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Amicus Therapeutics, Inc. (FOLD)

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Amicus Therapeutics, Inc. (FOLD)

UBS Global Life Sciences Conference Call

September 20, 2012, 10:30 am ET

Executives

Bradley Campbell - Chief Business Officer

Analysts

Presentation

Unidentified Analyst

Good morning everyone. Welcome to the next session of the UBS 2012 Global Life Sciences Conference. It’s my pleasure to present the next speaker from Amicus Therapeutics, Mr. Bradley Campbell; the Chief Business Officer of Amicus Therapeutics. And I would just like to remind everyone that after this session, there will be a breakout session in the [Carnegie Room at the Campus level]. Thank you very much

Bradley Campbell

Great; thank you [Terence]. Thanks to UBS for having us here this morning; welcome everybody. I’ll refer to you to our Safe Harbor provisions. I will be making certain forward-looking statements this morning.

So let me start by sharing with you how we position Amicus and why it's such an exciting value proposition for our shareholders. First and foremost, we have our small molecule, pharmacological chaperones which target and bind to misfolded and unstable proteins, has two fundamental uses. One as a monotherapy for patients with the appropriate genetic mutations where they can take our small molecules chaperones instead of their enzyme replacement therapy for the only treatment for their Fabry disease. And second, for patients who lack the appropriate genetic mutations, we can take our small molecules, combine them with enzyme replacement therapy and make that enzyme replacement therapy better.

Second, with our global clinical capabilities and capacities that puts us at the forefront of developing therapies for rare and orphan diseases, we have clinical sites in over 20 countries around the world. We have two ongoing Phase III registration studies for Fabry disease. We have two ongoing global Phase II studies in co-administration; one for Fabry and one from Pompe and I will talk about both of those later in the conversation. And then we also have a recently expanded deal with the GlaxoSmithKline rare diseases; discovers all uses of migalastat for Fabry disease as global co-development and cost sharing and now Amicus has U.S. rights to all uses of Fabry disease and GSK has ex-US rights.

So again, one technology with two novel applications, first for patients with appropriate genetic mutations that can take our small molecule competitive inhibitors that bind to stabilize and elevate the enzyme in a patient’s own mutated enzyme increasing its trafficking for lysosome with a small molecule can dissociate and the enzyme is free to turnover substrate.

For patients who lack the appropriate genetic mutations who have to stand their enzyme replacement therapy just as we can bind to and stabilize a patient’s own mutated enzyme, we can also bind to and stabilize exogenous recombinant protein increasing its activity in the blood, increasing the amount of active enzyme taking up in the key tissues of disease and reducing substrate over ERT alone.

We have now demonstrated this in seven different enzyme replacement therapies in preclinical animal models, three for Fabry disease, three for Pompe disease and one for Gaucher disease; so a very important extension of our technology.

This helps lead to a very robust product pipeline from a monotherapy perspective as I mentioned we have our two ongoing Phase III studies for Fabry disease Study 011 which is our U.S. registration study and Study 012 which is our ex-U.S. registration study. We also have ongoing preclinical work in Parkinson’s disease which takes advantage of our knowledge in Gaucher disease and uses a small molecule chaperone to target GCase to help reduce synuclein in preclinical anima models.

From a combination therapy perspective, as I mentioned we have our ongoing on Phase II co-administration study with migalastat combined with either Fabrazyme or Replagal. We also have our Study 010 which our Phase II co-administration study for our small molecule chaperone AT2220 co-administered with either Myozyme or Lumizyme. We've ongoing preclinical work looking at combination therapy for Gaucher disease using our small molecules AT2101 and AT3375 combined with ERT’s for Gaucher disease and we have a recently announce Fabry co-formulation study which I'll talk more about later in the conversion. This is in preclinical phase and its part of the rational for expanding our relationship with GSK. And then finally, we do have multiple ongoing studies in preclinical animal models for other LSD targets.

Now again, we recently expanded our collaboration with GSK. This is a collaboration that began back in November of 2010 under the leadership of Marc Dunoyer, who heads up the GSK rare diseases business unit. Originally it was focused on the migalastat for monotherapy and for co-administration, but after lot of the work that we did together looking at the possibilities around combination therapy we expanded the collaboration to include co-formulation as well.

Now Amicus will be responsible for commercializing these products in the US and GSK will be responsible for commercializing outside the US. In addition, GSK put another $18.5 million in equity into Amicus to raise their ownership stake back to 19.9%. We do continue to have global development cost sharing which is 25-75 this year and 40-60 in the remaining years of the collaboration. And finally, GSK is eligible for certain moderate approval and launch milestones for each of the Fabry programs. It’s a very important part of our value proposition and a key partner for us.

This is in part would lead us to our strong financial position. We announced at the end of second quarter that we had $95.8 million in cash, we do project that we’ll end the year in over $90 million in cash, which we think will fund us well beyond our 2013 operating plan. Importantly too, we worked hard in 2012 to strengthen our balance sheet.

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