Amicus scraps a top rare disease drug after it flunks a pivotal test — and shares spike

Just two months after Amicus Therapeutics $FOLD got a rare reprieve from the FDA, clearing the path for a new marketing application for its lead drug migalastat, the biotech has been hammered by a severe setback on its number two drug development effort. The company reported this morning that SD-101 failed a Phase III study, missing the co-primary and key secondary endpoints in treating epidermolysis bullosa.

John Crowley, Amicus

There was no evidence of statistical superiority in wound closing — with the drug actually underperforming the placebo on the key measures — and Amicus execs say they will scrap any other development efforts, though patients in the pivotal can remain on therapy if they choose.

Amicus shares dropped 14% soon after the news hit, but then quickly — and remarkably — turned back north into the green as investors and analysts rallied behind the company.

This rare and devastating genetic disease is characterized by papery thin skin subject to severe blistering and tearing. Most patients —and there are some 30,000 to 40,000 in the world’s major drug markets — die before the age of 30.  There are no therapies approved for the ailment.

Amicus CEO John Crowley arranged to buy the drug in an $847 million deal — $229 million up front — for Scioderm back in 2015. At the time, he told me he felt certain that the therapy was on track to become a blockbuster.

Analysts, though, weren’t nearly as enthusiastic, which helps explain the muted investor response. Leerink’s Joseph Schwartz put it this way:

We believe the negative news will cause nominal weakness of a dollar or so for the stock today, but based on already cautious investor sentiment for the EB program, we believe this blip could be short-lived as investors anticipate additional positive Ph.1/2 Pompe data, which is also expected in 3Q.

Crowley can still look to a renewed effort to gain an approval for migalastat, which regulators at the FDA initially stopped in its tracks with a demand for a new study. That was before Scott Gottlieb took over as commissioner, and President Donald Trump voiced a full-throated call for faster action at the agency.

Crowley said:

In seeking to develop novel, high quality therapies for those living with devastating rare diseases we may sometimes fail. But we would rather be the first to fail, than the last to try. Our vision at Amicus remains steadfast and is focused on building a leading global biotechnology company that delivers significant benefits for people living with rare diseases.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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