This Day On The Street
Continue to site
This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here

A Legit Reason To Give Amicus' Fabry Drug Another Chance

CRANBURY, NJ ( TheStreet) -- Amicus Therapeutics (FOLD) presented an alternative theory Friday to explain the failure of its Fabry disease drug Amigal in a late-stage clinical trial. Not surprisingly, investors reacted with skepticism and Amicus shares sank 25% to $2.89.

It's easy to dismiss Amicus' analysis for what it looks like on the surface: Just another example of a drug company resorting to retrospective data mining in order to stave off the painful reality of a negative clinical trial. But this time, what Amicus is saying about Amigal might be right: The drug works against Fabry disease. The challenge -- more difficult than the company anticipated -- is designing a clinical trial to prove it.

Here's what happened Friday: Amicus dissected the Amigal phase III study and found slightly more than half of the Fabry patients had relatively mild disease at the start of the trial, way more than the company intended to enroll. Mild Fabry disease is harder to measure so in these patients, the study was unable to detect a significant difference in kidney response between Amigal and placebo.

However, in Fabry patients with severe disease at baseline and therefore more measurable, the difference in kidney response detected between Amigal and placebo was greater. Had the study enrolled more patients with severe disease, as originally planned, the difference in response between Amigal and placebo might have been statistically significant, the company said.

Now, to be clear, Amicus conducted this six-month analysis on a post-hoc basis, meaning separating patients by disease severity was not part of the original statistical analysis plan. However, Amicus is speaking to FDA about amending the study design before the next scheduled look at the Amigal data at 12 months.

Amicus expects 12-month data to be ready late in the second quarter. Investors have largely written off Amigal today -- and it's hard to blame them -- but skepticism about the drug could turn into optimism if the 12-month data are strong. If that happens, Amicus shares could rebound and more.

To understand why Amigal chances shouldn't be discarded quite yet, it's important to go back over some background on Fabry disease and review the design and initial results from the phase III study.
1 of 3

Check Out Our Best Services for Investors

Action Alerts PLUS

Portfolio Manager Jim Cramer and Director of Research Jack Mohr reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
Quant Ratings

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
Stocks Under $10

David Peltier uncovers low dollar stocks with serious upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
14-Days Free
Only $9.95
14-Days Free
Dividend Stock Advisor

David Peltier identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Updates with exact steps to take - BUY, HOLD, SELL
Trifecta Stocks

Every recommendation goes through 3 layers of intense scrutiny—quantitative, fundamental and technical analysis—to maximize profit potential and minimize risk.

Product Features:
  • Model Portfolio
  • Intra Day Trade alerts
  • Access to Quant Ratings
Real Money

More than 30 investing pros with skin in the game give you actionable insight and investment ideas.

Product Features:
  • Access to Jim Cramer's daily blog
  • Intraday commentary and news
  • Real-time trading forums
Only $49.95
14-Days Free
14-Days Free
FOLD $7.47 0.00%
GSK $42.91 0.00%
AAPL $93.74 0.00%
FB $117.58 0.00%
GOOG $693.01 0.00%


Chart of I:DJI
DOW 17,773.64 -57.12 -0.32%
S&P 500 2,065.30 -10.51 -0.51%
NASDAQ 4,775.3580 -29.9330 -0.62%

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs