of Phase II data.

The company said it needs to look at the detailed feedback from EMEA before deciding how to proceed. The extra clinical evidence required possibly could be supplied through a further analysis of the Phase II data, or it may need data from the Phase III trial.

In April, the London-based company announced that it completed recruitment in a 250-patient Phase III trial. "While it would have been nice to get earlier approval, we have always worked on the basis that we would need Phase III data," Parker said. "What we didn't want were any technical issues that would have meant the Phase III was based on the wrong material."

Ark had cash of £48.4 million (US$96.5 million) at the end of 2006, which it says is more than enough to get beyond the point at which Cerepro would be approved under the most conservative route. Parker said the company has carefully managed investors' expectations on this point, and although the shares dropped £0.11 to £1.18, they came back to close at £1.30 when the news was announced on April 27.

The EMEA decision has no impact on plans to conduct trials of Cerepro in the U.S. The company said its U.S. development plan always depended on finalizing the EMEA review and progressing the current Phase III trial before engaging with the FDA.

In the Phase II trials that formed the basis of the EMEA submission, patients receiving Cerepro survived for 15.5 months, compared to eight months for patients receiving standard care.

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