Daniela Semedo, PhD
May 18, 2017

Albireo Pharma has secured two new patents from the U.S. Patent and Trademark Office (USPTO) for its lead drug candidate A4250 to treat liver diseases such as nonalcoholic steatohepatitis (NASH).

Boston-based Albireo has reported promising preclinical results with A4250 in an animal model of NASH, and is currently conducting a preclinical program directed towards novel bile acid modulators to treat NASH — a serious chronic liver disease that resembles alcoholic liver disease, but occurs in people who drink little or no alcohol.

In such patients, fat accumulation in the liver, known as nonalcoholic fatty liver disease (NAFLD) or steatosis, and other factors such as high LDL cholesterol and insulin resistance induce chronic liver inflammation, causing progressive scarring of tissue and cirrhosis. That in turn, may lead to liver failure and death.

The USPTO has also granted A4250 a patent to treat progressive familial intrahepatic cholestasis (PFIC), a rare and life-threatening genetic liver disorder, as well as other liver diseases.

“The allowance of this patent further fortifies our intellectual property protection for our lead product candidate, providing expected exclusivity into 2031 for A4250 in the treatment of PFIC among other indications,” Albireo President and CEO Ron Cooper said in a press release, adding that a Phase 3 trial in PFIC patients will begin later this year. “This patent allowance is another step forward in our efforts to bring a pharmacological treatment option for children with PFIC who greatly need it.”


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