One of the challenges in developing drugs that address neurological disorders is getting enough therapy in the brain. Exicure has proprietary technology that improves a therapy’s ability to penetrate cells and last longer compared to currently available drug types and Ipsen sees enough potential to start a camaraderie in drugs for two rare neurological disorders without therapeutic options.
Under the terms of the deal announced Monday, Paris-based Ipsen will pay Exicure $ 20 million in advance to start the alliance. In return, Ipsen gets exclusive options to license the Huntington’s disease and Angelman syndrome therapies that Exicure is developing with its proprietary technology.
Exicure will fund drug discovery and development work. If Ipsen exercises its option in one or more of the programs, it will assume responsibility for further drug development and commercialization if approved. The agreement also requires Ipsen to pay its partner up to $ 1 billion in option exercise fees and milestone payments if it exercises the option in both programs. Exicure will receive royalties on the sales of the drugs if they hit the market.
Some of the drugs for neurological disorders are oligonucleotides, synthetic nucleic acid structures that can modulate gene expression. But the use of this type of drug is limited by the inability to deliver a sufficient amount of the therapy to the organs and tissues.
Chicago-based Exicure is developing a technology 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, according to the company, improves a therapy’s cellular 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 documents. The company added that this mechanism of entry into cells is different from that used by other nucleic acid therapies that generally bind to receptors found only on liver cells. In a prepared statement, Exicure CEO David Giljohann said that the treatment of both Huntington and Angelman requires penetration of a drug deep into the brain.
“We believe that our platform technology with its deep penetration and persistent medicinal effect will allow Exicure and Ipsen to overcome the challenges of first-generation oligonucleotides and bring new drugs to patients who need them,” he said.
Exicure’s technology has spawned 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 drug for Friedreich’s ataxia that the company hopes to incorporate into human trials next year. A dermatology program for hair loss disorders is in preclinical development in collaboration with AbbVie.
For Ipsen, the deal with Exicure is the third the company has completed in recent weeks. On July 15, the company agreed pay IRLAB $ 28 million in advance for the global rights to mesdopetam, a drug in intermediate stage of clinical development for patients with Parkinson’s disease. The milestone payments could add another $ 335 million to the deal. In the second deal, Ipsen agreed last week pay BAKX Therapeutics $ 14.5 million in advance to obtain the rights to a preclinical small molecule under development for potential applications in the treatment of leukemia, lymphoma and solid tumors. The milestone payments could bring an additional $ 837.5 million to BAKX.
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