U.S. regulators put two clinical trials of a hemophilia drug Ipsen SA (IPN) is developing with Inspiration Biopharmaceuticals Inc. on hold because of “a potential safety concern.” Ipsen fell the most in two years.
Inspiration noticed that patients treated with the experimental product, called IB1001, were more likely to test positive for antibodies to Chinese hamster ovary protein, the product’s host cell protein, Ipsen said in a statement today. Inspiration alerted the Food and Drug Administration and is investigating, according to the statement.
“While this finding may be a potential safety concern, no evidence suggests a change in the current overall clinical benefit and risk profile of IB1001,” Ipsen, based in Boulogne- Billancourt near Paris, said in the statement.
The suspension of the trials may mean a delay in winning approval for the drug, and could even result in the treatment being dropped, said Tara Raveendran, an analyst at Jefferies International Ltd. Ipsen needs new products after the failure in 2010 of the experimental diabetes drug taspoglutide, which contributed to an overhaul that included the appointment of Chief Executive Officer Marc de Garidel late that year.
Ipsen dropped as much as 12 percent to 17.51 euros, the biggest intraday decline since June 18, 2010, and was trading down 11 percent at 1:46 p.m. in Paris, valuing the company at 1.5 billion euros ($1.85 billion).
The IB1001 setback is “definitely bad in terms of sentiment, and that explains the market reaction,” Mathieu Chabert, an analyst at Bryan Garnier & Co. in Paris, said in a telephone interview today. “It’s yet another disappointment coming from Ipsen, and a reminder of the dark hours of taspoglutide’s failure, even if the two stories are clearly not the same.”
CEO De Garidel has dropped some projects, focused research on disease-causing substances known as toxins and peptides, and has sought a partnership for Ipsen’s French primary-care operations. Ipsen is also expanding in hemophilia through its work with Cambridge, Massachusetts-based Inspiration.
The experimental medicine, like other recombinant drugs, uses Chinese hamster ovary cells to produce a protein known as factor IX that’s key to coagulation. The people treated with IB1001 showed a higher-than-expected level of antibodies against the hamster cells, suggesting the patients’ immune systems had started mounting a response to the treatment.
Inspiration and Ipsen are testing whether IB1001 can prevent bleeding in two late-stage trials, one in adults with hemophilia B and the other in children with the illness who have received prior treatment. Inspiration has control of the IB1001 late-stage trials, Didier Veron, an Ipsen spokesman, said in a telephone interview today.
The U.S. company received the letter from the FDA on July 6 and Ipsen was notified the following day, he said.
“The FDA has asked Inspiration to conduct a root cause analysis to understand the higher-than-expected level of anti- CHO antibodies,” Veron said. Results of that study will be known sometime from September through December, he said.
“We cannot speculate at this time on whether there will be any impact on the marketing applications for IB1001,” Veron said. “This doesn’t in any way change Ipsen’s strategy” in hemophilia.
Ipsen filed for approval of IB1001 in the European Union in October and in the U.S. in April, Veron said. The company has been looking at introducing the treatment in Europe in the first half of 2013 and about six months later in the U.S., he said.
“If an additional filtration step in manufacturing is found to be required, this could be a significant setback to time lines,” Jefferies analyst Raveendran wrote in a note to clients today.
Ipsen owns a stake in closely held Inspiration. The companies are developing drugs to treat hemophilia, an inherited blood disorder caused by low levels or an absence of a protein called clotting factor essential for coagulation. Hemophilia B is caused by a shortage of factor IX, while Hemophilia A is caused by a lack of factor VIII, according to Inspiration.
A total of 86 people with hemophilia B have received the drug in trials, with no adverse events related to the development of antibodies to the CHO protein, Ipsen said.
The French company’s stock, including reinvested dividends, has dropped 25 percent this year, compared with a 19 percent gain for the Bloomberg Europe Pharmaceuticals Index.
Today’s announcement “will add pressure on the management to deliver a deal for the French primary-care business, which could be a positive catalyst,” said Garnier analyst Chabert, who is forecasting 98 million euros in annual peak sales for IB1001 by 2022. He has a buy recommendation on Ipsen shares.
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