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

afternoon call. And here's Bill Maloney. Good afternoon, Charlie. A bit of a quiet day for the main US averages with a dal crently hired by twenty two points, Sesamees gain six and NASAC outperforms gaining thirty one. The small cap six hundred is up a point and the US ten yield at one point five percent. Half of the sub sectors are trading higher, led by games and technology,

materials and the financials. Energy and consumer staples led to the downside down Transports fall seven, as a biotex rise ten, and the vix is down by two percent. Down leaders included DuPont, Apple and Home Depot, while Mark and Chevron led to the downside. Hasbro fell eight percent after earnings, while Bank of America rose as much as four point two after its results some of the names pointing after the belt and I include IBM, Netflix, Yahoo, and vm ware.

Live from the first breaking news ascom Bill Maloney. Charlie, and we thank you very much, Bill Maloney, and to hear live breaking news over your Bloomberg type squawk squ a w K on your terminal. I'm Charlie Pellett and that is a Bloomberg business flash. Now on Bloomberg Radio, we take stock of healthcare chre. We call it k B for sure. It's a major source of healthcare expenditures. Yes you have. Drug pricing has been at the forefront, certainly in the political brown for the last few months.

I think we're living in a very special time in the history of the pharmaceutical industry. Before we start any pharmaceutical discovery, we want to understand the patient biology. For when three percent of all biotext that start actually get a drug of fruit, it's a very hard thing to do. Bloomberg takings to healthcare and focus on Bloomberg Radio. Orphan

drugs in focus. What is an orphan drug? Well, according to the Orphan Drug Act going back to nine eighty three, and orphan drug qualifies if they are intended to treat a disease affecting less than two hundred thousand Americans and under the Acts, sponsors would qualify for a seven year FDA administered orphan drug exclusivity as well as tax credits for R and D costs, as well as grants and protocol assistants. Here to tell us more as John Crowley,

he is the chairman and the chief executive of Amarchus Therapeutics. John, thanks very much for being with us. Great thanks to him. Great to be here. Tell us about Amaricus Therapeutics and how this fits into the rare drug orphan drug marketplace. Sure, of course, the Amarchus Therapeutics as a biotechnology company where we focus on making medicines for rare human genetic diseases

invac you mentioned orphan disorders. Many of the disease as we work in would really be characterized as ultra orphan diseases, diseases that might only affect several thousand people living with rare diseases. Tall us the personal history of Amicus Therapeutics and what you're trying to do. Sure, so, our company's

history is a little bit different than others. I got into the field of biotechnology late in the nineteen nineties after our two youngest children, Megan and Patrick, were diagnosed with one of these orphan disorders, a neuromuscular disease called

Pompey disease. And back in the time when Megan was fifteen months old and Patrick was seven days old, we were told that there was no research, there was nothing that could be done, and we went from you know, I was only a couple of years at a business school and started a small little biotech at the time that's now grown to become Amicus Therapeutics. We were focused on making medicines for a range of these rare orphan diseases, including Pompey disease now Pompey disease. As you say, the

treatment is in early trials, give us an update. Sure, so there is an approved drug. One of the first drugs we worked on years ago that was brought to market by gen Zime that was approved in two thousands and six, and our kids have been on that drug as a first generation therapy for a number of years now. But what we will focused on at Amicus is making um novel biologics where they're differentiated ways in making these medicines where they have the potential to offer other benefits

for people living with diseases like Pompey. So for our pump pay drug, it's at a very exciting stage. After a decade of research and development, it's now just beginning its trials in patients and by the end of this year we'll get the first look at that data and it's something we're very excited about. And it follows on the heels of research we've done in another rare orphan disease called fabry disease, where we actually were successful now in Europe and have that drug approved and are now

just commercializing it in Europe. So it fits in with our bigger vision of we want to build one of the world's largest biotechnology companies focused on the rare and orphan diseases. And although anchored in our family's journey, that's only one important part of the overall picture of what's now becoming Amicusts. Tell us about a piece that you've added to the picture in terms of an acquisition and how that fills out your strategy. Yeah, we've done several acquisitions.

You know, We've got great research and development, very steeped and excellent science at Amaricus. But where something fits in with our mission of addressing the devastating diseases, where technologies can make a meaningful difference, we will acquire companies. We just recently acquired a small company called Miamed working in one of these devastating, rare diseases, a disease so new it doesn't even have a name. It's only known by

its genetic mutation c d k L five. Started by a family here in New York who had a daughter Mia with the disease in me is now six years old, and a dad who went out found scientists, raise money, started a small company very familiar for me, and we met each other and started to talk about how what we at Amicists could do to advance the science. So first and foremost it has to be excellent science, and

we think that's what we have here. It's an early program that when we're excited about that, we think we can also make a difference for people living with that that devastating disorder as well. Can you commented all about the stock of Amicus Therapeutics. It's got to be a little bit painful because the stock is down about so far this year. Is that something that concerns you. Well, you know, we're we're in line with our peers in biotechnology.

That's still not good enough. I think that we should separate from our peers. I think we have a tremendous pipeline and portfolio. Over the last two years we've significantly increased the value of Amicus. Has been a tough year in biotech, but I think with the programs that we have now, having just a month ago an approved drug for a rare disease puts Us in a very special place within biotechnology, being one of those unique companies that has a now marketed product that it took through its

own pipeline. So the goal would be to take our science, art technology, our drugs and are really really intense patient focus and continue to increase the stock Brice and the value of the company. And I really believe if we make great medicines that will translate into tremendous your older value. John Crowley is the chairman and the chief executive of Amicus Therapeutics. This is Bloomberg. Bloomberg Taking Stock is brought

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