Inventiva Announces Positive DSMB Review in Phase IIb FASST Trial in Systemic Sclerosis with Lanifibranor
January 04, 2018
> DSMB recommends trial to continue unchanged
> 145 patients randomized in trial
> Topline results anticipated early 2019
Daix (France) January 4, 2018 – Inventiva, a biopharmaceutical company developing innovative breakthrough therapies, particularly for the treatment of fibrotic diseases, today announced that the Data Safety Monitoring Board (DSMB) has completed its review of the Phase IIb FAAST (For A Systemic Sclerosis Treatment) trial in systemic sclerosis with lanifibranor. After reviewing all safety data, including adverse events, and the study’s conduct, the DSMB recommended that the study continue without any modifications to the protocol. Of the 145 randomized patients enrolled into the trial, 100 patients have been treated for 6 months including 54 patients that have already completed the one year treatment. The study is progressing as planned with no specific concerns and topline results are anticipated in early 2019.
Lanifibranor is a next generation panPPAR modulator designed as a moderately potent and well balanced PPAR α δ and y. This unique profile was conceived in order to obtain an optimal therapeutic margin with strong efficacy and tolerance. Lanifibranor displayed an antifibrotic efficacy superior to selective PPARα, PPAR δ or PPARy agonists in several relevant preclinical models. Inventiva is also conducting a Phase IIb clinical study in NASH with lanifibranor.
About the Phase IIb FASST trial:
The Phase IIb FASST trial is a one-year randomized double-blind study designed to measure the effect of lanifibranor on the progression of systemic sclerosis (SSc). Patients will receive either lanifibranor or placebo according to the study protocol, approved by the European Medicines Agency (EMA). The primary endpoint is change in the Modified Rodnan Skin Score (MRSS), which is a measure of disease progression accepted by both Food and Drug Administration (FDA) and the EMA. Inclusion criteria are based on a MRSS between 10 and 25, and diffuse systemic sclerosis diagnosed from less than 3 years. Patients are allowed to continue their ongoing treatments, including immunosuppressive therapies. Lanifibranor has been granted orphan drug status for the treatment of systemic sclerosis by the EMA and the FDA. The orphan drug status provides certain advantages for the sponsor such as reduced procedure costs and commercial exclusivity.
About systemic sclerosis:
Systemic sclerosis is a rare and complex disease affecting the auto-immune system, the microvascular system and conjunctive tissues. This fibrotic disease mainly affects the skin, but also the lungs, the heart, the gastro-intestinal tract, and the kidneys. Due to the progressive failure of different organs, systemic sclerosis is a severe disease with a high mortality rate. Once patients are diagnosed with systemic sclerosis, generally between the ages of 40 and 50, the median survival period is of 11 years. Close to 170,000 people suffer from systemic sclerosis worldwide, with women outnumbering men by a ratio of more than five to one1.
The disease owes its original name to scleroderma, the skin condition that it provokes, which derives from the Greek words skleros (hard) and derma (skin). The disease causes severe physical and psycho-social consequences; the former may be fatal for patients whose vital organs are affected. Deeper study of this skin fibrosis has led to its classification into two sub-categories, respectively called limited cutaneous systemic sclerosis and diffuse cutaneous systemic sclerosis. The latter is more serious and is targeted in the FASST trial.
To date, only symptomatic drugs with limited therapeutic effects are available in order to attenuate the consequences of fibrosis progression. However, they do not prevent, delay or reverse the disease’s devastating process.
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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