By John Carroll and Ryan McBride
|CEO John Crowley|
Shares of Amicus Therapeutics ($FOLD) plunged this morning after the biotech alerted investors that the FDA had blocked a possible route to a near-term approval for its experimental Fabry disease drug Amigal, forcing it to wait for a fresh batch of Phase III data that could pave the way to a new application in late 2014. And CEO John Crowley tells FierceBiotech that the company may look to new development pacts to help raise fresh funds.
Late last year the Cranbury, NJ-based biotech reported that its first late-stage trial for Amigal had failed, done in by a high placebo response after patients had been treated for 6 months. The biotech had hoped that the FDA would allow them to change the rules of the game, to let them review a follow-up 12-month response rate as a basis for an approval, with some added subgroup analysis to help illustrate the efficacy. The agency OK'd the design change but made it clear that the company still had a failure on its hands and avoided any commitment to using the 12-month data for an approval. Now Amicus must wait for more data from an ongoing Phase III study before it can move forward.
Amicus ended the first quarter with close to $85 million in cash, leaving it with a runway that extends into the second half of next year. But CEO John Crowley--who has a partnership on the Fabry program with GlaxoSmithKline ($GSK)--is looking for some positive data reports in the coming months to help bolster the company as he explores some new deals on other work in the pipeline to help provide added cash.
"One attractive opportunity is to partner the broad platform" for next-gen enzyme-replacement therapeutics or do a "Pompe-specific deal, while retaining residual rights," says Crowley.
In December investigators noted that after 6 months of treatment in Study 011, biopsies showed that 41%, or 13, of 32 patients on Amigal reached at least a 50% reduction in a lipid substrate called GL3 that accumulates in the kidneys of Fabry patients because of an enzyme deficiency. Twenty-eight percent of patients on placebo, or 9 of 32, hit the same mark. The difference fell short of statistical significance, falling squarely in the failure category. At the time, though, Cowen analyst Edward Nash wrote that "we believe that the positive trend will likely become more pronounced after the 6-month follow-up treatment period."
The company's second late-stage study, dubbed Study 012, compares Amigal to enzyme replacement therapy, which is the current treatment for patients with the inherited disease.
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