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Angiochem teams with GSK to discover and develop treatments of lysosomal storage diseases
by Jeffrey Bouley  |  Email the author


MONTREAL, Quebec—Forging a deal potential worth in excess of $300, Angiochem has entered into a global collaboration with GlaxoSmithKline (GSK) to discover, develop and commercialize treatments for lysosomal storage diseases (LSDs).  
LSDs comprise a large group of rare inherited disorders, including Tay Sachs Disease, Fabry Disease, Gaucher's Disease, Pompe Disease and Hunter syndrome—they arise from enzyme deficiency resulting from inherited gene mutations. Specifically, the enzymes involved are required for metabolism of lipids or glycoproteins within cells; accumulation of these molecules within the cell lysosome underlies the pathology of the disease. Each LSD is associated with reduced or ablated expression of a different protein, and exhibits symptoms arising from different organs.  
The problem is getting drugs into the central nervous system (CNS) to restore enzyme function, and that is where Angiochem plays a key role in the collaboration. The team-up of it and GSK is intended to combine Angiochem's expertise in creating novel therapeutics that cross the blood-brain barrier (BBB) with the scientific, development and commercialization capability of GSK in rare diseases.   
Under the collaboration agreement, Angiochem will create new compounds (EPiC- enzymes) intended to penetrate the BBB and restore enzyme function in the CNS. Current enzyme replacement therapies are unable to restore enzyme function in the CNS and therefore fail to ameliorate neurological symptoms associated with LSDs. Administration of the resulting EPiC-enzyme drug candidates to patients is anticipated to result in brain penetration as well as systemic distribution, thereby addressing CNS as well as peripheral symptoms of LSDs.  
As part of the collaboration, Angiochem will initially create and develop an enzyme replacement therapy for a specified lysosomal storage disease while GSK will have the right to assume responsibility for development and commercialization of the resulting drug candidate. The agreement allows for expansion of the collaboration to include additional lysosomal storage disease targets.
Under the terms of the deal, Angiochem is eligible to receive more than $300 million, including as much as $31.5 million in upfront cash, research funding and other fees if GSK accesses the few LSD targets available to the collaboration. In addition, Angiochem is eligible to receive royalties on future sales of EPiC-enzymes that arise from the collaboration.  
"Our collaboration with GSK reflects our belief in the need to effectively address neurological symptoms of lysosomal storage diseases. We are pleased to collaborate with GSK, a committed leader in this rare disease area," said Dr. Jean-Paul Castaigne, president and CEO of Angiochem. "This collaboration will further validate the wide-ranging potential for our BBB platform across multiple therapeutic areas and classes of compounds while providing Angiochem with additional resources to advance our own internal pipeline including other EPiC-enzymes."

Code: E02271201



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