BioMarin snares orphan drug upstart in $115M buyout

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Continuing its year-long buying spree, BioMarin Pharmaceutical has agreed to pay up to $115 million to snare ZyStor Therapeutics and its preclinical candidate for Pompe disease. BioMarin is slapping down $22 million upfront to seal the deal, which will add an enzyme therapy scheduled to enter the clinic next year.

The FDA has already approved ZyStor's IND app for ZC-701. And Novato, CA-based BioMarin, which specializes in orphan diseases, is pumped about its prospects. The deal "not only provides us with a promising product candidate for Pompe disease but also an exciting new platform technology," said BioMarin CEO Jean-Jacques Bienaime. "ZC-701 has been shown to be more effective in the Pompe mouse model than commercially available replacement enzymes for Pompe disease. In addition, ZyStor's proprietary Glycosylation Independent Lysosomal Targeting technology is applicable to other ERTs and has the potential to deliver more enzyme to lysosomes compared to traditional mannose-6-phosphate targeted approaches. Also, relative to our internal candidate for Pompe, BMN-103, ZC-701 has a faster clinical development timeline, lower development costs, significantly lower cost of goods and lower capital investment."

BioMarin has racked up a string of small buyouts like this. Back in the early part of the year the biotech struck a deal to buy Lead Therapeutics for $18 million. And BioMarin paid $15 million upfront for Huxley Pharmaceuticals.

A busy BioMarin also unveiled a new research collaboration on Monday with the Buck Institute for Age Research. Investigators from both groups will explore new therapies for early-onset familial Alzheimer's.

- read the BioMarin release on the buyout
- here's the story from the San Francisco Business Times

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