In $900M+ deal, rare disease biotechs team up on RNA therapies for CNS developmental disorders
Two biotechs that have built their reputations in rare disease research signed a new collaboration Monday morning aiming to develop three new RNA-based therapies. And the deal could be worth nearly $1 billion in biobucks if every milestone is met.
Acadia Pharmaceuticals and Stoke Therapeutics will team up on the treatments in rare genetic neurodevelopmental diseases of the CNS, the companies announced. Up first will be the recently discovered SYNGAP1 syndrome, followed by Rett syndrome (MECP2) and an undisclosed target the biotechs say is of mutual interest.
“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,” Acadia CEO Steve Davis said in a statement.
Stoke will net a $60 million upfront payment from Acadia to kick things off and is eligible to receive up to $907 million in milestones.
The companies will split R&D costs and profits evenly for the SYNGAP1 candidate, according to the press release. Stoke will take the lead in preclinical development for the other two programs — efforts that will be “fully funded” by Acadia — after which Acadia will head up clinical studies and commercialization.
SYNGAP1 syndrome was first identified in 2009, the companies say, and can be characterized by developmental delay or intellectual disability, generalized epilepsy and autism spectrum disorder, among other things, presenting in early childhood. The condition is caused by mutations in the SYNGAP1 gene and is said to account for “1% to 2%” of all intellectual disability cases.
Rett syndrome, meanwhile, typically occurs in young girls due to a mutation of a gene on the X chromosome. It’s a condition in which Acadia is working on another program, having revealed positive topline data in a Phase III study last month showing the candidate, known as trofinetide, beat placebo on two co-primary endpoints.
Though many collaborations in the space are signed between a Big Pharma company and a smaller biotech, Monday’s deal comes from two biotechs with histories of developing rare disease and CNS therapies. Acadia’s Nuplazid was the first drug approved to treat hallucinations and psychosis related to Parkinson’s disease, while Stoke revealed the first clinical data for its lead Dravet syndrome program last month.
Acadia has run into some trouble more recently in trying to expand Nuplazid’s indications, however, as the FDA in April issued a CRL for dementia-related psychosis. The biotech said a few weeks ago it’s planning to resubmit the sNDA with a focus on Alzheimer’s induced psychosis sometime this quarter.