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Amicus Takes Aim at Rare Brain Disorder with MiaMed Acquisition

Xconomy New York — 

Rare disease drug developer Amicus Therapeutics has added a new prospect to its pipeline with the acquisition of a biologic program that the company hopes will eventually treat a genetic disorder whose seizures and neurological impairments have no approved treatment or cure.

That preclinical program comes from MiaMed, a company that Amicus (NASDAQ: FOLD) said it acquired on Wednesday. Amicus paid about $6.5 million—$1.8 million in cash and $4.7 million in Amicus stock, according to a press release announcing the deal. The Cranbury, NJ-based company will pay MiaMed shareholders up to $18 million more if the program achieves clinical and regulatory milestones, and an additional $65 million if Amicus successfully brings a new treatment to market. Those milestones could be paid in some combination of cash or Amicus stock, the company said in a regulatory filing.

MiaMed focused on replacing a missing protein involved in normal brain development. People who don’t make enough of this protein, called cyclin-dependent kinase-like 5 (CDKL5), start having seizures in infancy, according to the National Organization for Rare Disorders. Later, these patients may go on to have trouble walking, talking, or feeding themselves, and many are confined to wheelchairs. Amicus says there are more than 1,200 documented cases of CDKL5 deficiency worldwide, a number that is increasing as genetic testing for the disorder becomes more common.

The CDKL5 program joins an Amicus pipeline that has several compounds in later stages of development. In May, the company received European approval for migalastat (Galafold), a treatment for Fabry disease. The rare disorder renders the body unable to clear out a certain type of fat, which can then damage the kidneys or lead to heart attacks. Amicus’ drug pipeline also includes a compound in mid-stage clinical trials as a potential treatment for the rare skin disorder epidermolysis bullosa, and another being studied in Pompe disease, a disorder in which an enzyme deficiency impairs muscular function.

In a prepared statement, Amicus CEO John Crowley said that while the company is focused on launching the Fabry disease drug and advancing its other drug candidates, the CDKL5 program fits the company’s strategy of expanding its biologics pipeline.

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