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