Researchers from Rectify Pharmaceuticals Inc. presented preclinical efficacy data on RTY-406, a novel dual-acting ABCB4/MDR3 and ABCB11/BSEP modulator, in animal models of primary sclerosing cholangitis.
Entering what Mirum Pharmaceuticals Inc. CEO Chris Peetz called “a new phase of growth and value creation,” the company plans to submit an NDA to the U.S. FDA in the second half of this year, based on phase IIb data from the Vistas trial with oral ileal bile acid transporter (IBAT) inhibitor volixibat in primary sclerosing cholangitis (PSC).
With the U.S. FDA’s approval of GSK plc’s ileal bile acid transporter (IBAT) inhibitor, Lynavoy (linerixibat), patients with primary biliary cholangitis no longer need off-label treatments for a debilitating internal itch symptom called cholestatic pruritus.
Increasing evidence exists regarding receptor-interacting protein kinase 1 (RIPK1), a necroptosis regulator, being involved in inflammation and fibrosis in chronic liver disorders. The relationship between necroptosis and the inhibition of RIPK1 were investigated in primary sclerosing cholangitis (PSC) in a recently published study using clinical specimens from patients.
Proqr Therapeutics NV has received clinical trial application (CTA) authorization under the EMA’s new centralized review process for a phase I study of AX-0810, which is being developed for the treatment of cholestatic diseases such as primary sclerosing cholangitis and biliary atresia.
Esperion Therapeutics Inc. has nominated ESP-2001 as a preclinical development candidate for the treatment of primary sclerosing cholangitis (PSC). The company will now begin IND-enabling studies with the aim of filing an IND application with the FDA to initiate first-in-human studies next year.
Primary sclerosing cholangitis is an autoimmune disease affecting the liver in which there is reduced expression and function of the bile acid receptor GPBAR1 in the cholangiocytes.
Rectify Pharmaceuticals Inc. has conducted preclinical testing on RTY-694, an oral dual-targeted positive functional modulator for use in the treatment of hepatobiliary disorders.
Rectify Pharmaceuticals Inc. has nominated the positive functional modulator (PFM) RTY-694 as a development candidate for primary sclerosing cholangitis (PSC). As the lead PFM in Rectify’s hepatobiliary program, RTY-694 is now advancing to first-in-human trials for PSC.
Parvus Therapeutics Inc.'s PVT-201 has received orphan drug status from the U.S. FDA for the treatment of primary biliary cholangitis (PBC), an autoimmune disease of the liver.
