Preclinical development of ITH13001 as a second-generation oral treatment for multiple sclerosis

Rafael León Martínez


    Rafael León Martínez


    Instituto de Investigación Sanitaria - Hosrpital Universitario de la Princesa, Spain


    Multiple sclerosis (MS) is an immune-mediated inflammatory disease of the central nervous system that affects more than 2.5 million people worldwide. It causes inflammation and demyelination of nerve cells, leading to progressive neurodegeneration and irreversible disability. While the prevailing therapies show limited efficacy and are mainly focused on relapse MS subtypes, there is a lack of treatment options for progressive MS subtypes. The development of new neuroprotective and remyelinating treatments is encouraging for patients suffering from progressive MS.

    To develop ITH13001, a second-generation drug that combines a strong neuroprotective effect with the ability to reduce autoimmune attacks, an advance in treatments for MS.

    Problem to Solve
    MS therapies urgently need to reverse disease progression through neuroprotective and neuroreparative mechanisms. Alongside this, most of the existing first-line treatments are injectable, which causes a high number of side effects. Developing adequate oral therapies will add value to current therapeutic options for MS.

    ITH13001 has been specifically designed to induce Nrf2, which is a key regulator of antioxidant and anti-inflammatory cell response. Moreover, this innovative drug also acts as a potent antioxidant. On top of this, ITH13001 can have a strong anti-inflammatory effect, independent of Nrf2 induction, and its neurogenic effects result in remyelination. In diverse in vitro and ex vivo models, the combination of activities exerted by ITH13001 results in a potent neuroprotective effect.

    Level of Innovation
    ITH13001 is a second-generation Nrf2 inducer with advanced features compared to other first-line MS drugs. This innovative drug stands out due to its strong neurogenic effects and promising results through oral administration.