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Acadia Pharmaceuticals And Stoke Therapeutics Announce Collaboration To Pursue Multiple RNA-Based Treatments For Severe And Rare Genetic Neurodevelopmental Diseases

– Establishes co-development and co-commercialization agreement for Stoke’s SYNGAP1 preclinical program
– Acadia receives exclusive worldwide licenses for two additional preclinical programs: Rett syndrome (MECP2) and undisclosed neurodevelopmental target
– Combines Stoke’s TANGO research platform with Acadia’s expertise in neurology drug development and commercialization
– Stoke receives a $60 million upfront payment and potential milestone payments of up to $907 million and royalties on future sales

Acadia Pharmaceuticals Inc. and Stoke Therapeutics, Inc. announced that the companies have entered a collaboration to discover, develop and commercialize novel RNA-based medicines for the potential treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system (CNS). The collaboration includes SYNGAP1 syndrome, Rett syndrome (MECP2), and an undisclosed neurodevelopmental target of mutual interest.

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“Stoke’s RNA-based approach to upregulating healthy proteins offers very exciting new possibilities for the treatment of rare, neurodevelopmental diseases like Rett syndrome,” said Steve Davis, Chief Executive Officer of Acadia Pharmaceuticals. “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.”

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“Rett syndrome and SYNGAP1 syndrome are two severe, intractable diseases of the central nervous system and both are associated with developmental delays, motor function loss, autism, seizures and other comorbidities that impact quality of life for patients and their families,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “Acadia shares our deep commitment to improving outcomes for people living with neurodevelopmental disorders. We believe their late-stage development and commercialization capabilities, in addition to their neuroscience expertise, complement our discovery research efforts and clinical learnings from our work in Dravet syndrome. Together, we believe we have a significant opportunity to improve treatment options by delivering new disease-modifying medicines to people who need them.”

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Terms of Collaboration
Under the terms of the agreement, Stoke will receive an upfront payment of $60 million from Acadia and is eligible to receive up to $907 million in milestones as well as royalties on future sales.

For the SYNGAP1 program, the two companies will jointly share global research, development and commercialization responsibilities and share 50/50 in all worldwide costs and future profits. In addition, Stoke is eligible to receive potential development, regulatory, first commercial sales and sales milestones.


For the Rett syndrome (MECP2) and the undisclosed neurodevelopmental program, Stoke will lead research and pre-clinical development activities, while Acadia will lead clinical development and commercialization activities. Acadia will fully fund the research and pre-clinical development activities related to these two targets and Stoke is eligible to receive potential development, regulatory, first commercial sales and sales milestones as well as tiered royalty payments on worldwide sales starting in the mid-single digit range and escalating to the mid-teens based on revenue levels.

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