The blood-brain barrier protects the functioning of that essential organ, but it’s also a hurdle to delivering neurological drugs.
Italy’s Chiesi Group has agreed to pay Guilford, CT-based Bioasis Technologies $3 million up front to use the preclinical firm’s technology to facilitate the delivery of enzymes across the blood-brain barrier to treat neurological symptoms associated with four lysosomal storage disorders (LSDs). Such diseases are caused by mutations in the genetic instructions for important enzymes that normally help our cells clear out waste. Without those enzymes, toxic materials build up in the body’s cells over time.
Under the agreement Chiesi is responsible for R&D and commercialization costs associated with the programs. Bioasis is eligible for up to $138 more in milestone payments, plus royalties on net sales of any product Chiesi commercializes using its technology. In exchange Chiesi gets the rights to what Bioasis calls its xB3 platform, the technology the company is developing for the delivery of therapeutics across the blood-brain barrier and the treatment of central nervous system disorders.
Bioasis says its xB3 technology uses a process called receptor-mediated transcytosis and a human transport protein, melanotransferrin, to move molecules across the blood-brain-barrier. The company says that in preclinical studies, this method was able to ferry molecules of varying sizes and types into the brain.
Enzyme replacement therapy is used to treat some LSDs, but neurological complications of the disorders remain largely unaffected even when other affected parts of the body respond to the treatment, according to Bioasis CEO Deborah Rathjen.
“The unique delivery method of [Bioasis’s] 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,” said Giacomo Chiesi, who heads the company’s Boston-based rare disease unit Chiesi, which launched in February.
The BioMarin Pharmaceuticals (NASDAQ: BMRN) drug erliponase alfa (Brineura), for example, which was approved in 2017 to treat a group of degenerative neurometabolic disorders caused by an enzyme deficiency, must be administered directly into the brain through a stent.
Chiesi isn’t disclosing the specific LSDs it is targeting. Nearly 50 exist, according to the National Organization for Rare Disorders. For most LSDs no or few treatment options exist.
The companies described their new partnership as a strategic alliance focused on rare diseases.