In contrast to recombinant enzymes, which constitute the current standard of care, odiparcil can be given orally and is more widely distributed in the body. For instance distribution and exposure data generated in rodents show that odiparcil reaches the heart, joints, cartilage and cornea. Many MPS VI patients suffer from heart problems, stiff joints, spinal cord compression and visual impairment where enzyme replacement therapy (ERT) has limited efficacy.
Inventiva is currently preparing a 26 week, double blinded, randomized, placebo controlled phase IIa clinical study to investigate the safety, pharmacokinetics and efficacy of oral administration of odiparcil in adult patients with MPS type VI receiving enzyme replacement therapy (ERT). The study also has an open label arm with patients currently not on ERT. The iMProveS trial will be recruiting patients at the end of the year.