Orphan disease liver
While surged earlier this year and has surged this week after agreed to purchase hepatitis stock at a 239% premium, it seems the herd has realized that small cap is also a liver disease stock as shares suddenly surged 56.69% on no apparent company news. But just what is Conatus Pharmaceuticals and is it worth taking a chance on?
What is Conatus Pharmaceuticals Inc?
Small cap Conatus Pharmaceuticals a biotechnology company focused on the development and commercialization of novel medicines to treat liver disease. The company is developing its lead compound, emricasan, for the treatment of patients with chronic liver disease and acute exacerbations of chronic liver disease. Emricasan is a first-in-class, orally active pan-caspase protease inhibitor designed to reduce the activity of all ten human caspases, which are enzymes that mediate inflammation and cell death, or apoptosis. Conatus Pharmaceuticals plans to study emricasan in patients with established liver cirrhosis and decompensated liver disease with the first planned trials in ACLF are to evaluate whether the treatment can halt the progression of decompensation to multi-organ failure - a requirement for liver transplant or death in an acutely decompensating cirrhotic patient population. Two studies are planned in this patient population.
As for potential peers in the liver disease or hepatitis space, Intercept Pharmaceuticals Inc is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic liver and intestinal diseases utilizing its proprietary bile acid chemistry; Achillion Pharmaceuticals, Inc is focused on solutions for the most challenging problems in infectious disease including hepatitis C and resistant bacterial infections; and Idenix Pharmaceuticals Inc’s current focus is on the treatment of hepatitis C virus infections plus the company has previously discovered and developed antivirals for the treatment of hepatitis B virus and HIV/AIDS.
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What is the difference between an Orphan Disease and a Rare Disease? - Quora
An orphan disease is a regulatory definition of a rare disease so it gets different regulatory treatment