Amicus spends $90M on MiaMed; Gains rare disease preclinical program

Amicus Therapeutics ($FOLD), which has seen an FDA delay on one hand and a key European approval on the other over the past year, has gained access to a new, early-stage genetic disease program from its buyout of MiaMed.   

The Cranbury, NJ-based biotech grabs the preclinical candidate from MiaMed that is targeting cyclin-dependent kinase-like 5 (CDKL5) deficiency, a rare, genetic neurological disease for which there is no currently approved treatment. Symptoms of the disease include seizures in infancy followed by severe delays in neurological development.

The preclinical program comes with the $90 million buyout of the tiny European biotech, which sees this split into a number of ways: First, $1.8 million will be paid up in cash with around $4.7 million in Amicus common stock to the former shareholders of MiaMed.

On top of this, the former shareholders of the company can then get up to $18 million should certain clinical and regulatory milestones be hit, with another $65 million on offer if the drug platform reaches key commercial milestones. Amicus said this purchase deal wouldn’t hit its full-year guidance.

“The CDKL5 program fits perfectly with our vision to build a leading global biotechnology company focused on rare and devastating diseases,” said John Crowley, chairman and CEO of Amicus, in a statement. “CDKL5 is a rare and devastating disease with no approved treatment. Most children with CDKL5 have frequent seizures that begin shortly after birth. They experience severe impairment in neurological development, and many of them are unable to walk, talk or care for themselves. We are pleased to partner with the CDKL5 patient and medical community to elevate disease awareness as we advance towards a treatment.”

This comes just over a month after the biotech gained European approval for its Fabry drug Galafold (migalastat), seeing its shares jump 7% on the news.

The biotech had expected to submit its drug to the FDA last year, but in October the agency demanded new data from its clinical trials, as well as another study for the drug, delaying a U.S. approval significantly and adding extra costs to its R&D program.

The drug was once backed by GlaxoSmithKline ($GSK), but the London-based Big Pharma walked away in 2012 after it flubbed a trial.

Amicus’ shares traded up this morning on the news by nearly 10%, after dropping down slightly after hours last night by 1.4%. 

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