Discovering significant breakthroughs in fibrosis.

Clinical Trials

Blade will initiate clinical trials in human fibrotic disease in the coming months.

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FIBROSIS-RELATED DISEASES

Diseases characterized by uncontrolled, progressive fibrosis include idiopathic pulmonary fibrosis (IPF), non-alcoholic steatohepatitis (NASH) and systemic sclerosis (SSc).

Idiopathic Pulmonary Fibrosis (IPF)

Idiopathic pulmonary fibrosis (IPF) is a rare, highly debilitating, and poorly understood disease. Most commonly affecting individuals over 50 years old, IPF is characterized by interstitial pneumonia and chronic progressive lung fibrosis. Over the course of the disease, healthy lung tissue is replaced with fibrotic tissue, causing a continuous decline in lung function. Disease progression is unpredictable and is often rapid, with many patients progressing to lung failure and potentially death within 2 - 3 years. Unfortunately, the disease’s early genesis is unknown, and there are no tests to identify patients that are at greatest risk of developing IPF. Members of Blade’s own team were instrumental in the pioneering work at InterMune that led to the approval of pirfenidone to treat IPF; however, there remains a pressing need for effective therapies for this serious and as-of-yet incurable disease.

Non-Alcoholic Steatohepatitis (NASH)

It is estimated that up to one-third of the populations in the US and Europe have a condition termed non-alcoholic fatty liver disease (NAFLD), which is characterized by steatosis, or excessive accumulation of fat in the liver (Wree, 2013; Blachier, 2013). Many of these individuals, for reasons not totally understood, subsequently develop liver inflammation, or steatohepatitis. This condition, called non-alcoholic steatohepatitis, or NASH, develops in roughly 10 - 20% of NAFLD patients, accounting for approximately 10 - 20 million individuals in the US (Schattenburg, 2011). Individuals experiencing chronic liver inflammation often develop liver fibrosis, with eventual risks of cirrhosis, hepatocellular carcinoma, and liver failure. Based on current projections, NASH is predicted to become the leading cause of liver transplantation by 2020 (Wree, 2013). Unfortunately, there are no therapies available to prevent or treat liver fibrosis.

Systemic Sclerosis – Interstitial Lung Disease

Systemic Sclerosis (SSc) – ILD Systemic sclerosis (SSc) is a rare and chronic condition characterized by organ fibrosis (including hardening of the skin) and vasculopathy. Fibrosis affects multiple organ systems including skin, lungs, kidneys, GI, and heart. Interstitial lung disease (ILD) and pulmonary arterial hypertension (PAH) are severe manifestations of SSc and when present, increase mortality significantly. ILD in SSc is often associated with a decline in lung function within the first several years of lung disease onset. Clinical symptoms of SSc-ILD can be variable. Patients with mild ILD can be asymptomatic in the early stages of their disease and may not be diagnosed until lung decline is considerable. Administration of treatment early in the course of SSc-ILD may lead to improved lung function. Thus, screening and initiation of treatment with an anti fibrotic at an early stage in the disease process is highly desirable.

References

Wree et al. 2013. Nat. Rev. Gastroenterol. Hepatol. 10: 627–636.
Blachier et al. 2012. J. Hepatol. 58: 593-608.
Schattenburg et al. 2011. Curr Opin Lipidol. 22:479-488.
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