Amicus Therapeutics ( FOLD) presented some phase II data on its Fabry disease drug Amigal on Wednesday. Amigal appears to be an active drug that can reduce the toxic buildup of a protein called GL-3 in the kidneys of patients. It's the inability to remove GL-3 from cells in the body (because of a genetic malfunction) that causes the severe physical problems in Fabry patients. However, investors' reaction to what seemed like positive news was anything but. Amicus shares were shellacked Thursday, falling $5.59, or 37%, to $9.54. Why the incongruence between the Amigal data and the stock price? The answer is in the details of the Amigal data. Amicus got its best results by measuring reductions in GL-3 levels in the urine of Fabry patients. The efficacy was less robust, however, when GL-3 levels were measured via biopsies of kidney cells.
Introgen Therapeutics ( INGN) has missed yet another promised deadline for its cancer gene therapy Advexin. All year, the company has sworn up and down that it would release results from a phase III study of Advexin in head and neck cancer patients and file Advexin for approval with both the FDA and the European Medicines Agency before the end of 2007. On Thursday, Introgen reneged on that promise, saying it needs more time to collect and analyze tissue samples from Advexin patients in the phase III study. Therefore, there will be no Advexin data or regulatory filings until the first half of 2008. The news sent Introgen shares sliding $1.30, or 28%, to $3.22. That's just 20 cents above the stock's 52-week low. On Friday, the stock was off almost another 4% to $3.10. Introgen is really blazing new trails in biotech bamboozlement, going where no company has gone before. Consider me impressed. Let's recap a brief history of Introgen's track record when it comes to Advexin: An FDA filing in 2004? Nope. A filing in 2005? Nada. 2006? Didn't happen. Now we add 2007 to the list of Introgen ignominy. What makes this year even more hilarious is that management was promising publicly, as late as the middle of November, that it was going to meet the deadline this time. And earlier this week, Introgen's board awarded CEO David Nance with another long-term employment contract.
Altus Therapeutics ( ALTU) announced Wednesday that it was dumped by Genentech ( DNA), its year-long partner in developing a long-acting human growth hormone. Not good news, clearly. When Genentech decided to partner with Altus last December, investors rightly saw the deal as a validation of Altus' drug delivery platform -- a proprietary protein crystallization and formulation technology that is designed to enhance a drug's efficacy and/or improve safety and dosing convenience. But a year went by with zero progress and no news from the Altus-Genentech partnership to develop ALTU-238, a once-weekly injection of human growth hormone. That is, until Wednesday, when Altus announced that Genentech had hit the road. This has to raise serious questions about Altus' technology platform. At this point, I'd place Altus in the same category as Nastech Pharmaceuticals ( NSTK) -- companies with unique and potentially valuable drug-delivery technologies that can't seem to execute and therefore can't be trusted until they prove themselves. Altus now says it will develop ALTU-238 on its own. The company is also in the midst of a phase III study of ALTU-135, a pancreatic enzyme replacement therapy for patients with cystic fibrosis and other diseases. Here too, unfortunately, I'd have significant concerns about ALTU-135's chances for success. And I used to like Altus a lot. I haven't forgotten my Jan. 19 column praising the company. The stock was just under $19 when I wrote bullishly about Altus' technology and its pipeline. On Thursday, it sank $4.38, or 44%, to $5.50. Clearly, this was a dismal call on my part.