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Inventiva Announces New Compelling Data on Odiparcil/IVA336 to be Presented at MPS Society National Conference

June 29, 2017

> Confirmation of potential to be the first orally available substrate reduction therapy for MPS VI patients

> Phase IIa iMProveS clinical study scheduled to begin before year end 2017

Daix (France), June 29, 2017 – 6:00pm CEST – Inventiva, a biopharmaceutical company developing innovative therapies, particularly to treat fibrosis, today announced that Professor Chris Hendriksz, of FYMCA Medical Ltd. and University of Pretoria, South Africa, will be presenting new preclinical data on Odiparcil (formerly IVA336) in a closed session at the MPS Society National Conference, which will be held July 7-9, 2017 in Coventry, UK.

The data, generated over a 6-month treatment with two doses of Odiparcil in a genetic mouse model for MPS VI, demonstrate that Odiparcil restored a normal corneal structure in the eye, which could lead to a complete rescue of the disease phenotype in the eye. Odiparcil also reduced GAG accumulation in the liver, kidney, spleen, heart, eye, and skin of diseased animals and produced a dose-dependent reduction of cartilage thickness in the trachea and femoral growth plate. Finally mobility was improved by Odiparcil in the diseased animals.

These results demonstrate that Odiparcil has the potential to be the first orally available substrate reduction therapy for MPS VI patients. “These breakthrough pre-clinical results support the potential of Odiparcil as a new and very promising approach to treating MPS patients,” said Professor Hendriksz. “MPS are devastating diseases, and there remains a significant unmet medical need despite the availability of enzyme replacement therapies, which unfortunately are not able to resolve the symptoms occurring in certain regions especially in the ophthalmological system, joints, cartilages, cardiac valves etc. Odiparcil could prove beneficial to MPS patients as a substrate reduction therapy as a stand-alone treatment or in adjunction to current treatments.”

We are very excited by the data, as well as the positive feedback received from patients’ associations and MPS key opinion leaders. We are looking forward to the upcoming iMProveS trial, which could confirm the therapeutic benefit of Odiparcil, leading potentially to ERT replacement,” added Pierre Broqua, Ph.D., Chief Scientific Officer and Co-Founder of Inventiva.

In addition to the Odiparcil data, Professor Hendriksz will also present the design of the planned Phase IIa iMProveS (improve MPS treatment) clinical study, which is expected to enroll its first patient before year end. The iMProveS clinical study will be a 26-week study designed to demonstrate the safety, tolerability, and efficacy of Odiparcil in 24 adult MPS VI patients and will be conducted at two European clinical sites. Eighteen patients receiving enzyme replacement treatment (ERT) will be randomized into 3 arms: two active dose levels of Odiparcil (250 mg and 500 mg, bid) or placebo. The study will also include an additional arm of six patients untreated by ERT who will receive 500 mg bid of Odiparcil. This study, if positive, will allow for enrollment of MPS VI patients into the pivotal Phase III trial.

For more information on the MPS Society National Conference, refer to

About MPS VI:

MPS VI (Maroteaux-Lamy syndrome), is a rare pediatric genetic degenerative diseases characterized by the abnormal functioning of the enzyme N-acetylgalactosamine 4-sulphatase (arylsulphatase B; ASB) leading to the accumulation of dermatan sulfate and chondroitin sulfate in the cells, tissues and organs. Patients have coarse faces, short stature, corneal clouding, hearing loss, dysostosis multiplex, hepatosplenomegaly, cardiac valve disease and reduced pulmonary function without intellectual deficit. As with other MPS, the time of onset, rate of progression and extent of the disease may vary between the affected individuals. The life expectancy of MPS VI patients, if untreated, is approximately 20 years for patients with severe forms of the disease and more for patients with less severe forms.

The prevalence of MPS VI is estimated to be 1 in 225,000 live births. It varies between countries. There is no cure for MPS VI and current treatment options such as enzyme replacement therapy (ERT) or hematopoietic stem cell transplant (HSCT) leave the patients with high unmet medical needs.


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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.

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