One of the challenges for developing drugs addressing neurological disorders is getting enough of the therapy into the brain. Exicure has proprietary technology that enhances the ability of a therapy to penetrate into cells and last longer compared to currently available types of drugs and Ipsen sees enough potential to begin a partnership on drugs for two rare neurological disorders with no therapeutic options.
According to deal terms announced Monday, Paris-based Ipsen will pay Exicure $20 million up front to begin the alliance. In exchange, Ipsen gains exclusive options to license the Huntington’s disease and Angelman syndrome therapies that Exicure is developing with its proprietary technology.
Exicure will fund the drug discovery and development work. If Ipsen exercises its option on one or more of the programs, it will take over responsibility for further development of the drugs, and commercialization if they are approved. The deal also calls for the Ipsen to pay its partner up to $1 billion in option exercise fees and milestone payments if it exercises option on both programs. Exicure will receive royalties from sales of the drugs if they reach the market.
Some of the drugs for neurological disorders are oligonucleotides, synthetic structures of nucleic acids that can modulate gene expression. But use of this type of drug is limited by the inability to deliver enough of the therapy into organs and tissues.
Chicago-based Exicure is developing a based on what it calls spherical nucleic acids (SNAs). These drugs are nanoscale constructs of densely packed synthetic nucleic acid sequences that are arranged in a three-dimensional structure that the company believes enhances a therapy’s cell penetration, distribution throughout the body, and its persistence in organs. Specifically, this structure enters cells through class A scavenger receptors, which are commonly found on the surface of cells throughout the body, the company said in regulatory filings. The company added that this mechanism of entering cells is different than the one used by other nucleic acid therapies that typically bind to receptors only found in liver cells. In a prepared statement, Exicure CEO David Giljohann said that treatment of both Huntington’s and Angelman requires the penetration of a drug deep into the brain.
“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,” he said.
Exicure’s technology has yielded programs in cancer, dermatology, and neuroscience. The most advanced cancer program is cavrotolimod, which is in Phase 1b/2 testing for merkel cell carcinoma and cutaneous squamous cell carcinoma. In neuroscience, the most advanced Exicure program is XCUR-FXN, a preclinical Friedreich’s ataxia drug candidate that the company expects to bring into human testing next year. A dermatology program for hair loss disorders is in preclinical development under a partnership with AbbVie.
For Ipsen, the Exicure deal is the third that the company has made in recent weeks. On July 15, the company agreed to pay IRLAB $28 million up front for global rights to mesdopetam, drug in mid-stage clinical development for patients with Parkinson’s disease. Milestone payments could add another $335 million to the deal. In the second deal, Ipsen last week agreed to pay BAKX Therapeutics $14.5 million up front for rights to a small preclinical molecule in development for potential applications treating leukemia, lymphoma, and solid tumors. Milestone payments could bring BAKX an additional $837.5 million.
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