E-Scape Bio, a biopharmaceutical company developing small-molecule drugs to treat patients with genetically-defined neurodegenerative diseases, has reached an agreement with AbbVie to obtain worldwide rights to selective S1P5 receptor agonists for the treatment of lysosomal storage disorders impacting the central nervous system (CNS).
These small molecule agonists are designed to reduce the synthesis of metabolites that, when not properly eliminated, become toxic at high levels in the brain and cause various CNS disorders. This pathophysiology is common to many lysosomal storage disorders such as Niemann Pick Type C Disease, GBA Parkinson’s disease, and Tay Sachs disease.
The addition of the S1P5 program for lysosomal storage disorders broadens E-Scape’s pipeline targeting genetic drivers of CNS disorders, including its Parkinson’s disease program targeting LRRK2, and its Alzheimer’s disease program targeting ApoE4.
“This agreement with AbbVie underscores our efforts to deliver solutions for genetically defined patient populations in CNS, and positions E-Scape to complete an investigational new drug (IND) application with the US FDA and initiate clinical development next year,” said Leon Chen, Ph.D., chief executive officer of E-Scape Bio.
“AbbVie has deep expertise in neuroscience and has assembled a robust preclinical data package for its S1P5 selective agonists that could enable advancement of the program into clinical development as we continue to build a company to treat unmet needs for patients with genetically-defined neurodegenerative diseases. We are looking forward to gaining AbbVie as an equity investor in E-Scape.”
Under the terms of the agreement, E-Scape obtains worldwide rights to all fields of use for selected small molecule agonists targeting S1P5 and assumes full responsibility for advancing the program going forward. E-Scape will immediately begin IND preparatory work, with an IND filing anticipated in 2019.
In exchange for the license, AbbVie receives an undisclosed upfront cash payment, potential future royalties and equity ownership in E-Scape, as well as the addition of an AbbVie-nominated observer to E-Scape’s board of directors.
Lysosomal storage diseases refer to dozens of rare inherited disorders characterized by the toxic accumulation of metabolites due to genetic mutations in enzymes or transporters necessary for the metabolism of these molecules. In some cases, enzyme replacement therapy and substrate reduction therapy have been effective therapeutic options for the peripheral forms of these diseases.
However, there remains a significant unmet need in CNS-related lysosomal storage disorders, where existing therapies are unable to cross the blood brain barrier.
“S1P5 agonists represent a unique, CNS targeted approach to substrate reduction therapy that could be broadly applicable to numerous lysosomal disorders,” said Sandy Williams, president emeritus, Gladstone Institutes and board member at E-Scape. “We are excited about the possibility of bringing a new therapeutic option to treat the neurodegenerative manifestations in these patients.”
(Source: E-Scape Bio)
Filed Under: Drug Discovery