Inventiva to present in vivo data with odiparcil at the 14th WORLDSymposium™
January 31, 2018
> Data confirms potential as first oral therapy for MPS VI patients
Daix (France), January 31st, 2018 – Inventiva, a biopharmaceutical company developing innovative breakthrough therapies, particularly for the treatment of fibrotic diseases, today announced that Dr Eugeni V. Entchev, Head of odiparcil preclinical pharmacology program at Inventiva, will give a presentation entitled “Odiparcil is a promising substrate reduction therapy in MPS VI murine model” at the 14th WorldSymposium;? on February 7, 2018, in San Diego, California.
After an initial closed session presentation during the MPS Society National Conference in July 2017, this will be the first public presentation of the data generated in a genetic mouse model for MPS VI (Maroteaux-Lamy syndrome) after a 6-month treatment period with two doses of odiparcil. The data demonstrate that odiparcil reduced GAG (glycosaminoglycan(s)) accumulation in the liver, kidney, spleen, heart, eye, and skin of diseased animals. Odiparcil also restored normal corneal structure in the eye and reduced thickening of the trachea and femoral growth plate cartilages. These important findings suggest that odiparcil could lead to treating eye- and cartilages-related clinical manifestations seen in MPS VI patients. Finally, mobility was improved by odiparcil in diseased animals.
Presentation title: “Odiparcil is a promising substrate reduction therapy in MPS VI murine model”
Presenter: Dr Eugeni V. Entchev, Head of odiparcil preclinical pharmacology program, Inventiva Session: Translational Research I
Date/Time: Wednesday, Feb. 7, at 10:45 a.m. PT
Location: Manchester Grand Hyatt, 1 Market Place, San Diego, California, U.S.A.
The WORLDSymposiumTM is a leading annual research conference dedicated to lysosomal diseases. Since 2002, the W.O.R.L.D. (We’re Organizing Research on Lysosomal Diseases) meeting has grown to an international research conference that attracts over 1600 participants from more than 50 countries around the globe.
Odiparcil is the first new treatment in development for MPS VI in over a decade. The current standard of care is enzyme replacement therapy (ERT), which requires weekly infusions. An orally available therapeutic such as odiparcil would greatly increase the quality of life of patients. More importantly, the data generated in MPS VI mice demonstrate that odiparcil could treat clinical manifestations linked to GAG accumulation in tissues and organs where current ERT is not effective. Odiparcil is well distributed in the body even in tissues that are poorly vascularized, such as cartilages, or protected by a barrier, such as the eye. Inventiva believes odiparcil could meaningfully improve the lives of MPS VI patients, and become the new standard of care. On December 30, 2017, the first patient was enrolled in the Phase IIa iMProveS (improve MPS treatment) trial of odiparcil in MPS VI patients. Results from this study are expected in H1 2019. Odiparcil has received orphan drug designation for MPS VI in the United States and Europe.
About MPS VI
MPS VI is a rare, pediatric, genetic, degenerative disease 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 suffer from short stature, corneal clouding, hearing loss, dysostosis multiplex, hepatosplenomegaly, cardiac valve disease and reduced pulmonary function. 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 in patients with severe forms of the disease, or longer in patients with less severe forms. The prevalence of MPS VI is estimated to be 1 in 225,000 live births and varies between countries. There is no cure for MPS VI and current treatment options such as ERT or hematopoietic stem cell transplant (HSCT) leave the patients with high 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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