With $229M Deal, Amicus Gets a New Drug And Perhaps a Voucher

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clinical testing, says Crowley. Shire (NASDAQ: SHPG), for instance, bought Cambridge, MA-based Lotus Tissue Repair back in 2013. Founded by Third Rock Ventures in 2011, Lotus was developing a genetically engineered form of collagen as a protein replacement therapy. Shire’s website shows that the effort is still in preclinical testing, however, just as it was when Shire bought Lotus two years ago.

SD-005, which would be marketed as Zorblisa, is a topical cream containing allantoin, a natural substance found in some plants and mammals that is already used in a variety of over-the-counter items like toothpaste and certain skin care products. Allantoin, however, has a short half-life. Scioderm’s cream, as Frank Vinluan explained in this piece last year, keeps allantoin more stable and delivers it in a higher concentration for a longer time. It softens keratin, and in Scioderm’s mid-stage trials it helped reduce and even close some patients’ chronic wounds in a small study. SD-005 would be used daily, and chronically, to deal with these wounds for patients with all forms of EB, Crowley says.

Crowley knows the drug well. He’s been on Scioderm’s board of directors for two years—his only non-Amicus board seat—and developed a “strong relationship” with the startup’s co-founder and CEO Robert Ryan. He approached Scioderm about a buyout several months ago and began exclusive talks. “This deal was never shopped,” he says.

For Amicus, the deal represents the latest step in what’s been a long and winding road. Last year, I profiled Amicus’s quest of more than a decade to create “pharmacological chaperones,” or small-molecule drugs designed to grab misfolded enzymes, force them into the correct shape, and shepherd them to the proper cell. Amicus has seen its share of failures trying to prove the worth of this approach, but it appears to finally be on the verge of a payoff. Its Fabry disease drug, migalastat (Galafold), is currently under review in Europe, and Amicus has been raising cash to prep for the drug’s launch, which could come next year. Amicus hopes to file papers for approval in the U.S. later this year as well, Crowley says.

Shares were worth around $2 apiece when things looked bleak in late 2013. Now they’re worth close to $15, and Amicus is acting as if its worst days are in the rear-view mirror—it’s now a buyer. Though no one should expect another Scioderm-sized deal in the near-term, Crowley says the company is scouting around for other opportunities.

“It’s been a great journey, that’s for sure,” he says.

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