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Amicus Therapeutics, Inc. (FOLD)
The 31st Annual J.P. Morgan Healthcare Conference Call
January 9, 2013 6:00 PM ET
John Crowley – Chairman and CEO
Anupam Rama – JP Morgan
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Thank you. Hi, good afternoon, everybody. Thanks for taking time to come to the Annual Amicus Therapeutics presentation. As we are a public company, I’ll refer you to the Safe Harbor Provisions I will be making forward-looking statements.
And I’ll begin with this slide that talks a little about the mission of the company and what we do at Amicus. And that is a company at the forefront of developing next generation medicines in the rear and orphan diseases. And our focus is in the lysosomal storage diseases on improved therapies. And I think as you see the platform evolve here, you’ll see why we say improved.
So, usually I save this slide for the end, at a JP Morgan presentation. I wanted to begin this year because we do think this is such an important and transformational year for Amicus.
So a handful of key messages. First our Fabry Phase 3 monotherapy study is an ongoing Phase 3. As many of you saw back in December we unveiled the interim or stage one results, those were very encouraging and they were consistent with our years of Phase 2 experience in this disease. We also now have updated regulatory guidance from the FDA indicating that they will consider the entirety of the data from stage one to six month stage as well as stage 2 in the study and we think that’s particularly encouraging.
We also are advancing our Pompe ERT Co-Administration Repeat Dose Study, so we had the phase 2 data released just last Friday and that study the repeat dose, the next stage of development in Pompe will begin in 3Q 2013 this year. We also continue to advance the next generation ERT product, the Fabry co-formulated Chaperone ERT product, that advances to the clinic. We’re also announcing at this conference that we have in development a proprietary next generation ERT. And our financial strength, we begin the year with approximately $100 million on the balance sheet.
So, when we talk about the platform technology at Amicus there are really three key parts to that technology. The first is the use of pharmacological Chaperones as a monotherapy, designed specifically to target to bind to and stabilize a patient’s own enzyme to replace the need for enzyme replacement therapy in people with amenable genetic mutations. The second use of the technology is in co-administration. So, in people who don’t have amenable mutations to take this as a monotherapy, like about half the people living with Fabry, our drug can be used to potentially improve the characteristics and therapeutic outcome of existing enzyme replacement therapy, that is a Chaperone take in directly with the ERT product.
And then finally the third application of the technology is the development of next generation enzyme replacement therapies, they offer all the benefit of co-administered ERT and Chaperone products and with co-formulated products that is our own proprietary ERT and also has the additional benefit of stabilizing the protein in the infusion bag as well as in the patient.
So, one platform technology, three novel uses, which leads to our pipeline as you see, multiple programs in clinical development, Chaperone monotherapy programs, two phase 3 programs in Fabry, a late pre-clinical program with a pharmacological Chaperone in Parkinson’s disease and a co-administered series of products, we now have human data, phase 2 data in Fabry as well as in Pompe and those continue to advance. As well as a co-administered program we’re developing in Gouache disease.
And again the third application of the technology is Amicus’ entry into the biologic space scenario which many of us in the management team came from in our days at Genzyme and other companies, here we’re partnered with GSK and JCR, as we are in Fabry and our monotherapy and co-admin programs with GSK. JCR, Japanese biologics company, developing and now manufacturing for GSK and Amicus, a next generation ERT product. And Dr. Rashed I see from JCR here, so welcome. And the co-formulated products continue to advance I’ll have more to say in the presentation of their development timelines.
So, when we talk about investment highlights and the investment thesis for Amicus, we focus on four key foundations of our success in 2013. The first of these are products, and we are again developing these products, multiple phase 3 studies, phase 2 extension studies for the monotherapy and now multiple phase 2 studies with the co-administration of Chaperone and ERT. So, we’re developing products, generating clinical data. And it’s also a platform technology company.
Amicus was originally founded on the concept that you could use these small molecule Chaperones to be combined, I’m sorry, to be used in lieu of enzyme replacement therapy, where they could be taken to enhance the endogenous protein in people with amenable mutations. The use of that platform is really expanded and we’re now aggressively moving into the notion that – with the development, with the notion that we can combine these drugs directly with the ERTs, either as co-administered products or as next generation ERTs.