Pathologic fibrosis is responsible for the morbidity and mortality of many serious diseases of the liver, lung, kidney, pancreas, eye, heart, skin, skeletal muscle tissue, intestine, and other organs and is estimated to contribute to over 45% of deaths in the developed world. Indalo has identified two serious fibrotic diseases, both of which have high unmet medical needs, as indications for our initial clinical studies:
Non-alcoholic steatohepatitis (NASH) is a progressive disease caused by excessive fat accumulation in the liver, unrelated to alcohol use, that induces chronic inflammation and damage (ballooning) of hepatocytes, resulting in fibrosis that can lead to cirrhosis, liver failure, and death. The stage of liver fibrosis is a strong predictor of clinical outcomes. Ongoing persistence of obesity with increasing rates of diabetes is expected to increase the prevalence of NASH dramatically in coming years, and the disease is projected to become the leading cause of liver transplants in the United States by as early as 2020. There are currently no FDA-approved therapies for NASH, and most therapies currently in development target metabolic processes rather than fibrosis directly.
Idiopathic pulmonary fibrosis (IPF) is an irreversible, relentlessly progressive, and ultimately fatal disease characterized by progressive loss of lung function and impaired gas exchange due to fibrosis. Patients experience worsening shortness of breath, diminished quality of life, and progressive functional impairment. Median survival time from diagnosis is two to five years, with a five-year survival rate of 20-40 percent. IPF is a large orphan disease, with a prevalence in the United States of approximately 150,000 patients, and with about 35,000 new cases diagnosed each year. Two therapies for IPF are currently approved in the United States and Europe, but both have limited market penetration due in part to tolerability issues.