Amicus Therapeutics, Inc. (FOLD)

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Amicus Therapeutics, Inc. (FOLD)

Lazard Capital Markets Healthcare Conference Call

November 14, 2012 3:30 pm ET


William D. "Chip" Baird, III – Chief Financial Officer

John F. Crowley – Chairman and Chief Executive Officer


Bill J. Tanner – Lazard Capital Markets LLC


Bill J. Tanner – Lazard Capital Markets LLC

Okay, good afternoon. I am Bill Tanner one of the biopharma analysts at Lazard Capital Markets. I’d like to thank everybody for attending the 9th Annual Lazard Capital Markets Healthcare Conference. We hope the last couple of days have been useful and valuable to you.

Saving the best for last, very pleased to have Amicus Therapeutics presenting. As most of you know Amicus is a pioneer in the development of chaperone technologies and this had some exciting news recently as it relates to using chaperones with in combination with enzyme replacement therapies and obviously the big milestone before the end of the year would be hopefully the immunotherapy data for migalastat.

So Chip Baird, CFO, who will be presenting for us also want to acknowledge Bradley Campbell, he is the Chief Business Officer at table, as well as Sara Pellegrino. So thanks very much for coming to the conference. Chip report your comments.

William D. "Chip" Baird, III

Thanks Bill. And I’d like to thank not only Bill Tanner, but Steve Sands and the rest of the team at Lazard. We’re really pleased to be here and updating everyone on Amicus and everything we’ve been doing it’s been a great 2012 with a lot of very important milestones coming up here in the near-term.

We’ll refer to our Safe-Harbor provision. We will be making forward-looking statements today. So Amicus Therapeutics by way of background is focused on the discovery, development and commercialization of pharmaceutical chaperones for the treatment of rare and orphan diseases. Since our founding as a small research-based organization, we’ve made tremendous progress in our goal of developing a fully integrated biopharma company, delivering innovative therapies for patients living with rare disease.

Our company is built on three pillars of value. The first is our pharmacological chaperone platform technology, which utilizes small molecules that’s selectively target niche folded and unstable proteins. Our chaperones can bind to it stabilizes the patients own enzyme increased in the patients and dramatic activity. It can also work in combination with existing enzyme replacement therapies again stabilizing or trafficking and ultimately enhancing the activity in patients with lysosomal storage disorders.

Our second pillar value is our pipeline. Using our chaperone technology, we built it a deep and robust pipeline. We developed a significant expertise in global expertise and disease, in rare disease, clinical research, medical affairs and patient advocacy and using that expertise, we’ve developed the pipeline includes multiple programs in Phase 3 and Phase 2 development. I’ll tell you more about those today.

And then finally, the last pillar that we mentioned here is our collaboration with GSK. We’ve collaborated with GSK on our more advanced program migalastat for the treatment of patients with Fabry disease. GSK provides important financial support as well as clinical and regulatory expertise. And under the collaboration, Amicus has responsibility for the U.S. commercial efforts, while GSK leads commercial efforts outside the U.S. This arrangement preserves important value for our shareholders, while at the same time, accelerating the introduction of migalastat for patients living with Fabry disease outside the United States.

So at Amicus, we are developing pharmaceutical chaperones as next generation treatments for patients with lysosomal storage disorders such as Fabry disease, Pompe disease Gaucher disease. Chaperones work in two distinct ways. As a monotherapy, our chaperones selectively bind and stabilize the patients own enzyme, stabilizing the mutated and misfolded enzyme, traffic in that enzyme to the lysosome where it can have its intended activity of cleaning substrate.

Our chaperones can also be used in combination with existing enzyme replacement therapies. They work in that similar manner. In this case, you are taking infused exogenous enzyme replacement therapies and then binding those therapies with a custom targeted chaperone again stabilizing that the chaperone and stabilizing the enzyme increasing tissue uptick and ultimately increasing substrate reduction.

On this latter point on the combination approach, we’d now evaluated our chaperones in conjunction with seven different marketed or investigational enzyme replacement therapies in preclinical animal models and in all seven cases, we are seeing clear evidence of increased enzymatic activity.

So using our pharmaceutical chaperone technology, we build a late stage and diversified pipeline again most of as program migalastat for Fabry disease and here we have two phase 3 studies ongoing, Study 011 and Study 012 as a model therapy. We also have a combination of co-administration program that’s in Phase 2 development and we have a longer-term next generation coformulation product that’s in late stage preclinical development.

Second most advanced programs AT2220, this is a co-administration program for the treatment of Pompe disease and there we have a Phase 2 program ongoing. In addition to those two assets, we have programs in Parkinson’s, Gaucher and other non-disclosed lysosomal storage disorders.

With the exception of migalastat Amicus owns 100% of the world right commercial rights to all of our pipeline. Focusing a little more on the GSK collaboration, we originally enter the collaboration fall at 2010 and we extend that collaboration in July of this year. Key points on the expansion of that collaboration include additional $18 million investment that was done in this summer; we also gained the U.S. commercial rights to migalastat in all its forms which accelerates our path to becoming a commercial stage company. We also gained U.S. commercial rights to the co-formulated program. That’s a longer term asset but that’s a very exciting program and we’ll show you some data from that program at the end of the presentation. And finally GSK recommitted to their 60% cost sharing arrangement to support all forms of development of migalastat monotherapy co-administration and co-formulation.

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