BOSTON, Aug. 27, 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, today announced the presentation of the discovery of RTY-406, the company’s clinical candidate, an oral, ABCB4 /BSEP dual-targeted PFM at the American Chemical Society Fall 2026 Meeting held August 22-27, 2026 in Chicago, IL.
The presentation, titled “Discovery of RTY-406: A Positive Functional Modulator of ABCB4 and BSEP as a Disease-Modifying Therapy for the Treatment of Primary Sclerosing Cholangitis,” was presented during the Division of Medicinal Chemistry (MEDI), First Time Disclosures session by Nate Fuller, PhD, VP, Chemistry at Rectify Pharmaceuticals.
“The data presented at ACS demonstrate that RTY-406 represents an entirely novel approach for the treatment of primary sclerosing cholangitis with pipeline-in-a-pill potential across multiple hepatobiliary diseases” said Rajesh Devraj, PhD, President and Chief Executive Officer of Rectify Pharmaceuticals. “By modulating ABCB4 and BSEP to restore normal bile composition and flow, we are directly addressing key pathophysiologic drivers of hepatobiliary disease. The meaningful improvements observed in bile composition, inflammation and fibrosis in an in vivo model give us confidence in this approach and its potential to become the first disease modifying therapy for PSC.”
The presentation highlights:
- The application of Rectify’s Positive Functional Modulator (PFM) platform, which resulted in the identification of a series of dual ABCB4 / BSEP PFMs.
- The rapid optimization of this series to deliver a set of compounds with improved potency on ABCB4 and BSEP and improved drug-like properties.
- The confirmation via high resolution cryo-EM structures that this series of compounds modulates ABCB4 and BSEP function by directly binding to the target proteins.
- The identification of RTY-406, a potent, dual PFM of ABCB4 and BSEP with an excellent pharmacokinetic profile, which in a mouse model of PSC demonstrated:
- ABCB4 and BSEP target modulation
- Dose dependent decreases in ALP and cholesterol crystals which are common clinical features of PSC progression
- Dose dependent improvements in key markers of ductular reaction, inflammation and fibrosis
The abstract is available on the publications page of the Rectify website.
RTY-406 is currently being evaluated in a phase 1 clinical study.
About RTY-406
RTY-406 is an orally administered ABCB4 and BSEP dual-targeted Positive Functional Modulator (PFM) in clinical development for primary sclerosing cholangitis (PSC). The dual acting mechanism of action of RTY-406 uniquely targets abnormal bile composition and reduced bile flow, two core pathophysiological drivers of PSC. In a preclinical model of PSC, 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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