Clinical data company TARGET PharmaSolutions of Chapel Hill will collaborate with Dublin-based Allergan, a global pharmaceutical company, to study a major liver disease and future treatment options for it.
The two companies will join forces on TARGET’s five-year study of up to 15,000 adults and children who have nonalcoholic steatohepatitis (NASH), a progressive form of fatty liver disease in which fat accumulates in the liver, causing inflammation and liver damage.
“This unique collaboration will help to further our collective understanding of the disease, effective modes of treatment and outcomes across the spectrum of patient groups,” David Nicholson, chief research and development officer of Allergan, said in a news release.
The North Carolina Biotechnology Center awarded TARGET a $500,000 Strategic Growth Loan in 2016.
TARGET-NASH is a longitudinal, observational study that involves academia, industry, regulatory agencies and the liver disease community. The study design is disease focused, not drug specific, allowing for continuous acquisition of natural history and outcomes data as new drugs enter the market and clinical treatment paradigms evolve, the companies said.
Study enrollment began last summer
The first patient was enrolled in TARGET-NASH in August 2016, and thousands more will be added in coming years.
The study model is based on the success of HCV-TARGET, a case study of Hepatitis C formed in 2011 by Michael Fried, M.D., of the University of North Carolina and David Nelson, M.D., of the University of Florida, both of whom subsequently became TARGET co-founders and scientific advisors. The hepatitis C study has enrolled over 10,000 patients and generated data that has been used by physicians, payers, and regulatory agencies around the world.
“TARGET-NASH is critical for the scientific and regulatory community as we prepare for new agents for the treatment of NASH,” said Arun Sanyal, M.D., co-chair of the TARGET-NASH steering committee. “In the immediate term, it will give us a critical understanding of NASH diagnosis and management in the real world across multiple populations. In the longer term, it is the perfect platform to have a deep understanding of the safety and effectiveness of these new agents across populations not included or underrepresented in Phase 3 clinical trials.”
About 30 percent of the U.S. population and 20 percent of people globally have nonalcoholic fatty liver disease. NASH affects about 5 percent of the U.S. population but is the nation’s fastest growing cause of liver cancer and liver transplant.
NASH increasing with obesity rates
The increasing prevalence of NASH is related to the growing obesity epidemic. The disease is often diagnosed in patients who have diabetes, high cholesterol or high triglycerides.
There is currently no approved treatment for NASH, but Allergan said the disease is “an adjacency” to its broad portfolio of gastrointestinal drugs.
“Allergan is focused on identifying, developing and bringing forward innovative treatments for patients with significant unmet medical needs, such as NASH,” said Nicholson.
The Allergan collaboration is the fifth secured by TARGET since TARGET’s formation in 2015. It has two with Bristol-Myers Squibb and two with Intercept Pharmaceuticals.
TARGET is backed by a Series A funding round completed in 2016 and led by Raleigh-based REX Health Ventures, the venture capital arm of UNC REX Healthcare, with participation by VentureSouth of Greenville, S.C., and local angel investors.
Meg Powell, Pharm.D., TARGET’s president, chief executive officer and co-founder, said in a January profile of the company by NCBiotech that TARGET had 20 employees and expected to hire about five more in anticipation of more company collaborations.
