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Oral dual-targeted ABCB4/BSEP positive functional modulator (PFM) improved cholestatic and cholangitis markers in orthogonal models of hepatobiliary disease
Data reinforce the potential of this differentiated mechanism of action (MOA) to address the core pathophysiological drivers of multiple hepatobiliary diseases including primary sclerosing cholangitis (PSC)
CAMBRIDGE, Mass., May 09, 2025 (GLOBE NEWSWIRE) -- Rectify Pharmaceuticals, Inc., ("Rectify”) a biotechnology company pioneering positive functional modulators (PFMs) that restore and enhance membrane protein function, today announced the presentation of preclinical proof of concept data from its hepatobiliary program clinical candidate during the European Association for the Study of the Liver (EASL) 2025 Congress, taking place in Amsterdam, The Netherlands, May 7 - 10, 2025. The presentation was recognized by the EASL committee as a noteworthy contribution to the Congress's scientific program and will be featured in a 'Best of EASL Congress” slide deck.
"Patients living with hepatobiliary diseases face limited treatment options, most of which fail to address the underlying mechanisms driving biliary pathophysiology,” said Rajesh Devraj, Ph.D., President and Chief Executive Officer of Rectify. "PFMs represent a new class of oral therapeutics with the potential to restore and enhance the function of membrane proteins and modify the course of disease. These preclinical results further strengthen the promise of our lead ABCB4/BSEP dual-targeted PFM to improve biliary and liver health, and we remain focused on swiftly driving this candidate toward clinical evaluation to help patients with hepatobiliary diseases including PSC."
Title: Novel dual-acting ABCB4/MDR3 and ABCB11/BSEP positive functional modulator demonstrates anti-cholestatic and anti-cholangitis activity in two orthogonal models of hepatobiliary disease
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Abstract Number: 842
Presenter: Eric Bell, Ph.D.
Session: Abstract Session - Immune-mediated & Cholestatic Diseases
Date and Time: Friday, May 9, 2025, 8:30 a.m. - 9:45 a.m. CEST
Key findings
- RTY-694, a dual-targeted ABCB4/BSEP PFM, increased both ABCB4 and BSEP protein levels and augmented efflux of their respective substrates, phospholipids and bile acids, in vitro
- In the BsepE297G mouse model that recapitulates PFIC2, therapeutic treatment with RTY-694 for two weeks resulted in:
- A reduction in serum bile acid levels and an increase in biliary bile acid levels, supporting RTY-694's mechanism of action
- Reduced markers of cholestasis (ALP) and inflammation (ALT)
- Reduced total liver weight, an aggregate marker of overall liver health
- In Abcb4+/- mice on a lithogenic diet, a novel model of biliary disease that recapitulates hallmarks of PSC, administration of RTY-694 resulted in:
- Increased biliary phospholipids
- Decreased serum markers of cholestasis (serum bile acids and ALP)
- Reduced markers of ductular reaction (CK19) and cholangitis (CXCL-1 and CD11b)
- Decreased markers of liver fibrosis
Pol Boudes, M.D., Chief Medical Officer of Rectify, added, "These encouraging translational results build on our pioneering work aimed at addressing pathology at the core of many liver and biliary diseases. With demonstrated on-target activity and clear proof of biology, we are excited to advance rapidly towards the clinic with the goal of offering a potentially transformative option to patients suffering from serious hepatobiliary diseases, starting with PSC.”
The presentation will be available on the Rectify website at https://rectifypharma.com/publications/.
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. Rectify was founded and seeded by Atlas Venture who co-led the $100M Series A round with Omega Funds and were joined by Forbion and Longwood Fund.
For more information, please visit www.rectifypharma.com or follow us on X and LinkedIn.
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