Acadia to Stoke up pipeline with $60M central nervous system research pact

Acadia to Stoke up pipeline with $60M central nervous system research pact

Acadia Pharmaceuticals is looking to Stoke Therapeutics to breathe some air into its pipeline, with a $60 million upfront research pact to develop drugs for rare genetic diseases of the central nervous system.

The companies will work together to develop new treatments for SYNGAP1 syndrome, Rett syndrome and an undisclosed neurodevelopmental target. The upfront fee is accompanied by up to $907 million in milestones and also royalties down the line for Stoke should any of the candidates be successful.

Acadia announced the deal at the ongoing virtual J.P. Morgan Healthcare Conference.

The SYNGAP1 program will be jointly developed by the two companies, with worldwide research, development and commercialization responsibilities and profits shared 50/50.

For Rett and the undisclosed program, Stoke will lead research and preclinical development activities with funding from Acadia, which will lead clinical development and commercialization.

SYNGAP1 is a rare neurological disease that occurs due to a mutation of the SYNGAP 1 gene, causing severe intellectual disability. Rett is another rare neurological disorder primarily occurring in females and is caused by mutations on the X chromosome of the MECP2 gene. The disorder disrupts brain function including fine motor skills and gross motor skills, with symptoms presenting in children between six months and 18 months.

Stoke is focused on addressing the underlying causes of severe diseases through the upregulation of protein expression using RNA-based medicines. The biotech has developed the TANGO approach, or targeted augmentation of nuclear gene output, which develops antisense oligonucleotides to selectively restore protein levels. The company’s first candidate is STK-001, which is in trials for the severe epilepsy disorder Dravet syndrome.

Acadia, meanwhile, has Nuplazid approved for Parkinson’s disease psychosis. The company also has therapies in development for Alzheimer’s disease, Rett syndrome, pain and more.

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