LSDs comprise a large group of rare inheriteddisorders, including Tay Sachs Disease, Fabry Disease, Gaucher's Disease, PompeDisease and Hunter syndrome—they arise from enzyme deficiency resulting frominherited gene mutations. Specifically, the enzymes involved are required formetabolism of lipids or glycoproteins within cells; accumulation of thesemolecules within the cell lysosome underlies the pathology of the disease. EachLSD 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 centralnervous system (CNS) to restore enzyme function, and that is where Angiochemplays a key role in the collaboration. The team-up of it and GSK is intended tocombine Angiochem's expertise in creating novel therapeutics that cross theblood-brain barrier (BBB) with the scientific, development andcommercialization capability of GSK in rare diseases.
Under the collaboration agreement, Angiochem willcreate new compounds (EPiC-enzymes) intended to penetrate the BBB and restoreenzyme function in the CNS. Current enzyme replacement therapies are unable torestore enzyme function in the CNS and therefore fail to ameliorateneurological symptoms associated with LSDs. Administration of the resultingEPiC-enzyme drug candidates to patients is anticipated to result in brainpenetration as well as systemic distribution, thereby addressing CNS as well asperipheral symptoms of LSDs.
As part of the collaboration, Angiochem willinitially create and develop an enzyme replacement therapy for a specifiedlysosomal storage disease while GSK will have the right to assumeresponsibility for development and commercialization of the resulting drug candidate.The agreement allows for expansion of the collaboration to include additionallysosomal storage disease targets.
Under the terms of the deal, Angiochem is eligibleto receive more than $300 million, including as much as $31.5 million inupfront cash, research funding and other fees if GSK accesses the few LSDtargets available to the collaboration. In addition, Angiochem is eligible toreceive royalties on future sales of EPiC-enzymes that arise from thecollaboration.
"Our collaboration with GSK reflects our belief inthe need to effectively address neurological symptoms of lysosomal storagediseases. We are pleased to collaborate with GSK, a committed leader in thisrare disease area," said Dr. Jean-Paul Castaigne, president and CEO ofAngiochem. "This collaboration will further validate the wide-ranging potentialfor our BBB platform across multiple therapeutic areas and classes of compoundswhile providing Angiochem with additional resources to advance our own internalpipeline including other EPiC-enzymes."
