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Exicure Stock Soars on Ipsen Huntington’s Disease Treatment Deal

Exicure will receive $20 million upfront and be eligible to receive as much as $1 billion in option exercise fees and milestone payments.

Exicure  (XCUR) - Get Exicure, Inc. Report took off Monday after the biotech company said it was collaborating with French drugmaker Ipsen on treatments for Huntington’s disease and Angelman syndrome.

Shares of the Chicago company surged 40% to $1.88 at last check.

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Under the agreement, Exicure will receive a $20 million upfront payment and be eligible to receive as much as $1 billion in option exercise fees and milestone payments.

The companies will develop and commercialize novel spherical nucleic acids 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, the company said, including gene activation, inhibition, and splice-modulation. 

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The molecules have demonstrated potential in many different therapeutic areas. In preclinical models, SNAs have been shown to enhance the cell penetration, biodistribution and organ persistence properties of oligonucleotides, which may potentially enhance drug delivery to previously inaccessible target tissues, including deep brain regions.

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Ipsen will receive exclusive options to license SNA-based therapeutics arising from the two collaboration programs.

In the event Ipsen exercises its option, Ipsen will be responsible for further development and commercialization of the licensed products.

"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," Exicure CEO David Giljohann said in a statement.

Huntington’s disease is a progressive, fatal neurodegenerative disorder and the most common monogenic neurological disorder in the developed world, affecting about 40,000 people in the US. 

Angelman syndrome is a neurodevelopmental disorder that is marked by severe intellectual deficit, speech impairment, epilepsy, ataxic movements and behavioral abnormalities.