Ipsen signs $ 20 million deal with Exicure for rare neurological disorders


Ipsen is deepening his neuroscience research, with an upfront payment of $ 20 million to Exicure as part of a discovery deal for up to two preclinical assets. The deal comes with up to $ 1 billion in fees and focuses on two rare genetic diseases.

French Big Pharma’s bet on Chicago’s biotechnology comes a week after Ipsen signed an initial $ 14.5 million deal with BAKX Therapeutics for a solid tumor program.

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This week, Ipsen is focusing on the deadly neurological disorder of Huntington’s disease, which affects approximately 40,000 people in the United States, and Angelman’s syndrome, which includes speech disorders, epilepsy and others. impacts on behavioral and intellectual health. Ipsen’s neuroscience activity contributed 14.8% to Ipsen’s turnover in 2020.

Exicure’s technology targets oligonucleotides, or synthetic nucleic acid structures that modulate gene expression but are difficult to deliver to target organs and tissues, such as the brain. In stages, Exicure’s spherical nucleic acids (ANS), which aim to stimulate drug delivery to deep regions of the brain that were previously inaccessible, the companies said.

ANS provide chemical and biochemical properties to oligonucleotides and have been shown to enhance the cell-penetrating properties of oligonucleotides in preclinical models, they noted.

Under their agreement, Exicure will conduct the discovery and preclinical development of ANS-based therapies for Huntington and Angelman, while Ipsen has the exclusive option to license up to two candidates. If Ipsen exercises its option, the pharmaceutical company will lead further development and commercialization and pay Exicure up to $ 1 billion in exercise fees and milestone payments.

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This is not Exicure’s first licensed rodeo. Chicago-based biotechnology tied arms with Allergan, now owned by AbbVie, in November 2019 over hair loss disorders. Allergan paid Exicure $ 25 million upfront for its ANS technology for two programs.


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