Inventiva Announces Enrollment of First Patient into Its Odiparcil Phase IIa Trial (iMProveS) for MPS VI
January 03, 2018
> Trial to enroll 24 patients at 2 clinical trial sites
> Results expected during the first semester of 2019
Daix (France), January 3, 2018 – Inventiva, a biopharmaceutical company developing innovative therapies, particularly to treat fibrosis, today announced the enrollment of the first patient in its Phase IIa iMProveS (improve MPS treatment) trial targeting mucopolysaccharidosis VI patients (MPS VI).
“Odiparcil is the first new treatment in development for MPS VI patients in over a decade and is a potentialsignificant step forward for patients,” said Jean-Louis Abitbol, M.D., M.Sc., Inventiva’s Chief Medical Officer and Head of Development, “Current enzyme replacement therapy requires weekly infusions, therefore an orally available therapeutic such as odiparcil would greatly increase the quality of life of patients. More importantly, thanks to its optimal distribution in the body, odiparcil has shown efficacy in tissues and organs where current enzyme replacement therapy is not effective. Overall we believe odiparcil could meaningfully improve the lives of these patients and become the standard of care in MPS VI.”
The iMProveS clinical study is a 26-week study designed to demonstrate the safety, tolerability, and efficacy of odiparcil in 18 adult MPS VI patients receiving enzyme replacement therapy (ERT). The study also has an open- label arm with 6 patients currently not on ERT. If positive, the Company plans to pursue a pivotal Phase III study of odiparcil in patients with MPS VI. Odiparcil has received orphan drug designation for MPS VI in the United States and Europe. Results from the Phase IIa study are expected during the first semester of 2019.
About MPS VI
MPS VI (Maroteaux-Lamy syndrome), 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 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 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 enzyme replacement therapy (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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