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Inventiva announces positive results from Phase IIa clinical study with odiparcil in mucopolysaccharidosis type VI

December 18, 2019

Daix (France), December 18, 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 results from its Phase IIa iMProveS (improve MPS treatment) clinical study evaluating odiparcil for the treatment of mucopolysaccharidosis (MPS) type VI, a rare, progressive genetic disorder with high unmet medical need.

The 26-week Phase IIa clinical trial included 20 patients aged 16 years or older suffering from advanced stages of MPS VI. 15 patients were randomized in a double-blind, placebo-controlled cohort and received an oral dose of 250mg or 500mg of odiparcil or placebo twice a day for six months, in addition to enzyme replacement therapy (ERT), the current standard of care. The remaining five patients were included in an open-label cohort and received an oral dose of 500mg of odiparcil twice a day for six months, without being treated with ERT. 13 patients completed the study: four patients who received placebo in addition to ERT and nine patients equally distributed in each of the three odiparcil groups.

Frédéric Cren, Chairman, Chief Executive Officer and cofounder of Inventiva, commented: “We are very pleased with today’s positive results and would like to thank all patients, caregivers, investigators and our team for their commitment and dedication to this study and the overall program. We believe that the efficacy shown after only six months of treatment and its oral formulation could make odiparcil a treatment of choice for MPS VI patients, particularly given the high unmet medical need in this disease field. Based on the observed efficacy data and odiparcil’s good safety profile, we have decided to move forward with the clinical development of odiparcil in MPS VI with a focus on children, the target population for this compound.”

Chris Hendriksz, member of the iMProveS clinical study steering committee and extraordinary professor of Paediatrics and Child Health, University of Pretoria, South Africa, said: “This is an exciting moment in the MPS field as we now have the first data for an oral compound which shows efficacy results in hard-to-reach tissues for MPS disorders. Despite the current approved therapies, there is still a huge unmet medical need related to eye, heart and bone manifestations and seeing positive changes in this study in a very short treatment time is very exciting for patients.”

The clinical study met its safety primary objective further supporting the good overall safety profile of odiparcil already observed in previous Phase I and Phase II clinical studies conducted for the prevention of thrombosis. All

investigators of the iMProveS study reported positive experience with odiparcil in terms of safety. The majority of adverse events were mild or moderate. One death occured in the placebo group and three serious adverse events (SAEs) were assessed as treatment-related in patients in the odiparcil groups. Two of these SAEs were biological findings qualified as laboratory false-positive. The third SAE was a skin reaction, which is frequently observed in ERT-treated MPS VI patients. Compared to previous Phase I and II clinical studies conducted with odiparcil for the prevention of thrombosis, no new safety findings were observed.

Considering the short study duration and the advanced status of the disease in patients included in the study, the iMProveS study showed positive results regarding the efficacy of odiparcil:

− Improvements were observed in patients treated with odiparcil, in addition to ERT, with regards to corneal clouding as well as cardiac and respiratory functions.

− Consistent with odiparcil’s mechanism of action, a dose-dependent urinary clearance of glycosaminoglycans (GAGs), used as an activity biomarker, was clearly demonstrated in the entire patient population treated with odiparcil. Similarly to ERT, odiparcil did not induce a reduction of leukocyte glycosaminoglycans (leukoGAGs), which was therefore not confirmed as a biomarker for the decrease of GAG accumulation in this study. Work is planned regarding skin GAG analysis.

− Regarding locomotor function, no clear difference was observed among the different patient groups.

Results from the pharmacokinetics analysis were in line with expectations and will be used for dose selection in the next study planned with MPS VI children. In the iMProveS study, the pharmacokinetic profile obtained in MPS VI patients treated with odiparcil is not impacted by ERT and is consistent with profiles previously observed in Phase I and Phase II studies in prevention of thrombosis.

Based on the iMProveS clinical study results, Inventiva has decided to continue the clinical development of odiparcil for the treatment of MPS VI. To this end, the Company aims to launch, as planned, a clinical study evaluating odiparcil in MPS VI children, the target population for this treatment. Inventiva is currently finalizing the study design to take into account today’s results. Details will be posted on once the study protocol has been finalized and validated with the relevant regulatory authorities.

Results presentation

Inventiva’s management team will present today’s study results during a dedicated conference call and webcast on Thursday, December 19, 2019 at 2:00 pm (Paris time).

To join the conference call, please use the code 9149459 after dialling one of the following numbers:

France: +33 1 70 73 27 27

Belgium: +32 10 39 12 06

Denmark: +45 32 72 75 18

Germany: +49 69 22 22 49 10

Netherlands: +31 20 71 57 366

Switzerland: +41 44 58 04 873

United Kingdom: +44 203 00 95 710

United States: +1 917-720-0178

The presentation accompanying this conference call will be simultaneously accessible on Inventiva’s website in the “Investors” – “Financial Results & Presentations” section. It can be followed live or by replay in the same section of the Company’s website and at:


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.