Bioasis and Chiesi Enter Lysosomal Storage Disorder Collaboration Worth up to $141 Million

Rare Daily Staff

Bioasis entered into a rare diseases strategic alliance with Chiesi Global Rare Diseases that gives the Italian drug company an exclusive license for Bioasis’ platform technology to develop enzyme therapies for four undisclosed lysosomal storage disorders that can cross the blood-brain barrier.

The deal provides Bioasis with a $3 million upfront payment and up to $138 million in milestones, along with royalties on the net sales of licensed products, pending approval. Chiesi will be responsible for all costs associated with research, development, and commercialization of the four undisclosed programs.

Lysosomal storage disorders are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies. The build-up of toxic materials can affect different parts of the body, including the skeleton, brain, skin, heart and central nervous system. There are nearly 50 known lysosomal storage disorders. While there has been progress in clinical research to develop treatments in recent years, there are either limited or no treatment options available for most of these diseases.

Bioasis is developing the xB³ platform, a proprietary technology for the delivery of therapeutics across the blood-brain barrier for the treatment of central nervous system disorders. The companies believe the delivery platform has the potential to solve one of the major challenges of using enzyme replacement therapy to treat lysosomal storage disorders that involve the central nervous system.

“With this collaboration, we are taking our commitment to bringing innovative therapies to rare disease patients, including those living with LSDs, to an entirely new level,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases, a business unit of Chiesi Group. “The unique delivery method of their xB³ platform has the potential to overcome a significant challenge in the treatment of many neurological disorders, which is the ability to cross the blood brain barrier.”

Photo: Giacomo Chiesi, head of Chiesi Global Rare Diseases