Company says dual-acting oral therapy improves bile acid transport and reduces inflammation in preclinical studies.

Rectify Pharmaceuticals has presented new preclinical data on its lead candidate RTY-694, a dual-targeted positive functional modulator (PFM), suggesting potential benefits for patients with hepatobiliary diseases including primary sclerosing cholangitis (PSC).

The findings were shared at the EASL 2025 Congress in Amsterdam and selected for the event’s ‘Best of EASL Congress’ highlights, the company said.

The company says RTY-694 is designed to enhance the function of ABCB4 and BSEP, two membrane transport proteins involved in bile flow. According to Rectify, the compound demonstrated a reduction in markers of cholestasis and liver inflammation across two disease models — one mimicking PFIC2 and another replicating PSC features.

“Patients living with hepatobiliary diseases face limited treatment options, most of which fail to address the underlying mechanisms driving biliary pathophysiology,” said Rajesh Devraj, PhD, President and CEO of Rectify. “These results further strengthen the promise of our dual-targeted PFM, and we remain focused on swiftly driving this candidate toward clinical evaluation.”

In a PFIC2 mouse model, treatment with RTY-694 reportedly led to lower serum bile acid levels and reduced markers of inflammation. In a second model replicating PSC features, the candidate improved bile phospholipid content and decreased markers of cholangitis and fibrosis, the company said.

“These encouraging translational results build on our work to address pathology at the core of many liver and biliary diseases,” said Pol Boudes,chief medical officer.

“With demonstrated on-target activity and clear proof of biology, we’re excited to advance rapidly toward the clinic.”

The findings were presented by Eric Bell, during an abstract session focused on immune-mediated and cholestatic diseases.