Odiparcil development advances with recruitment of first patients in a new biomarker study in MPS VI children and adults
September 2, 2019
Daix (France), September 2, 2019 – Inventiva (Euronext: IVA), a clinical-stage biopharmaceutical company developing oral small molecule therapies for the treatment of diseases in the areas of fibrosis, lysosomal storage disorders and oncology, today announced the recruitment of the first patients in a new biomarker study in children and adults with mucopolysaccharidosis type VI (MPS VI)
Conducted at the UCSF Benioff Children’s Hospital in Oakland, California, by Dr Paul R. Harmatz, the trial will investigate leukocyte glycosaminoglycan (leukoGAGs) levels in three children and skin glycosaminoglycan (skinGAGs) levels in three adults with MPS VI before and post enzyme replacement therapy (ERT), and in six age-matched control subjects not affected by MPS VI. This study is part of Inventiva’s strategy to develop biomarkers to evaluate the efficacy of odiparcil, the Company’s drug candidate for the treatment of MPS, in reducing leukoGAGs and skinGAGS. This approach has been launched following the Food and Drug Administration’s (FDA) guidance on the relevance of biomarkers for diseases such as MPS1. Results of this study are expected in the first half of 2020.
A first biomarker study sponsored by Inventiva showed that, despite ERT treatment, the current standard of care, leukoGAG levels remained very high and were not impacted when measured one hour after ERT infusion although the activity of the arylsulfatase B2 enzyme was very high. This finding suggests that leukoGAG levels may be further reduced with a new treatment such as odiparcil.
Measurement of leukoGAGs and skinGAGs is performed in the ongoing Phase IIa iMProveS (improve MPS treatment) clinical study evaluating odiparcil for the treatment of MPS VI patients. Given the recent progress achieved in the iMproveS study, headline results for all treatment arms are now expected by the end of the year.
Inventiva had initially planned to publish the study results in two steps: the headline results of the double-blind placebo controlled arms by the end of the year followed by the results of the open label cohort during Q1 2020.
Dr. Paul R. Harmatz, the principal investigator of this new biomarker study, said: “I am very excited about the launch of this second biomarker study with the recruitment of the first patients. Following our first biomarker study that demonstrated the limitations of ERT in reducing leukoGAGs, this trial will help us better understand the dynamic of the response to this therapy in leukocytes. It could also possibly provide a second measure of GAG storage in a readily accessible tissue such as the skin, thereby strengthening the appraisal of intracellular GAG variations.”
Lanifibranor, its lead product, is an anti-fibrotic treatment acting on the three alpha, gamma and delta PPARs (peroxisome proliferator-activated receptors), which play key roles in controlling the fibrotic process. Its anti-fibrotic action targets two initial indications with substantial unmet medical need: NASH, a severe and increasingly prevalent liver disease already affecting over 30 million people in the United States, and systemic sclerosis, a disease with a very high mortality rate and for which there is no approved treatment to date.
Inventiva is also developing a second clinical program with odiparcil (IVA 336) for the treatment of patients with mucopolysaccaridosis type VI (or Maroteaux-Lamy syndrome), a rare and severe gene disease affecting children. Odiparcil has also the potential to address other MPS types, characterized by the accumulation of chondroitin or dermatan sulfate (MPS I or Hurler/Sheie syndrome, MPS II or Hunter syndrome, MPS IVa or Morquio syndrome and MPS VII or Sly syndrome). Inventiva is also developing a portfolio of early research projects in the field of oncology.
Inventiva benefits from partnerships with world-leading research entities such as the Institut Curie in the field of oncology. Two strategic partnerships have also been established with world-class major pharmaceutical companies AbbVie and Boehringer Ingelheim in the fields of autoimmune diseases (specifically in psoriasis) and fibrosis respectively. These partnerships provide milestone payments to Inventiva upon the achievement of pre-clinical, clinical, regulatory and commercial milestones, in addition to royalties on the products resulting from the partnerships.
Inventiva employs over 100 employees and owns R&D facilities near Dijon, acquired from the international pharmaceutical group Abbott. The Company owns, a proprietary chemical library of over 240,000 molecules as well as integrated biology, chemistry, ADME and pharmacology platforms.
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