RTY-406 / Rectify Pharma - LARVOL DELTA

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 (AASLD 2026) - "RTY-406, a first-inclass orally active dual PFM for ABCB4/BSEP, reduced clinically relevant markers of cholestasis, cholangitis, and fibrosis in a PSC mouse model, and demonstrated translational evidence of target modulation and proof of mechanism in non-human primates. These data support the advancement of RTY406 into the clinic as a disease modifying therapy for PSC and related hepatobiliary diseases.1. Bell et al, Liver Int Comm 2026 DOI: 10.1002/lci2.700402."

Preclinical • Cholestasis • Fibrosis • Gastrointestinal Disorder • Hepatology • Immunology • Inflammation • Primary Sclerosing Cholangitis • ABCB1 • ABCB4 • CXCL1 • CXCL8 • SPP1 • TIMP1

Discovery of RTY-406, a positive functional modulator of ABCB4 and BSEP (ABCB11) as a disease-modifying therapy for the treatment of primary sclerosing cholangitis (ACS-Fall 2026) - "In an in vivo mouse model of PSC, RTY-406 reduced cholestasis, cholelithiasis, cholangitis, ductular reaction, and fibrosis. RTY-406 has advanced to Phase 1 clinical trials as a potential new treatment for PSC."

Biliary Cancer • Cholangiocarcinoma • Cholestasis • Fibrosis • Gastroenterology • Hepatology • Immunology • Liver Cirrhosis • Liver Failure • Solid Tumor • ABCB1 • ABCB4

RTY-406 is a dual-acting ABCB4/MDR3 and ABCB11/BSEP positive functional modulator that demonstrates efficacy in a model of primary sclerosing cholangitis (EASL 2026) - "RTY-406, a first-in-class, orally active dual PFM for ABCB4/BSEP, was characterized in a mixed genetic/diet induced model of PSC. By enhancing the function of Abcb4 and Bsep, RTY-406 conferred a beneficial impact on relevant markers of cholestasis and biliary health. These data demonstrate the potential of our dual BSEP/ABCB4 PFM to provide a benefit for hepatobiliary diseases such as PSC."

Clinical • Cholestasis • Fibrosis • Gastrointestinal Disorder • Hepatology • Immunology • ABCB1 • ABCB4

RTY-406, a candidate drug for primary sclerosing cholangitis (PSC), a dual-acting ABCB4/MDR3 and ABCB11/BSEP positive functional modulator, demonstrates target modulation in non-human primates (EASL 2026) - "These 14- and 28-day studies in Cynomolgus monkeys establish a translatable biomarker profile for RTY- 406. Use of bile acid synthesis markers combined with ALP, GGT, ALT, and AST provides a robust panel for parallel monitoring of target modulation and hepatobiliary health. These data de-risk translation to human studies, offering a specific set of serum biomarkers that further support clinical development of RTY-406 for PSC."

Cholestasis • Hepatology • ABCB1 • ABCB4