WEBVTT - Amicus' Crowley on Expanding Pipeline for Rare Diseases (Audio)

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<v Speaker 1>Global business news twenty four hours a day's Bloomberg dot Com,

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<v Speaker 1>Charlie Howett. Stocks are higher. We have got forty three

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<v Speaker 1>right over the first Word Breaking news desk for today's

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<v Speaker 1>afternoon call. And here's Bill Maloney. Good afternoon, Charlie. A

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<v Speaker 1>bit of a quiet day for the main US averages

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<v Speaker 1>two after its results some of the names pointing after

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<v Speaker 1>Live from the first breaking news ascom Bill Maloney. Charlie,

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<v Speaker 1>and we thank you very much, Bill Maloney, and to

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<v Speaker 1>hear live breaking news over your Bloomberg type squawk squ

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<v Speaker 1>a w K on your terminal. I'm Charlie Pellett and

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<v Speaker 1>that is a Bloomberg business flash. Now on Bloomberg Radio,

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<v Speaker 1>we take stock of healthcare chre. We call it k

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<v Speaker 1>B for sure. It's a major source of healthcare expenditures.

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<v Speaker 1>Yes you have. Drug pricing has been at the forefront,

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<v Speaker 1>certainly in the political brown for the last few months.

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<v Speaker 1>I think we're living in a very special time in

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<v Speaker 1>the history of the pharmaceutical industry. Before we start any

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<v Speaker 1>pharmaceutical discovery, we want to understand the patient biology. For

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<v Speaker 1>when three percent of all biotext that start actually get

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<v Speaker 1>a drug of fruit, it's a very hard thing to do.

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<v Speaker 1>Bloomberg takings to healthcare and focus on Bloomberg Radio. Orphan

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<v Speaker 1>drugs in focus. What is an orphan drug? Well, according

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<v Speaker 1>to the Orphan Drug Act going back to nine eighty three,

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<v Speaker 1>and orphan drug qualifies if they are intended to treat

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<v Speaker 1>a disease affecting less than two hundred thousand Americans and

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<v Speaker 1>under the Acts, sponsors would qualify for a seven year

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<v Speaker 1>FDA administered orphan drug exclusivity as well as tax credits

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<v Speaker 1>for R and D costs, as well as grants and

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<v Speaker 1>protocol assistants. Here to tell us more as John Crowley,

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<v Speaker 1>he is the chairman and the chief executive of Amarchus Therapeutics. John,

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<v Speaker 1>thanks very much for being with us. Great thanks to him.

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<v Speaker 1>Great to be here. Tell us about Amaricus Therapeutics and

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<v Speaker 1>how this fits into the rare drug orphan drug marketplace. Sure,

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<v Speaker 1>of course, the Amarchus Therapeutics as a biotechnology company where

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<v Speaker 1>we focus on making medicines for rare human genetic diseases

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<v Speaker 1>invac you mentioned orphan disorders. Many of the disease as

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<v Speaker 1>we work in would really be characterized as ultra orphan diseases,

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<v Speaker 1>diseases that might only affect several thousand people living with

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<v Speaker 1>rare diseases. Tall us the personal history of Amicus Therapeutics

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<v Speaker 1>and what you're trying to do. Sure, so, our company's

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<v Speaker 1>history is a little bit different than others. I got

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<v Speaker 1>into the field of biotechnology late in the nineteen nineties

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<v Speaker 1>after our two youngest children, Megan and Patrick, were diagnosed

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<v Speaker 1>with one of these orphan disorders, a neuromuscular disease called

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<v Speaker 1>Pompey disease. And back in the time when Megan was

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<v Speaker 1>fifteen months old and Patrick was seven days old, we

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<v Speaker 1>were told that there was no research, there was nothing

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<v Speaker 1>that could be done, and we went from you know,

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<v Speaker 1>I was only a couple of years at a business

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<v Speaker 1>school and started a small little biotech at the time

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<v Speaker 1>that's now grown to become Amicus Therapeutics. We were focused

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<v Speaker 1>on making medicines for a range of these rare orphan diseases,

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<v Speaker 1>including Pompey disease now Pompey disease. As you say, the

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<v Speaker 1>treatment is in early trials, give us an update. Sure,

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<v Speaker 1>so there is an approved drug. One of the first

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<v Speaker 1>drugs we worked on years ago that was brought to

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<v Speaker 1>market by gen Zime that was approved in two thousands

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<v Speaker 1>and six, and our kids have been on that drug

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<v Speaker 1>as a first generation therapy for a number of years now.

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<v Speaker 1>But what we will focused on at Amicus is making

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<v Speaker 1>um novel biologics where they're differentiated ways in making these

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<v Speaker 1>medicines where they have the potential to offer other benefits

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<v Speaker 1>for people living with diseases like Pompey. So for our

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<v Speaker 1>pump pay drug, it's at a very exciting stage. After

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<v Speaker 1>a decade of research and development, it's now just beginning

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<v Speaker 1>its trials in patients and by the end of this

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<v Speaker 1>year we'll get the first look at that data and

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<v Speaker 1>it's something we're very excited about. And it follows on

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<v Speaker 1>the heels of research we've done in another rare orphan

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<v Speaker 1>disease called fabry disease, where we actually were successful now

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<v Speaker 1>in Europe and have that drug approved and are now

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<v Speaker 1>just commercializing it in Europe. So it fits in with

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<v Speaker 1>our bigger vision of we want to build one of

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<v Speaker 1>the world's largest biotechnology companies focused on the rare and

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<v Speaker 1>orphan diseases. And although anchored in our family's journey, that's

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<v Speaker 1>only one important part of the overall picture of what's

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<v Speaker 1>now becoming Amicusts. Tell us about a piece that you've

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<v Speaker 1>added to the picture in terms of an acquisition and

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<v Speaker 1>how that fills out your strategy. Yeah, we've done several acquisitions.

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<v Speaker 1>You know, We've got great research and development, very steeped

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<v Speaker 1>and excellent science at Amaricus. But where something fits in

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<v Speaker 1>with our mission of addressing the devastating diseases, where technologies

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<v Speaker 1>can make a meaningful difference, we will acquire companies. We

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<v Speaker 1>just recently acquired a small company called Miamed working in

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<v Speaker 1>one of these devastating, rare diseases, a disease so new

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<v Speaker 1>it doesn't even have a name. It's only known by

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<v Speaker 1>its genetic mutation c d k L five. Started by

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<v Speaker 1>a family here in New York who had a daughter

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<v Speaker 1>Mia with the disease in me is now six years old,

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<v Speaker 1>and a dad who went out found scientists, raise money,

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<v Speaker 1>started a small company very familiar for me, and we

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<v Speaker 1>met each other and started to talk about how what

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<v Speaker 1>we at Amicists could do to advance the science. So

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<v Speaker 1>first and foremost it has to be excellent science, and

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<v Speaker 1>we think that's what we have here. It's an early

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<v Speaker 1>program that when we're excited about that, we think we

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<v Speaker 1>can also make a difference for people living with that

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<v Speaker 1>that devastating disorder as well. Can you commented all about

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<v Speaker 1>the stock of Amicus Therapeutics. It's got to be a

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<v Speaker 1>little bit painful because the stock is down about so

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<v Speaker 1>far this year. Is that something that concerns you. Well,

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<v Speaker 1>you know, we're we're in line with our peers in biotechnology.

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<v Speaker 1>That's still not good enough. I think that we should

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<v Speaker 1>separate from our peers. I think we have a tremendous

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<v Speaker 1>pipeline and portfolio. Over the last two years we've significantly

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<v Speaker 1>increased the value of Amicus. Has been a tough year

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<v Speaker 1>in biotech, but I think with the programs that we

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<v Speaker 1>have now, having just a month ago an approved drug

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<v Speaker 1>for a rare disease puts Us in a very special

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<v Speaker 1>place within biotechnology, being one of those unique companies that

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<v Speaker 1>has a now marketed product that it took through its

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<v Speaker 1>own pipeline. So the goal would be to take our science,

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<v Speaker 1>art technology, our drugs and are really really intense patient

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<v Speaker 1>focus and continue to increase the stock Brice and the

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<v Speaker 1>value of the company. And I really believe if we

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<v Speaker 1>make great medicines that will translate into tremendous your older value.

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<v Speaker 1>John Crowley is the chairman and the chief executive of

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<v Speaker 1>Amicus Therapeutics. This is Bloomberg. Bloomberg Taking Stock is brought

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