Ipsen and Exicure Announce Over $1 Billion Collaboration to Develop SNA-Based Therapies for Rare Neurological Disorders

Compiled & Translated by | Tom Li

Recently, Ipsen, a global biopharmaceutical company focused on neuroscience, oncology, and rare diseases, has entered into a collaboration with Exicure.

Under the agreement, Ipsen will pay Exicure a $20 million upfront payment in exchange for an exclusive license to SNA-based therapies arising from the collaboration. Additionally, Exicure is eligible to receive up to $1 billion in option exercise fees and milestone payments in the future. Exicure will be responsible for the discovery and preclinical development of the new therapies.

Exicure primarily develops therapies for neurological, inflammatory, and genetic diseases, including novel treatments for Angelman syndrome and Huntington's disease (HD). Exicure’s proprietary SNA technology is the core of this collaboration agreement. Exicure believes that this architecture may offer advantages over conventional nucleic acid therapies and can treat diseases that are beyond the therapeutic capabilities of the latter.

Currently, Exicure’s XCUR-FXN is in preclinical development. This SNA-based lipid nanoparticle holds therapeutic potential for Friedreich's ataxia and advanced solid tumors. Research indicates that SNAs can enhance the cellular uptake, biodistribution, and organ persistence of oligonucleotides, which will facilitate the modulation of gene expression. This also signifies that SNAs possess the capability to overcome the longstanding challenge of delivering therapeutics to patient tissues that were previously inaccessible to conventional medications, including pathological lesions located deep within the brain.

Angelman syndrome and Huntington’s disease are severe neurodevelopmental and neurodegenerative disorders for which no targeted therapies have been approved to date. In the United States alone, there are 40,000 patients with Huntington’s disease, which is also the most common monogenic neurological disorder in developed countries. The prevalence of Angelman syndrome is also relatively high, affecting approximately 1 in 12,000 to 20,000 individuals in the general population.

Ipsen has been actively expanding its strategic presence in the field of neuroscience. Just a week ago, Ipsen and BAKX Therapeutics announced an exclusive global collaboration agreement for the research, development, manufacture, and commercialization of BKX-001 as a potential treatment for leukemia, lymphoma, and solid tumors. Previously, Ipsen also entered into an exclusive licensing agreement with IRLAB, securing global exclusive development and commercialization rights for mesdopetam, a D3 receptor antagonist, for the treatment of levodopa-induced dyskinesia (LID) in patients with Parkinson’s disease.

Reference:

1.Ipsen Goes All In on Neuro with $1 Billion+ Deal For Exicure's Oligonucleotides

2.Ipsen puts down $20M in $1B deal with Exicure for rare neuro disorders