Ipsen dives into $1.8B Skyhawk pact to fire up RNA modulator R&D

Ipsen has swooped on a $1.8 billion R&D opportunity, joining with Skyhawk Therapeutics for the chance to option two candidates that could benefit from its “neuroscience expertise in movement disorders.”

Skyhawk is, based on financing, deals and pipeline progress, at the forefront of efforts to expand the universe of druggable proteins by using small molecules to target RNA. Faced with undruggable targets, Skyhawk and peers such as Arrakis Therapeutics and Remix Therapeutics are betting they can alter levels of disease-driving proteins by hitting RNA upstream. Skyhawk has landed deals with Genentech, Merck & Co., Vertex and a who’s who of other leading drug developers on the strength of its platform. 

Ipsen is the latest addition to Skyhawk’s star-studded list of R&D partners. The French drugmaker, which ended last year with 1.9 billion euros ($2 billion) in financial firepower for external innovation, is paying an upfront fee of undisclosed size for an option to pick up the global rights to two candidates. All told, the deal is worth up to $1.8 billion in upfront and milestone fees, plus potential tiered royalties. 

Skyhawk will handle the projects up to the nomination of development candidates, at which point Ipsen will take over. Public details of what Ipsen will have the chance to develop are limited. Ipsen said it will use its “neuroscience expertise in movement disorders” to develop and commercialize the molecules. The statement also refers to rare neurological diseases. Everything else remains a secret.

Neuroscience is a focus of Skyhawk’s internal pipeline, which is led by a phase 1 splicing modulator that is designed to treat Huntington's disease by reducing the production of mutated HTT. The biotech began the multiple ascending dose portion of its phase 1 study earlier this year. Skyhawk also has preclinical programs in frontotemporal dementia and the movement disorder spinocerebellar ataxia type 3.

Ipsen sells neuromuscular blocking agent Dysport and is testing a longer-acting neurotoxin in people with upper limb spasticity. The company expects to report phase 2 data on the new neurotoxin in 2025.