Massachusetts-based Rectify Pharmaceuticals has selected a lead positive functional modulator (PFM) for its hepatobiliary program and is advancing to first-in-human clinical trials for primary sclerosing cholangitis (PSC).
PSC is a debilitating orphan bile duct disease characterized by inflammation, fibrosis, and narrowing of the medium and large intra and extra-hepatic bile ducts that leads to inflamed bile ducts, the reduction or stoppage of bile flow and ultimately, liver failure. There are currently no approved medications for the treatment of PSC.
Rectify's lead candidate, RTY-694, is an orally acting dual-targeted PFM that addresses the core pathophysiology of PSC disease progression by increasing the function of the ABCB4 and BSEP transporters — two membrane proteins that play a critical role in maintaining bile composition and bile flow.
In translational mouse models, RTY-694 demonstrated improvements in bile duct health, inflammation and fibrosis.
Launched in 2021 with a $100M Series A round led by Atlas Venture, Rectify is developing a pipeline of PFMs to restore ABC transporter function for patients with serious genetic diseases. ABC transporters are a 48-member family of membrane-bound proteins with etiologic loss-of-function mutations in multiple organ systems and therapeutic areas including the lungs, liver, gastrointestinal tract, eye, and central nervous system.