John F. Crowley, chairman and CEO of Amicus Therapeutics, said, "With the culmination of several transformative business development transactions and a new financing, we are advancing an Amicus that is better resourced and more sharply focused toward our valuable biologics business. Not only do we own global rights to all of our CHART and Fabry monotherapy programs, but we also have brought in a uniquely engineered, proprietary next-generation ERT in late preclinical development for Pompe disease. We believe we now have the optimal set of technologies, portfolio, financing and leadership team to be able to advance our vision of developing improved therapies for people living with many rare and orphan diseases."
Amicus has acquired Callidus Biopharma, a privately-held biologics company focused on developing best-in-class enzyme replacement therapies (ERTs) for lysosomal storage diseases (LSDs). Callidus' lead ERT is a recombinant human acid-alpha glucosidase (rhGAA) for Pompe disease in late preclinical development.
"With the addition of Callidus' Pompe program, we can move a next-generation Pompe ERT into the clinic in early 2015, approximately 12 months faster than our internal Pompe program,” said Crowley. “Even more significantly, we believe that the Callidus ERT for Pompe is a highly innovative and potentially superior ERT due to its carbohydrate structure which should provide for greater uptake of the ERT into target muscle cells in Pompe patients. For these reasons we believe that the best course forward for patients is to advance the next-generation ERT as quickly as possible instead of the planned phase II study of AT2220 co-administered with ERT. In addition, we believe Callidus' targeting technology complements our CHART platform and can be used together to develop bio-better ERTs."
Callidus shareholders will receive $15 million in shares of Amicus common stock, up to $10 million in milestone payments through phase II development of the Pompe program and up to $105 million for milestones. Hung Do, Ph.D., founder and chief scientific officer of Callidus Biopharma, has been appointed Amicus' senior vice president, discovery biology.
Amicus has restructured the organization to reduce costs and to align its resources with its biologics business strategy. The workforce, including full-time employees across all levels and departments, has been reduced by approximately 14% to 91 employees. Amicus will also close its San Diego research facility and consolidate all operations at its Cranbury, N.J. headquarters. David J. Lockhart, Ph.D., will step down from his role as chief scientific officer and will continue as a member of Amicus' scientific advisory board.
"In broadening our biologics business strategy, and as part of our commitment to judiciously manage our cash flow, we have had to make some difficult decisions in restructuring and realigning our organization," said Crowley.
The company estimates it will record charges of approximately $2.5 million during the fourth quarter of 2013 for employment termination costs, as well as facilities closing costs. The restructuring is expected to save approximately $4 million annually.