One of the challenges in developing drugs to treat neurological diseases is to get enough treatment into the brain. Exicure has a proprietary technology that enhances the ability of the therapy to penetrate into cells and lasts longer than the types of drugs currently available, and Ipsen believes that it has enough potential to start partnership Two drugs for rare neurological diseases with no treatment options.
According to the terms of the deal announced on Monday, Paris-based Ipsen will pay Exicure US$20 million in advance to start the alliance. In exchange, Ipsen obtained the exclusive right to license Exicure to use its proprietary technology to develop treatments for Huntington’s disease and Angelman syndrome.
Exicure will fund drug discovery and development work. If Ipsen exercises options for one or more of these projects, it will be responsible for the further development of the drug and commercialization after approval. The transaction also requires Ipsen to pay its partners up to $1 billion in option exercise fees and milestone payments if it exercises options in these two projects. If the drug is on the market, Exicure will receive royalties from the sale of the drug.
Some drugs for the treatment of neurological diseases are oligonucleotides, which are nucleic acid synthesis structures that can regulate gene expression. But the use of this drug is limited by the inability to deliver sufficient treatment to organs and tissues.
Chicago-based Exicure is developing a product based on its so-called spherical nucleic acid (SNA). These drugs are densely arranged nano-scale structures of synthetic nucleic acid sequences, which the company believes can enhance the cell penetration, systemic distribution and persistence of treatments in organs. The company stated in the regulatory document that, specifically, this structure enters cells through class A scavenger receptors, which are commonly found on the surface of cells throughout the body. The company added that this cell entry mechanism is different from that used by other nucleic acid therapies, which usually bind to receptors found only in liver cells. In a prepared statement, Exicure CEO David Giljohann stated that both Huntington’s disease and Angelman’s treatment require drugs to be deep into the brain.
“We believe that our platform technology has deep penetration and durability, which will enable Exicure and Ipsen to overcome the challenges of first-generation oligonucleotides and bring new drugs to patients in need,” he said.
Exicure’s technology has produced projects in the fields of cancer, dermatology, and neuroscience. The most advanced cancer project is Kavromod, which is in phase 1b/2 testing for Merkel cell carcinoma and skin squamous cell carcinoma. In the field of neuroscience, the most advanced Exicure project is XCUR-FXN, a preclinical candidate for Friedreich’s ataxia, which the company expects to introduce into human trials next year. In cooperation with AbbVie, the dermatology project for hair loss disorders is in the pre-clinical development stage.
For Ipsen, the Exicure deal is the company’s third deal in recent weeks. On July 15, the company agreed $28 million paid to IRLAB The global rights of metopetane are used in the mid-term clinical development of Parkinson’s disease patients. The milestone payment may add another $335 million to this transaction.In the second transaction, Ipsen agreed last week Payment of US$14.5 million to BAKX Therapeutics Obtained the rights of preclinical small molecules under development in advance for potential applications in the treatment of leukemia, lymphoma and solid tumors. Milestone payments may bring BAKX US$837.5 million in additional revenue.
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