GlaxoSmithKline (NYSE:GSK) is moving to address rare, inherited disorders such as Tay-Sachs disease through a new drug partnership with Angiochem.
The companies will work together to discover and develop treatments for lysosomal storage diseases, or LSDs, rare diseases that arise from inherited gene mutations that cause enzyme deficiencies. Angiochem brings to the collaboration expertise in creating drugs that cross the blood-brain barrier. The Montreal, Canada company calls these compounds “EPiC-enzymes.” The agreement calls for Angiochem to create and develop an enzyme replacement therapy for a lysosomal storage disease. Britain-based GSK, which has its U.S. headquarters in Research Triangle Park, North Carolina, could take on further development and commercialization of a resulting drug candidate.
Angiochem receives $31.5 million up front under the agreement with GSK. Including that payment, the company stands to gain more than $300 million in research funding and other payments if GSK pursues additional LSD targets developed in the partnership. Angiochem would also be eligible to receive royalties on future sales of drugs resulting from the collaboration.
The collaboration could be expanded to address more lysosomal storage diseases. Lysosomal enzymes are needed to metabolize lipids or glycoproteins in cells. Besides Tay-Sachs, other LSDs include Fabry’s disease, Gaucher’s disease, Pompe’s disease and Hunter’s syndrome. Some of these diseases are evident in infancy or childhood, though they can appear later in life. LSDs occur in an estimated one in 7,000 births. There are no cures for LSDs, though they have been treated by enzyme replacement therapy to address patient symptoms. These treatments do not cross the blood-brain barrier and do not address neurological symptoms.
The blood-brain barrier is the separation of the brain blood from brain cells or cerebrospinal fluid. While that barrier protects the brain, it also blocks drugs from reaching brain cells. Angiochem says that because its experimental treatments can cross the blood-brain barrier, they can address neurological symptoms as well as other symptoms of LSDs. The company’s drug pipeline is focused on addressing a wide range of central nervous system diseases. Angiochem explains its technology in the following video:
