Ipsen and Exicure enter into exclusive collaboration targeting rare neurodegenerative disorders

• Ipsen obtains exclusive options to Spherical Nucleic Acids (SNAsTM) currently under discovery evaluation for Huntington’s disease and Angelman syndrome

• Exicure will be responsible for discovery and certain pre-clinical development activities. In the event Ipsen exercises its option to the two programs, Ipsen will be responsible for further development and worldwide commercialization

• Exicure will receive a $20m upfront payment and is eligible to receive up to $1B in option exercise fees and milestone payments should Ipsen opt into both programs, as well as tiered royalties

Paris (France), and Chicago, IL, Cambridge, MA (USA) Monday 2 August 2021 – Ipsen (Euronext: IPN; ADR: IPSEY) and Exicure Inc. (NASDAQ: XCUR) have signed an exclusive collaboration agreement to research, develop, and commercialize novel Spherical Nucleic Acids (SNAs) as potential investigational treatments for Huntington’s disease and Angelman syndrome.

Oligonucleotides are synthetic structures of nucleic acids that can be used to modulate gene expression via a range of processes, including gene activation, inhibition, and splice-modulation. These molecules have demonstrated potential in many different therapeutic areas.1 Achieving efficient oligonucleotide delivery to target organs and tissues, including the brain, remains a major limitation to their use.1,2 Exicure’s SNAs provide distinct chemical and biochemical properties to oligonucleotides. In preclinical models, SNAs have been shown to enhance the cell penetration, biodistribution and organ persistence properties of oligonucleotides,3,4 which may potentially enhance drug delivery to previously inaccessible target tissues, including deep brain regions.5,6

Philippe Lopes-Fernandes, Chief Business Officer at Ipsen, said “Neuroscience is deeply rooted within Ipsen as a key strategic driver for our business. We are pleased to partner with Exicure to progress development of investigational treatment options for Huntington’s disease and Angelman syndrome, two areas of significant unmet need. This collaboration marks an important step in maximizing the potential of this novel technology, bringing together the expertise of Exicure and the robust heritage of Ipsen in neuroscience. With this new collaboration we will deepen our commitment to people living with neurological conditions around the world.”

“We are thrilled to partner with Ipsen, a leading global company with significant expertise and commitment to developing treatments for patients with rare neurological diseases,” said David Giljohann, Ph.D., Chief Executive Officer, Exicure, Inc. “In collaboration with Ipsen, we have the opportunity to apply our technology to Huntington’s disease and Angelman syndrome, both indications requiring deep brain penetration and technological advances to reach previously hard-to-drug targets. We believe our platform technology with its deep penetration and persistence of medicinal effect will allow Exicure and Ipsen to overcome challenges from first-generation oligonucleotides and bring new medicines to patients in need.”

Under the agreement, Ipsen will receive exclusive options to license SNA-based therapeutics arising from two collaboration programs for Huntington’s disease and Angelman syndrome. Ipsen will pay Exicure a cash upfront payment of $20m upon closing and Exicure will be responsible for discovery and certain pre-clinical development activities. In the event Ipsen exercises its option, Ipsen will be responsible for further development and commercialization of the licensed products. Exicure will receive a $20m upfront payment and is eligible to receive up to $1B in option exercise fees and milestone payments should Ipsen opt into both programs, as well as tiered royalties.

1. Roberts TC, Langer R, Wood MJA. Advances in oligonucleotide drug delivery. Nature Reviews Drug Discovery 2020;19:673–694. Available here: https://www.nature.com/articles/s41573-020-0075-7
2. Crawford L, Rosch J, Putnam D. Concepts, technologies, and practices for drug delivery past the blood–brain barrier to the central nervous system. Journal of Controlled Release. 2016;240: 251-266. Available here: https://www.sciencedirect.com/science/article/pii/S0168365915302923?via%3Dihub#bb0020
3. https://investors.exicuretx.com/news/news-details/2019/Exicure-Announces-Preclinical-Data-Supporting-Development-of-SNA-Technology-in-the-Central-Nervous-System/default.aspx
4. https://s1.q4cdn.com/907903764/files/doc_news/archive/b984683d-76f4-4759-9add-d2c65150ebb6.pdf
5. Rosi NL, Giljohann DA, Thaxton S, et al. Oligonucleotide-modified gold nanoparticles for intracellular gene regulation. Science 2006;312(5776):1027–1030. Available here: https://science.sciencemag.org/content/312/5776/1027?ijkey=8fbfd37b2763498a2658cc0dfaa5dc15df0e81a0&keytype2=tf_ipsecsha
6. https://www.news-medical.net/life-sciences/What-is-an-Oligonucleotide.aspx

Associated Documents

Ipsen-Exicure-collaboration-2-August-2021.pdf

Attachment

• Ipsen-Exicure-collaboration-2-August-2021