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Angiochem Forms Collaboration with GlaxoSmithKline to Discover and Develop Treatments of Lysosomal Storage Diseases


Angiochem Forms Collaboration with GlaxoSmithKline to Discover and Develop Treatments of Lysosomal Storage Diseases

Montreal, Canada, February 27, 2012 – Angiochem announced today a global  collaboration with GlaxoSmithKline (GSK) to discover, develop and commercialize treatments for lysosomal storage diseases (LSDs). The collaboration will 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 central nervous system (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 agreement, Angiochem is eligible to receive in excess of $300M, including up to $31.5M 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 Jean-Paul Castaigne, MD, 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.”

About Lysosomal storage diseases (LSDs)

Lysosomal storage diseases (LSDs) comprise a large group of rare inherited disorders, including Tay Sachs Disease, Fabry Disease, Gaucher’s Disease, Pompe Disease, Hunter syndrome and other mucopolysaccharidosis (MPS). LSDs 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. Many of these diseases are evident in infancy or childhood, however, some appear later in life. As a group, LSDs occur with incidences of about one in every seven thousand births. While there are no cures for LSDs, intravenous delivery of the deficient enzyme (enzyme replacement therapy, ERT) has been used to ameliorate symptoms in patients with some LSDs. Currently available ERTs do not cross the BBB, therefore, neurological symptoms, such as cognitive decline and behavioral changes, have not been addressed by ERT.


About Angiochem

Angiochem is a clinical-stage biotechnology company discovering and developing new breakthrough peptide drug conjugates that leverage the LRP-1 mediated pathway to cross the BBB to treat neurological diseases. These new EPiC compounds have the potential to address significant medical needs, many of which are insurmountable due to the fundamental physiological challenge posed by the BBB.


Angiochem is developing a focused product pipeline, including small molecules and biologics, for the potential treatment of a wide range of CNS diseases, including brain cancer, neurodegenerative and lysosomal storage diseases, pain, and others. Founded in 2003, Angiochem maintains headquarters in Montreal, Canada. For additional information about the Company, please visit