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Amicus' Crowley on Expanding Pipeline for Rare Diseases (Audio) · Bloomberg Intelligence

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  Speaker 1: and we thank you very much, Bill Maloney, and to
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  Speaker 1: a w K on your terminal. I'm Charlie Pellett and
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  Speaker 1: that is a Bloomberg business flash. Now on Bloomberg Radio,
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  Speaker 1: we take stock of healthcare chre. We call it k
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  Speaker 1: B for sure. It's a major source of healthcare expenditures.
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  Speaker 1: Yes you have. Drug pricing has been at the forefront,
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  Speaker 1: certainly in the political brown for the last few months.
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  Speaker 1: I think we're living in a very special time in
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  Speaker 1: the history of the pharmaceutical industry. Before we start any
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  Speaker 1: pharmaceutical discovery, we want to understand the patient biology. For
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  Speaker 1: when three percent of all biotext that start actually get
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  Speaker 1: a drug of fruit, it's a very hard thing to do.
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  Speaker 1: Bloomberg takings to healthcare and focus on Bloomberg Radio. Orphan
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  Speaker 1: drugs in focus. What is an orphan drug? Well, according
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  Speaker 1: to the Orphan Drug Act going back to nine eighty three,
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  Speaker 1: and orphan drug qualifies if they are intended to treat
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  Speaker 1: a disease affecting less than two hundred thousand Americans and
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  Speaker 1: under the Acts, sponsors would qualify for a seven year
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  Speaker 1: FDA administered orphan drug exclusivity as well as tax credits
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  Speaker 1: for R and D costs, as well as grants and
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  Speaker 1: protocol assistants. Here to tell us more as John Crowley,
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  Speaker 1: he is the chairman and the chief executive of Amarchus Therapeutics. John,
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  Speaker 1: thanks very much for being with us. Great thanks to him.
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  Speaker 1: Great to be here. Tell us about Amaricus Therapeutics and
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  Speaker 1: how this fits into the rare drug orphan drug marketplace. Sure,
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  Speaker 1: of course, the Amarchus Therapeutics as a biotechnology company where
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  Speaker 1: we focus on making medicines for rare human genetic diseases
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  Speaker 1: invac you mentioned orphan disorders. Many of the disease as
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  Speaker 1: we work in would really be characterized as ultra orphan diseases,
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  Speaker 1: diseases that might only affect several thousand people living with
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  Speaker 1: rare diseases. Tall us the personal history of Amicus Therapeutics
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  Speaker 1: and what you're trying to do. Sure, so, our company's
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  Speaker 1: history is a little bit different than others. I got
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  Speaker 1: into the field of biotechnology late in the nineteen nineties
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  Speaker 1: after our two youngest children, Megan and Patrick, were diagnosed
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  Speaker 1: with one of these orphan disorders, a neuromuscular disease called
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  Speaker 1: Pompey disease. And back in the time when Megan was
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  Speaker 1: fifteen months old and Patrick was seven days old, we
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  Speaker 1: were told that there was no research, there was nothing
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  Speaker 1: that could be done, and we went from you know,
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  Speaker 1: I was only a couple of years at a business
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  Speaker 1: school and started a small little biotech at the time
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  Speaker 1: that's now grown to become Amicus Therapeutics. We were focused
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  Speaker 1: on making medicines for a range of these rare orphan diseases,
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  Speaker 1: including Pompey disease now Pompey disease. As you say, the
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  Speaker 1: treatment is in early trials, give us an update. Sure,
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  Speaker 1: so there is an approved drug. One of the first
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  Speaker 1: drugs we worked on years ago that was brought to
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  Speaker 1: market by gen Zime that was approved in two thousands
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  Speaker 1: and six, and our kids have been on that drug
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  Speaker 1: as a first generation therapy for a number of years now.
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  Speaker 1: But what we will focused on at Amicus is making
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  Speaker 1: um novel biologics where they're differentiated ways in making these
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  Speaker 1: medicines where they have the potential to offer other benefits
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  Speaker 1: for people living with diseases like Pompey. So for our
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  Speaker 1: pump pay drug, it's at a very exciting stage. After
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  Speaker 1: a decade of research and development, it's now just beginning
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  Speaker 1: its trials in patients and by the end of this
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  Speaker 1: year we'll get the first look at that data and
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  Speaker 1: it's something we're very excited about. And it follows on
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  Speaker 1: the heels of research we've done in another rare orphan
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  Speaker 1: disease called fabry disease, where we actually were successful now
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  Speaker 1: in Europe and have that drug approved and are now
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  Speaker 1: just commercializing it in Europe. So it fits in with
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  Speaker 1: our bigger vision of we want to build one of
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  Speaker 1: the world's largest biotechnology companies focused on the rare and
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  Speaker 1: orphan diseases. And although anchored in our family's journey, that's
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  Speaker 1: only one important part of the overall picture of what's
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  Speaker 1: now becoming Amicusts. Tell us about a piece that you've
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  Speaker 1: added to the picture in terms of an acquisition and
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  Speaker 1: how that fills out your strategy. Yeah, we've done several acquisitions.
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  Speaker 1: You know, We've got great research and development, very steeped
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  Speaker 1: and excellent science at Amaricus. But where something fits in
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  Speaker 1: with our mission of addressing the devastating diseases, where technologies
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  Speaker 1: can make a meaningful difference, we will acquire companies. We
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  Speaker 1: just recently acquired a small company called Miamed working in
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  Speaker 1: one of these devastating, rare diseases, a disease so new
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  Speaker 1: it doesn't even have a name. It's only known by
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  Speaker 1: its genetic mutation c d k L five. Started by
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  Speaker 1: a family here in New York who had a daughter
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  Speaker 1: Mia with the disease in me is now six years old,
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  Speaker 1: and a dad who went out found scientists, raise money,
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  Speaker 1: started a small company very familiar for me, and we
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  Speaker 1: met each other and started to talk about how what
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  Speaker 1: we at Amicists could do to advance the science. So
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  Speaker 1: first and foremost it has to be excellent science, and
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  Speaker 1: we think that's what we have here. It's an early
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  Speaker 1: program that when we're excited about that, we think we
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  Speaker 1: can also make a difference for people living with that
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  Speaker 1: that devastating disorder as well. Can you commented all about
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  Speaker 1: the stock of Amicus Therapeutics. It's got to be a
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  Speaker 1: little bit painful because the stock is down about so
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  Speaker 1: far this year. Is that something that concerns you. Well,
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  Speaker 1: you know, we're we're in line with our peers in biotechnology.
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  Speaker 1: That's still not good enough. I think that we should
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  Speaker 1: separate from our peers. I think we have a tremendous
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  Speaker 1: pipeline and portfolio. Over the last two years we've significantly
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  Speaker 1: increased the value of Amicus. Has been a tough year
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  Speaker 1: in biotech, but I think with the programs that we
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  Speaker 1: have now, having just a month ago an approved drug
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  Speaker 1: for a rare disease puts Us in a very special
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  Speaker 1: place within biotechnology, being one of those unique companies that
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  Speaker 1: has a now marketed product that it took through its
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  Speaker 1: own pipeline. So the goal would be to take our science,
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  Speaker 1: art technology, our drugs and are really really intense patient
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  Speaker 1: focus and continue to increase the stock Brice and the
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  Speaker 1: value of the company. And I really believe if we
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  Speaker 1: make great medicines that will translate into tremendous your older value.
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  Speaker 1: John Crowley is the chairman and the chief executive of
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  Speaker 1: Amicus Therapeutics. This is Bloomberg. Bloomberg Taking Stock is brought
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