January 15, 2022 Perspective

Stoke Therapeutics Collaboration Drives Discovery & Development for Rare Disease Treatments

At Acadia, innovation means striving to bring potential new treatment options to patients living with diseases that can take so much away. Our recently announced collaboration with Stoke Therapeutics will enable that and more.

Exciting New Possibilities

Stoke Therapeutics is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines. Stoke’s proprietary research platform, TANGO (Targeted Augmentation of Nuclear Gene Output), offers exciting new possibilities for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system (CNS).

Our collaboration offers unique opportunities to discover, develop and commercialize novel-RNA-based medicines for potential treatment of Rett syndrome, a debilitating neurological disorder that occurs primarily in females following apparently normal development for the first six months of life. Acadia currently holds an exclusive license to develop and commercialize trofinetide, an investigational drug, in North America as a potential treatment for Rett Syndrome. The opportunity to further build our Rett syndrome franchise is energizing.

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.

Steve Davis, Chief Executive Officer at Acadia

Increased Support for the Rare Disease Community

The Stoke collaboration also includes options for SYNGAP1 syndrome and an undisclosed neurodevelopmental target. Like Rett, SYNGAP1 syndrome is a rare severe, intractable disease of the central nervous system associated with developmental delays, motor function loss and other comorbidities that impact quality of life for patients and their families. We are excited by the significant opportunity ahead to innovate and improve treatment options for rare diseases by delivering new disease-modifying medicines to people who need them.