Ipsen puts down $20M in $1B deal with Exicure for rare neuro disorders

Ipsen is digging deeper into neuroscience, with a $20 million upfront payment to Exicure in a discovery deal for up to two preclinical assets. The agreement is back-loaded with up to $1 billion in fees and focuses on two rare genetic disorders.

The French Big Pharma's bet on the Chicago biotech comes a week after Ipsen penned a $14.5 million upfront deal with BAKX Therapeutics for a solid tumor program.

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This week, Ipsen is focused on the fatal neuro disorder Huntington's disease, which affects about 40,000 people in the U.S, and Angelman syndrome, which includes speech impairment, epilepsy and other impacts on behavioral and intellectual health. Ipsen's neuroscience business contributed 14.8% to Ipsen's revenue in 2020. 

Exicure's technology takes aim at oligonucleotides, or synthetic structures of nucleic acids which modulate gene expression but are challenging to deliver to target organs and tissues, like the brain. In steps Exicure's spherical nucleic acids (SNAs), which aim to boost drug delivery to deep brain regions that have been previously inaccessible, the companies said.

The SNAs provide chemical and biochemical properties to oligonucleotides and have shown they boost the cell penetration properties of oligonucleotides in preclinical models, they noted. 

According to their agreement, Exicure will carry out discovery and preclinical development of SNA-based therapies for Huntington's and Angelman, while Ipsen has the exclusive option to license up to two candidates. If Ipsen exercises its option, the pharma will lead further development and commercialization and pay Exicure up to $1 billion in exercise fees and milestone payments. 

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This isn't Exicure's first licensing rodeo. The Chicago biotech linked arms with Allergan, now owned by AbbVie, in November 2019 on hair loss disorders. Allergan paid Exicure $25 million upfront for its SNA technology for two programs.