We develop novel and differentiated oral small molecule therapies for patients suffering from diseases with significant unmet medical need

We are a clinical-stage biopharmaceutical company focused on the development of oral small molecule therapies for the treatment of patients with significant unmet medical needs in the areas of fibrosis, lysosomal storage disorders and oncology.

We have built a pipeline backed by a discovery engine with an extensive library of proprietary molecules, a wholly-owned research and development facility and a team with significant expertise and deep experience in the development of compounds that target nuclear receptors, transcription factors and epigenetic modulation. By leveraging these assets and expertise, we are advancing two clinical candidates in two indications, as well as a deep pipeline of earlier stage programs.

Two royalty bearing collaborations with Sino Biopharm and Hepalys Pharma, Inc.

Two innovative clinical programs: NATIVE and IMPROVES

Pre-clinical pipeline in oncology and fibrosis

State-of-the-art R&D capabilities inherited from Abbott, Solvay, Fournier

Research Areas & Pipeline

With many potential new therapies that are currently in clinical trials, our goal is to be the industry leader in developing drugs that interact with nuclear receptors, transcription factors and epigenetic modulators.

Lanifibranor, our lead product candidate, is an anti-fibrotic treatment acting on the three PPARs (peroxisome proliferator-activated receptors), which plays a key role in controlling the metabolic, inflammatory and fibrotic processes. We are investigating lanifibranor for the treatment of NASH, a severe and increasingly prevalent liver disease that is already affecting over 30 million people in the United States.

We are developing a second clinical program with odiparcil for the treatment of patients with mucopolysaccaridosis type VI (MPS), a rare and severe genetic disease. Odiparcil also has 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).
Our pipeline

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