The collaboration covers several neurodevelopmental targets, including Rett syndrome, SYNGAP1 syndrome, and one other disease yet to be disclosed.
Acadia Pharmaceuticals has partnered with Stoke Therapeutics for the development and commercialization projects on RNA-based therapies to treat severe and rare genetic neurodevelopmental diseases of the central nervous system.
The collaboration covers several neurodevelopmental targets, including Rett syndrome (MECP2), SYNGAP1 syndrome, and one other disease yet to be disclosed. SYNGAP1 and Rett are two severe and intractable CNS diseases that are linked to loss of motor function, developmental delays, seizures, autism, and other conditions that lead to poor quality of life for both the patient and their families.
Under the terms of the deal, the two firms will share global research, development, and commercialization activities for the SYNGAP1 program and later divide costs and future profits 50-50. Stoke also stands to obtain payment from future sales, regulatory, and development milestones.
The research will take advantage of Stoke’s proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) platform, which works to restore missing proteins by increasing the protein output from healthy genes.
For the Rett syndrome program and the other undisclosed disease, Stoke will take the lead in pre-clinical development and research, while Acadia will take charge of clinical development and commercialization efforts. Funding for both research activities will come from Acadia. Stoke also expects to gain payments from milestones achieved and tiered royalties.
Stoke will receive $60 million upfront from Acadia and is eligible to receive as much as $907 million in total royalties and milestones achieved down the line.
“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,” commented Steve Davis, chief executive officer of Acadia, in a statement.
SYNGAP1 syndrome is a rare neurological disease that surfaces in early childhood and manifests moderate to severe intellectual disability. The brain needs normal levels of the SynGap protein to function properly, and mutations in the SYNGAP1 gene can lead to several conditions, including epileptic encephalopathies, autism spectrum disorder, generalized epilepsy, and intellectual disability. SYNGAP1 syndrome accounts for 1% to 2% of cases associated with intellectual disability.
Rett syndrome is another rare neurological disorder that mostly happens during the first six months of life in females. It is usually misdiagnosed as cerebral palsy, autism, or non-specific developmental delay and is caused by mutations in the MECP2 gene. The disease occurs in one of 10,000 to 15,000 female births worldwide.
Currently, there are no FDA-approved treatments for both diseases.
