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GEN News Highlights : Feb 27, 2012

GSK, Angiochem to Work on CNS-Penetrating Drugs for Lysosomal Storage Diseases

Collaboration will exploit Angiochem’s LRP-1 receptor-targeting peptide technology for crossing BBB.

GlaxoSmithKline (GSK) and Angiochem inked a global collaboration focused on the development and commercialization of treatments for lysosomal storage diseases (LSDs). The partnership will combine GSK’s expertise in rare diseases with Angiochem’s capabilities in the development of compounds that can cross the blood-brain barrier (BBB). The ultimate aim will be development of engineered peptide compound (EPiC) drugs, or EPiC enzymes, which can restore enzyme function in the central nervous system, a feature not possible with current enzyme replacement therapies (ERTs). This lack of penetration into the CNS means existing ERTs can’t address neurological symptoms associated with LSDs, the firms claim.

The partnership will initially focus on the creation and development of an ERT for a specified LSD. GSK will be able to assume all development and commercialization rights to the product. Other LSD targets may subsequently be included in the scope of the agreement. Angiochem could receive over $300 million from its partner, including up to $31.5 million in up-front cash, research funding, and other fees, if GSK accesses the LSD targets available to the partnership.

“This collaboration will further validate the wide-ranging potential or our BBB platform across multiple therapeutic areas and classes of compounds, while providing Angiochem with additional resources to advance our own internal piopeline including other EPiC-enzymes,” states Jean-Paul Castaigne, M.D., president and CEO at Angiochem.

Montreal-based Angiochem is leveraging its EPiC technology to develop drugs for treating brain diseases and other disorders with CNS-relevant components. The peptide-based EPiC candidates are designed to bind to the BBB’s lipoprotein receptor-related protein (LRP-1) receptor, and cross into the CNS via LRP-1 receptor mediated transcytosis, to deliver their drug cargos. The firm has developed a library of more than 100 Angiopep peptide structures, ranging in size from eight to 34 amino acids, which form the basis of its small molecule and biologic EPiC drug candidates.

Lead clinical-stage anticancer candidate GRN1005 is currently undergoing development in partnership with Geron. In December 2011 the latter initiated separate Phase II studies evaluating GRN1005 in patients with brain metastases arising from either non-small cell lung cancer or breast cancer. GRN1005 comprises an LRP-1 receptor-targeting peptide-drug conjugate (LRP-directed PDC) comprising paclitaxel linked to the LRP receptor-targeting peptide Angiopep-2.

Geron and Angiochem are also collaborating on an EPiC telomerase inhibitor, which is still in the discovery phase. Angiochem’s in-house pipeline comprises a preclinical program of EPiC candidates for the treatment of cancer and pain, in addition to EPiC monoclonal antibodies, and an in-house LSD candidate.