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Inventiva’s latest research on odiparcil’s mechanism of action published in leading peer-reviewed scientific journal PLOS ONE

May 18, 2020

Daix (France), May 18, 2020 – Inventiva (Euronext: IVA), a clinical-stage biopharmaceutical company developing oral small molecule therapies for the treatment of non-alcoholic steatohepatitis (NASH), mucopolysaccharidoses (MPS) and other diseases with significant unmet medical need, announced today the publication of its latest research supporting Inventiva’s understanding of the mechanism of action of odiparcil, the Company’s product candidate for the treatment of MPS VI, in the scientific journal PLOS ONE. Published by the US-based Public Library of Science (PLOS) since 2006, PLOS ONE is one of the leading peer-reviewed, open access scientific journals worldwide. The non-clinicalstudies conducted by Inventiva showed that in vitro, treatment with odiparcil was associated with reduced intracellular GAG content in skin fibroblasts isolated from MPS VI patients and concomitant the secretion of GAGs outside the cells in the culture media. Furthermore, Inventiva’s research team observed in vivo in wildtype rats that odiparcil was well distributed in MPS VI disease-relevant tissues and organs such as bone, cartilage, heart and cornea, where enzyme replacement therapy (ERT), the current standard of care for MPS VI, has shown poor penetration. In a mouse genetic model of MPS VI, treatment with odiparcil was consistently associated with urinary excretion of GAGsthroughout the treatment period and significantly reduced GAG accumulation in tissues such as liver and kidney. In addition, treatment with odiparcil was also associated with diminished pathological cartilage thickening of trachea and femoral growth plates of MPS VI mice. The data set obtained with this latest study supports Inventiva’s understanding of the mechanism of action of odiparcil, which acts to divert the synthesis of cellular GAGs into secreted soluble species. This data set is also consistent with positive results observed in the clinical development of odiparcil, which is undergoing clinical trials as a potential orally-delivered GAG clearance therapy for MPS VI patients.

Pierre Broqua, CSO and cofounder of Inventiva, commented: “We are very pleased to see our latest in vitro and in vivo research on odiparcil published in the renowned PLOS ONE scientific journal, which testifies to our innovative R&D approach in the field of MPS. Our results support our hypothesis about the mechanism of action of odiparcil, following the release of positive results from our Phase IIa iMProveS clinical trial with odiparcil in MPS VI at the end of last year. We are excited by these latest findings, further encouraging the development of our drug candidate for the treatment of MPS VI, as we prepare the Phase I/II clinical trial with odiparcil in MPS VI children.”


Frédéric Cren

Chief Executive Officer
+33 3 80 44 75 00


Aude Hillion/ Yannick Tetzlaff
Media relations
+33 1 53 96 83 83

LifeSci Advisors
Monique Kosse

Investor relations

About Inventiva
Inventiva is a biopharmaceutical company specialized in the development of drugs interacting with nuclear receptors, transcription factors and epigenetic modulators. Inventiva’s research engine opens up novel breakthrough therapies against fibrotic diseases, cancers and orphan diseases with substantial unmet medical needs.

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.

Important Notice
This press release contains forward-looking statements, forecasts and estimates with respect to the clinical development
plans, business and regulatory strategy, and anticipated future performance of Inventiva and of the market in which it operates. Certain of these statements, forecasts and estimates can be recognized by the use of words such as, without limitation, “believes”, “anticipates”, “expects”, “intends”, “plans”, “seeks”, “estimates”, “may”, “will” and “continue” and similar expressions. Such statements are not historical facts but rather are statements of future expectations and other forward-looking statements that are based on management”s beliefs. These statements reflect such views and assumptions prevailing as of the date of the statements and involve known and unknown risks and uncertainties that could cause future results, performance or future events to differ materially from those expressed or implied in such statements. Actual events are difficult to predict and may depend upon factors that are beyond Inventiva”s control.

There can be no guarantees with respect to pipeline product candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. Therefore, actual results may turn out to be materially different from the anticipated future results, performance or achievements expressed or implied by such statements, forecasts and estimates. Given these uncertainties, no representations are made as to the accuracy or fairness of such forward-looking statements, forecasts and estimates. Furthermore, forward-looking statements, forecasts and estimates only speak as of the date of this press release. Readers are cautioned not to place undue reliance on any of these forward-looking statements.

Please refer to the “Document de référence” filed with the Autorité des Marchés Financiers on April 13, 2018 under n° R.18-013 for additional information in relation to such factors, risks and uncertainties.

Inventiva has no intention and is under no obligation to update or review the forward-looking statements referred to above. Consequently, Inventiva accepts no liability for any consequences arising from the use of any of the above statements.