BOSTON, Nov. 05, 2026 (GLOBE NEWSWIRE) — Rectify Pharmaceuticals, Inc. (“Rectify”), a biotechnology company advancing Positive Functional Modulators (PFMs), a novel class of oral small molecules designed to restore and enhance membrane protein function, announce the presentation of preclinical data characterizing RTY-406, the company’s clinical dual-acting ABCB4/BSEP PFM, at The Liver Meeting®, the Annual Meeting of the American Association for the Study of Liver Diseases (AASLD) in Denver, Colorado.
The presentation, titled “Targeting bile composition in PSC: RTY-406, a dual-acting ABCB4/BSEP Positive Functional Modulator reduces disease-relevant markers in a mouse model and non-human primate” will be presented during the Industry Research Showcase on Saturday November 7th, 2026.
“Many existing treatment options for hepatobiliary diseases do not address the underlying mechanisms that drive disease progression,” said Pol Boudes, M.D., Chief Medical Officer of Rectify. “RTY-406 is designed to modulate ABCB4 and BSEP to restore bile composition and flow, with the goal of addressing the root cause of the disease. These data underscore the potential of PFMs as a new therapeutic class for patients with hepatobiliary diseases.”
Highlights from the presentation:
- By positively modulating the ABCB4 transporter, RTY-406 demonstrates anti-cholelithiasis (gallstone) activity and an improvement in biliary health
- By positively modulating the BSEP transporter, RTY-406 demonstrates anti-cholestasis activity
- By increasing the function of both protein transporters, RTY-406 demonstrates dose dependent efficacy across multiple disease relevant endpoints including ductular reaction, inflammation, and fibrosis
- RTY-406 demonstrates ABCB4/BSEP target modulation in non-human primates
“RTY-406 has true pipeline-in-a-pill potential, with a dual mechanism of action that could address multiple severe hepatobiliary diseases,” said Rajesh Devraj, Ph.D., Chief Executive Officer of Rectify. “It is currently being evaluated in Ph1 clinical trials with data from cholestatic patients anticipated in 2027.”
RTY-406 is currently being evaluated in the clinic for the potential treatment of multiple hepatobiliary diseases including primary biliary cholangitis (PBC), cholestatic patients with mutations in ABCB4, and primary sclerosing cholangitis (PSC).
About RTY-406
RTY-406 is an orally administered ABCB4 and BSEP dual-targeted Positive Functional Modulator (PFM) currently in clinical development for multiple hepatobiliary diseases. The dual acting mechanism of action of RTY-406 uniquely targets abnormal bile composition and reduced bile flow, two core pathophysiological drivers of hepatobiliary diseases and PSC. In a mouse preclinical model, RTY-406 demonstrated efficacy across multiple disease-relevant endpoints, including inflammation, cholangitis, cholestasis, and fibrosis. RTY-406 has pipeline-in-a-pill potential across multiple hepatobiliary diseases and the opportunity to become a first-in-class disease-modifying therapy for PSC.
About Rectify Pharmaceuticals, Inc. (“Rectify”)
Rectify is advancing Positive Functional Modulators (PFMs), a novel class of oral, small molecules that restore and enhance membrane protein function to address the underlying cause of serious diseases. Rectify’s PFMs have potential to modulate the activity of wild-type and mutated membrane-bound proteins, a historically difficult challenge with a small molecule approach. The Company’s breakthrough product platform enables efficient and rapid discovery of first- and best-in-class small molecule therapies with the potential to address membrane protein dysfunction for treatment of rare and common diseases, including liver, cardio-renal-metabolic, and neurodegenerative diseases. For more information, please visit www.rectifypharma.com or follow us on X and LinkedIn.
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