Acadia Pharmaceuticals has partnered with Stoke Therapeutics to discover, develop and commercialise novel RNA-based medicines for severe and rare genetic neurodevelopmental diseases.
Under the terms of the agreement, Acadia will make an upfront payment of $60m to Stoke.
Stoke will also be eligible to receive payments of up to $907m based on certain milestones as well as royalties on future sales.
Under the collaboration, Stoke’s TANGO research platform will be combined Acadia’s expertise in neurology drug development and commercialisation.
The collaboration will cover SYNGAP1 syndrome, Rett syndrome (MECP2), and an undisclosed neurodevelopmental target.
Acadia and Stoke will jointly share global research, development and commercialisation responsibilities for the SYNGAP1 programme.
Characterised by moderate to severe intellectual disability, SYNGAP1 syndrome is a rare neurological disorder that is observed in early childhood. The severity and onset of symptoms are said to differ from patient to patient.
Stoke will be responsible for research and pre-clinical development activities for the Rett syndrome (MECP2) and the undisclosed neurodevelopmental programme, while Acadia will lead clinical development and commercialisation activities.
Research and pre-clinical development activities related to the two targets will be fully funded by Acadia.
Acadia Pharmaceuticals CEO Steve Davis said: “Stoke’s RNA-based approach to upregulating healthy proteins offers very exciting new possibilities for the treatment of rare, neurodevelopmental diseases like Rett syndrome.
“Combining Stoke’s capabilities with Acadia’s extensive expertise in neuroscience drug development and commercialization enables us to push harder and faster in exploring some of the new frontiers in rare central nervous system disorders.
“We are excited to have the opportunity to further build our Rett syndrome franchise and pursue treatments in SYNGAP1 syndrome and other neurodevelopmental disorders.”
Mainly witnessed in females, Rett syndrome is a rare, debilitating neurological disorder that occurs after apparently normal development for the first six months of life.
The syndrome leads to problems in brain function that are responsible for cognitive, sensory, emotional, motor and autonomic function.
