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Amicus Therapeutics, Inc. at BioCentury Publications Inc. Future Leaders in the Biotech Industry Conference - Final.

Article Excerpt
EDITOR: Good morning. I'm Elemer Piros, senior biotech analyst at Rodman and Renshaw. I would like to introduce the next company, Amicus.

Amicus is looking to start, in the first half, a Phase III trial with Amigal (migalastat) to treat Fabry disease. Shire is the partner for the compound. Amicus raised $75 million in an IPO in 2007. Presenting for the Company, Chief Operating Officer Matt Patterson, and there will be a breakout immediately following the presentation in room 309. Matt?

MATT PATTERSON, COO, AMICUS THERAPEUTICS, INC.: Thank you very much. Good morning, everyone. It's my pleasure to give you an overview of Amicus Therapeutics today.

I will start by referring you to the safe Harbor statement, as I will be making forward-looking statements today.

We believe 2009 will be a transformational year at Amicus, and we believe this for the reasons that are listed here on this slide. I will go through these today in the presentation.

But specifically, our lead program for Fabry disease will be entering Phase III. We have an extremely strong financial position at the Company. We have multiple clinical and regulatory milestones over the course of this year, a strategic partnership with Shire Human Genetic Therapies, and a technology platform that I will go through briefly that we believe offers extreme breadth and versatility for the treatment of a broad range of human genetic diseases.

We started this science in the field of lysosomal storage diseases, which many of you are probably familiar with. These are severe, chronic genetic diseases. We started specifically with Fabry, Gaucher and Pompe. Each of these diseases is characterized by a genetic mutation that leads to a deficiency of a single enzyme which leads to an accumulation of a single substrate, which goes on to cause clinical symptoms for patients.

The current treatments for the LSDs are biweekly infusions of recombinant enzyme known as enzyme replacement therapy.

So this slide gives you an overview of the pharmacological chaperone technology and specifically in the lysosomal storage diseases. So, in each of these diseases -- each of these diseases is caused by a broad range of genetic mutations that all cause that same fundamental defect of a deficiency of one enzyme.

Interestingly, a lot of these mutations allow for the production by the cell of a form of the enzyme that is capable of functioning but simply is unstable or casually referred to as misfolded because of a single mutation. It's often a missense mutation leading to a substitution of one amino acid. But the active site of the enzyme is intact, and the enzyme as a result is what we call catalytically competent. But, because it is unstable, the cell will recognize it as a foreign or mutant protein and the quality-control mechanisms will ensure that it is discarded.

So, the role of the pharmacological chaperone is to take an oral medication, an oral drug -- it's orally bioavailable, has excellent buyer distribution, and these molecules are designed to specifically target, bind to and stabilize the enzyme in each case, each molecule very specific for the enzyme deficients in that disease -- bind to the active site, confer stability to that enzyme, as a result essentially allowing the cell to recognize it more like the normal or wild type enzyme which results in it being released from the endoplasmic reticulum, traffic through the golgi and eventually to the lysosome as it is supposed to be in the natural mechanism of the cell. That's the first major step of the science.

The second is, importantly, we need to get our molecule out of the way so the enzyme is...

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