Amicus bags PhIII rare disease drug in $847M Scioderm buyout

Amicus Therapeutics has stepped in to snap up the late-stage rare disease biotech Scioderm, beefing up its orphan drug pipeline in exchange for $229 million in stock and cash along with a promise of up to $618 million more for meeting a slate of milestones. 

The buyout leaves Amicus ($FOLD) with its lead drug Galafold under regulatory review, a new drug that could be filed in the near term and a third program entering the clinic--with CEO John Crowley prepping for more deals as the company builds out a global commercial team.

Amicus completes this buyout a little more than a month after Scioderm started a rolling submission of its NDA for Zorblisa (SD-101), an experimental drug for epidermolysis bullosa, a condition that leaves children's skin papery thin and fragile, subject to tearing and blistering. Most patients--and there are some 30,000 to 40,000 in the world's major drug markets--die before the age of 30. 

Data from the late-stage study is due in the first half of next year, with Amicus looking for a regulatory green light on a drug Crowley believes could be a blockbuster.

The market for Zorblisa, says the CEO, is worth "a billion dollars-plus." Adds Crowley: "We want to be one of the leading biotech companies focused on rare diseases."

Not that the deal is without risk. TheStreet notes that Zorblisa failed its Phase IIb study, with a small subgroup of patients in the top dose achieving statistical significance compared to a placebo. That top dose is being taken into the Phase III with the blessing of regulators. 

There's an added bonus involved in this deal. If Zorblisa is approved, the owners could qualify for a priority review voucher--an asset that's been worth hundreds of millions of dollars in recent deals. Amicus will pay Scioderm's investors either $100 million or half the sales price for the voucher, whichever is less.

Crowley has had a front row seat on Scioderm's progress as a board member of the company for the last two years. 

Little Durham, NC-based Scioderm was unique among the virtual crowd back in 2013, when the FDA handed out one of its first breakthrough therapeutic designations to the biotech. The company raised a $20 million B round late last year and was named a Fierce 15 company back in 2013, when it was still in early development. At the time of the buyout, its staff had grown to 9 and CEO Robert Ryan will now join Amicus in a senior executive position.

Amicus has been focused primarily on Galafold (migalastat), a treatment for Fabry disease. The biotech experienced a severe setback a few years ago when the drug failed a comparison study with a placebo. The failure spurred GlaxoSmithKline ($GSK) to drop out of its partnership, but Amicus came back with a new plan to use a different biomarker on symptoms of the disease. That strategy paid off with successful Phase III studies of the drug, comparing well with Sanofi's ($SNY) Fabrazyme and Shire's ($SHPG) Replagal in two measures of kidney function. And that should help make the case for switching patients from a regularly infused drug that costs more than $200,000 a year to an oral therapy.

European regulators have granted an accelerated review of the treatment while an NDA at the FDA is slated for delivery before the end of this year.

Amicus has hired 20 people for its European commercial operation, says the CEO. As new products are added, he forecasts, that should grow into a global operation with hundreds of staffers pursuing sales of $500 million to more than a billion dollars on each of three products. As this deal cost $125 million in cash, he says Amicus's business development team is well positioned to hunt down more tech and product deals, with an early focus on some early-stage deals this time around.

Amicus is hunting new deals in a hot market. Rare disease therapies can earn well into 6 figures, making it an attractive market for a host of companies. Other companies like Alexion ($ALXN) and BioMarin ($BMRN) have done extremely well with investors looking to share the profits.

Crowley has a personal connection to the genetic diseases he focuses on. In 1998, two of his children were diagnosed with Pompe disease. 

Special Report: FierceBiotech's 2013 Fierce 15 - Scioderm

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