I Want To Thin Your Blood: Vampire Bat-Based Stroke Drug Trial Resumes

Potential safety signal in Forest/PAION’s desmoteplase study no longer spooks data monitoring committee.

Forest and PAION expect to have midline results from their Phase IIb/III desmoteplase stroke study by mid-2007, after getting the green light to resume the trial, the companies announced Oct. 27.

On Oct. 25, the firms said they were temporarily halting patient recruitment for the DIAS-2 (Desmoteplase in Acute Ischemic Stroke) study until an independent data monitoring committee could evaluate a "potential safety signal" (¹ (Also see "Forest’s Desmoteplase Stroke Study Halted Due To Safety Signal" - Pink Sheet, 25 Oct, 2006.)).

"It didn't seem to be such a big issue because after they evaluated all the data available, [the DMC] gave a flat go-ahead for the trial," Germany based-PAION told "The Pink Sheet" DAILY.

PAION suggested that quickly providing the DMC with the requested information helped expedite the evaluation process.

"They were provided with the data within one day, so they had the chance to look at that pretty quick," PAION told "The Pink Sheet" DAILY. "And after evaluating the data, they came to the conclusion that the trial can be resumed without any change in the protocol."

If the trial proceeds according to plan, PAION said desmoteplase could reach the market by 2009. Forest had previously said the firms could be in a position to file for approval in early 2009 (² (Also see "Desmoteplase Approval For Stroke Expected In 2009, Forest Says" - Pink Sheet, 16 Mar, 2005.)).

However, PAION told "The Pink Sheet" DAILY that a BLA submission could take place by the end of 2007 or in early 2008 "if the data yields compelling results."

"We now expect that the last patients can be recruited by the end of the year," PAION said. "And then [there will be] a 90-day follow-up period. If the last patient would be recruited on [Dec. 31] then the follow-up period would last until the end of March, and then you'd need another three months for data evaluation and to do some statistics."

After midline results are in, PAION said the firms "will look at the data and if they are compelling data, we would...discuss possible filings with the authorities."

While Forest is PAION's marketing and development partner for desmoteplase in North America, Denmark-based Lundbeck is PAION's partner outside Canada and the U.S. The firms have not released information regarding their European filing strategy.

"That is something we will update the financial community [about] later on," PAION said. "This is partly due to new regulations. Like fast-track in the U.S., they are establishing something similar in Europe. We are discussing with our partners whether we could and should take advantage of that."

PAION noted that desmoteplase, a genetically engineered version of a clot-dissolving protein found in the saliva of the vampire bat Desmodus rotundus, has fast-track status at FDA.

- Daniel Fowler ([email protected])