Ipsen partners with Exicure on SNA therapies for neurodegenerative disorders

Ipsen entered into an agreement with Exicure potentially worth more than $1 billion to develop spherical nucleic acids (SNAs) as treatments for Huntington's disease and Angelman syndrome, the companies announced Monday. Under the deal, Ipsen will make an upfront cash payment of $20 million to Exicure, gaining exclusive options to license therapeutics from the two collaboration programmes. Shares in Exicure were up 34% on the news.

As part of the agreement, Exicure will be responsible for discovery and certain preclinical development activities. It stands to receive up to $1 billion in option exercise fees and milestone payments should Ipsen opt into both programmes, plus would be eligible for tiered royalties as well. In the event that Ipsen exercises its option, then the company will take over responsibility for further development and commercialisation.

Enhanced cell penetration 

Philippe Lopes-Fernandes, chief business officer at Ipsen, stated "this collaboration marks an important step in maximising the potential of this novel technology," which the companies hope will help overcome challenges from first-generation oligonucleotides. According to the drugmakers, preclinical studies have shown that SNAs can enhance the cell penetration, biodistribution and organ persistence properties of oligonucleotides, which they noted may enhance drug delivery to previously inaccessible target tissues, including deep brain regions.

The deal with Exicure comes shortly after Ipsen announced an agreement with BAKX Therapeutics potentially worth over $800 million to develop BKX-001, the latter's oral small-molecule activator of the BAX apoptosis regulator, for leukaemia, lymphoma and solid tumours.

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