Gilead Sciences will acquire Phenex Pharmaceuticals’ Farnesoid X Receptor (FXR) program, consisting of small molecule FXR agonists designed to treat liver diseases that include nonalcoholic steatohepatitis (NASH), the companies said today. The deal could generate up to $470 million for Phenex in upfront and development milestone payments.
FXR is a nuclear hormone receptor that regulates bile acid, lipid and glucose homeostasis. The FXR program is designed to help reduce liver steatosis and inflammation, and may help prevent liver fibrosis, Gilead and Phenex said.
Phenex has sought to develop one FXR agonist, Px-102, for NASH as well as non-alcoholic fatty liver disease. Px-102 has shown promise, significantly clearing severe steatosis in a mouse model of streptozotocin–induced NASH after 10 weeks of treatment. Treatment with Px-102 also led to reduction in lipid droplet size as well as a decrease of fibrotic structures after six weeks in a rat model.
As of Nov. 3, Phenex had completed two Phase I safety and tolerability studies for Px-102—a single ascending oral dose study over a 24-hour timeframe; and a dose-escalation study following seven days of multiple oral dosing, the company stated on ClinicalTrials.gov.
Also as of that date, Phenex was recruiting patients for a Phase I safety pilot study of Px-104 in patients with in NAFLD. The study’s primary aim is to o evaluate the safety and tolerability of Px-104 in NAFLD patients and assess the influence of Px-104 on hepatic fat, the company added on ClinicalTrials.gov.
Phenex is in preclinical phases of development for another FXR agonist, Px-103, according to its website.
“The acquisition of Phenex’s FXR program represents an important opportunity to accelerate Gilead’s efforts to develop new treatment options that address fibrotic liver diseases,” Norbert W. Bischofberger, PhD, Gilead’s evp, research and development, and CSO, said in a statement. “We look forward to working closely with Phenex’s research and development team to advance the FXR program into clinical development as quickly as possible to explore its potential in areas of significant unmet need.”
Added Phenex CEO Claus Kremoser, Ph.D.: “After 15 years of research, FXR is now one of the few clinically validated targets for NASH and we are delighted that Gilead will be continuing the research necessary to more fully realize its potential for advanced liver disease.”
Not affected by the deal is Phenex’s other R&D program—the development of Retinoid-Acid Receptor-related Orphan Receptor gamma t (RORγt) inverse agonists designed to treat autoimmune diseases, such as psoriasis, rheumatoid arthritis, and related disorders.