Odiparcil iMProveS MPS

Dermal fibroblasts from a MPS VI patient overloaded with GAG. Source: Company data.

First substrate reduction therapy

First substrate reduction therapy

Odiparcil (IVA336) is a small molecule developed by Inventiva Pharma with the potential to become the first oral substrate reduction therapy for Mucopolysaccharidosis I, II and VI. Odiparcil has received orphan drug designation by the FDA and EMA.

Major unmet medical need

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.

Demonstrated safety and tolerability

Odiparcil has demonstrated good tolerability, safety and efficacy in phase I and phase IIa clinical studies , in approximately 700 healthy volunteers and 1100 patients and it’s unique mechanism of action  has been investigated in several preclinical models.

The iMProveS trial

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.

Our Contact

For further information on the iMProveS trial please contact us

Phone +33 (0) 380 447 500 - Ask for Mireille Tallandier
Address Inventiva Pharma - 50, rue de Dijon - 21121 Daix, France