BioWorld International Correspondent
LONDON - Shares in Oxford GlycoSciences plc fell by 92.5 pence to £3 Monday after the company said the FDA had ruled its lead product, Vevesca, not approvable. At that price the market capitalization of OGS is less than its £170 million (US$255 million) cash pile.
The FDA said the company had not provided sufficient support for the safety and efficacy of Vevesca (OGT 918) in the treatment of Type I Gaucher's disease. Stephen Parker, the company's chief financial officer, told BioWorld International, "Clearly you have to be disappointed with this sort of outcome. We believe in the drug, or we wouldn't have filed for an NDA."
The Abingdon-based company is requesting a meeting with FDA representatives to review the letter and find the best way forward. "We will have an active dialogue with the FDA, to understand what their concerns are," Parker said. "The letter does talk in terms of further trials, but what the nature of those trials might be, we don't know until we have had the meeting."
Until then it is unclear what the implications are in terms of the timing of a launch of Vevesca. "Clearly there will be a delay. But again, I'm afraid, it comes down to the FDA how long that is," he said.
OGS has also filed Vevesca with the European regulatory authority EMEA, and a decision is expected during the third quarter. The drug has orphan status in both the U.S. and Europe. Parker said he does not expect the FDA's ruling to have implications for the drug in Europe. "The [FDA and EMEA] are separate and it is not unheard of for EMEA to approve drugs that the FDA does not."
In March, OGS said it was extending OGT 918 trials into potential new indications, in Nieman-Pick Type C disease, neuropathic Gaucher's disease and late-onset GM2 gangliosidosis. "Everything we have described so far continues," pending further discussion with the FDA, Parker said.
At the start of 2002 OGS's stock stood at £6.75, valuing the company at £372.5 million. But before Monday's fall, two earlier announcements relating to Vevesca had also taken a toll on the share price.
In March, OGS published data indicating that 11 percent of patients in Vevesca trials had central nervous system side effects. The company's intention was to highlight that figure against the occurrence of nervous system symptoms in 32 percent of Gaucher's sufferers not taking Vevesca, but the shares fell by 42.5 pence to £3.87 when the announcement was made on March 21. The shares recovered to reach £4.15 in early April but then lost more than 20 percent of their value, falling to £3.27 when OGS halted Vevesca treatment in an extended-use trial in Israel. The move was made as a precaution after a 66-year-old patient developed cognitive dysfunction of an unknown cause.
Whether the FDA's non-approvable letter signals the end of Vevesca, or merely a delay, it leaves OGS's CEO-in-waiting, David Ebsworth, with an even greater challenge when he takes over the reigns in July. OGS owns a wealth of targets, having identified more than 4,000 disease-related proteins with its proteomics platform technology. It has a collaboration with NeuroGenesis Inc., of Cambridge, Mass., to find small molecules, and with Genmab A/S, of Copenhagen, Denmark, and BioInvent International, of Lund, Sweden, to discover and develop antibodies to those targets, but other than Vevesca, all of OGS's compounds are still at the preclinical stage.
Ebsworth, former head of the pharmaceutical business group of Bayer AG, told BioWorld International when he was appointed CEO of OGS last month that the key task was to close the gap in the pipeline between Vevesca and the preclinical products and that could involve licensing or acquisitions.