Exicure’s Neurodegenerative Disease SNA Drugs Backed By Ipsen

French pharmaceutical company Ipsen has been through a whirlwind of licensing deals in the span of a few weeks. The latest acquisitions, which line up neatly with Ibsen’s neuroscience and rare disease dealings, are two drug programs from U.S. biotech company Exicure meant to combat the effects of neurodegenerative disorders Angelman syndrome and Huntington’s disease. In a deal that has billion-dollar potential for Exicure through exercise fees, milestone payments, and tiered royalties, Ipsen is shelling out $20 million for rights to the candidates, which as of now remain in the discovery stage.

Through this arrangement, Ipsen has secured the rights to spherical nucleic acid drugs that mitigate the toxicity present in alternative delivery mechanisms. SNA drugs can be utilized to modify gene expression through the transfer of oligonucleotides into cells. Exicure believes that a honed delivery model could open up body location possibilities for gene-modifying therapies. The deep brain regions that bear the brunt of many central nervous system diseases might be reachable.

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Exicure’s investigator-led study of SNA for brain cancer glioblastoma demonstrated that the drug is capable of accumulating in target cells, engaging with its target gene, and penetrating the blood-brain barrier. Its chief SNA candidate is tailored for degenerative neuromuscular disease Friedreich’s ataxia, and will begin clinical testing next year.

Ipsen’s deal entails financing discovery and partial preclinical development of SNA candidates for Angelman syndrome and Huntington’s; the company will have an option to buy in in the interest of obtaining exclusive commercial and development rights.