Following last week’s theme, I will be discussing two smaller companies that just released positive Phase III results on their lead candidates. Amicus Therapeutics (FOLD), currently down to $7.98 a share from its 52 week high of $18.83, went up 12% premarket Tuesday after releasing encouraging data on migalastat in Fabry disease. Members of the Amicus team delivered an oral explanation on migalastat to the European Union’s (EU) Committee for Medicinal Products for Human Use (CHMP) in February, and they expect to receive an opinion on the drug at the next CHMP meeting (March 29 - April 1, 2016). They also expect to meet with the FDA in Q2 to present data and discuss a potential pathway to submit a New Drug Application (NDA) for migalastat in
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However patients often die prematurely of complications from strokes, heart disease, or kidney failure. Current Fabry treatments such as Sanofi’s (SNY) fabrazyme and Shire’s (SHPG) replagal require bi-weekly intravenous infusions and work to replace the patient’s mutated or missing native enzyme and clear GL3 from the body. Migalastat takes a different approach, acting as a “chaperone” that accompanies, or fixes, a patient’s native enzyme. Its effect is to stabilize the native enzyme and increase cellular activity to break down GL3. Without this chaperone, the native enzyme in a Fabry patient does not function well and can't break down …show more content…
After the voting period ends, Slingshot Insights will interview a Subject Matter Expert, then post a transcript and recording of the conversation on our website.
Vote now in the comments below/direct messages to me within the voting period to express your interest. Seeking Alpha readers can also join and add questions on Option 1: FOLD here or Option 2: ACRX here and get expert insights to enhance your investment diligence.
Readers of Seeking Alpha that would like to access the winning call may do so free of charge with the coupon code listed on the project page.
This column is designed to be engaging for a wide variety of biotech investors. Not every company might be of interest to everyone, so stay tuned for next week's match-up!
Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.
I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this
However patients often die prematurely of complications from strokes, heart disease, or kidney failure. Current Fabry treatments such as Sanofi’s (SNY) fabrazyme and Shire’s (SHPG) replagal require bi-weekly intravenous infusions and work to replace the patient’s mutated or missing native enzyme and clear GL3 from the body. Migalastat takes a different approach, acting as a “chaperone” that accompanies, or fixes, a patient’s native enzyme. Its effect is to stabilize the native enzyme and increase cellular activity to break down GL3. Without this chaperone, the native enzyme in a Fabry patient does not function well and can't break down …show more content…
After the voting period ends, Slingshot Insights will interview a Subject Matter Expert, then post a transcript and recording of the conversation on our website.
Vote now in the comments below/direct messages to me within the voting period to express your interest. Seeking Alpha readers can also join and add questions on Option 1: FOLD here or Option 2: ACRX here and get expert insights to enhance your investment diligence.
Readers of Seeking Alpha that would like to access the winning call may do so free of charge with the coupon code listed on the project page.
This column is designed to be engaging for a wide variety of biotech investors. Not every company might be of interest to everyone, so stay tuned for next week's match-up!
Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.
I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this