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Old 10-08-2014, 08:35 PM
Tupelo3 Tupelo3 is offline
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Tupelo3 Tupelo3 is offline
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Join Date: Mar 2013
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Quote:
Originally Posted by badboy99 View Post
I find this report very confusing. I've been following this drug, AT2101, for many years as I've been a shareholder in the manufacturer, Amicus Therapeutics (FOLD). They are headquartered close to where I live. They've reported directly on this, or similar, pre-clinical research at least 8 years ago and maybe even longer. I'm not sure why they are now coming out with similar research out of UCLA, or why it never went forward with human clinical studies. Possibly it's a money issue because they are a small biotech. They have just reported very good phase 3 results for the same drug for Fabry Disease, an inherited lysosomal storage disorder. They will be applying for an NDA soon. Fabry is a rare disease, and thus, Amicus was able to get orphan drug status. There is a lot of money to be made, and much easier approval, for orphan drugs for rare diseases. The drug prices are huge and many small biotechs would rather spend their time and money on that research rather than more complicated trials that would be required for PD.

The stock price has more than tripled recently because of the Fabry news, so maybe they will raise some more money to work on PD, but it will be many years, if at all, before they can complete all of the required trials. Of course, they will also need money just to see this drug through to the finish line for Fabry's.

Just to show you want I mean about the research being somewhat outdated, MJFF funded and reported on their pre-clinical studies back in 2006:

https://www.michaeljfox.org/foundati...p?grant_id=284

Confusing............
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badboy99 (10-09-2014), lab rat (10-09-2014)