Interested in licensing this patent?
MTEC can help explore whether this patent might be available for licensing for your application.
Publication Number
US-10813896-B2
Publication Date
2020-10-27
Expiration Date
Abstract
Treatment of intrahepatic cholestatic diseases by therapy with seladelpar or a salt thereof.
Core Innovation
The invention relates to treating intrahepatic cholestatic diseases using oral seladelpar or pharmaceutically acceptable salts. The biochemical cholestasis markers ALP and GGT are emphasized as endpoints for assessing treatment effects, and the document provides rationale for use in comparison with standard care approaches.
The problem being solved is inadequate treatment for intrahepatic cholestatic diseases, including primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC). The background emphasizes that existing standard care includes ursodeoxycholic acid (UDCA) and obeticholic acid (OCA), and it frames the need for improved biochemical response based on cholestasis markers.
Seladelpar is described as a PPARδ agonist, and the document summarizes clinical evidence for biochemical improvements. A Phase 2 high-dose PBC trial is described with rapid, potent ALP reductions and high responder rates, using oral dosing of seladelpar as L-lysine dihydrate salt in once-daily administration at multiple dose levels.
A PSC trial design is described using oral dosing of seladelpar and monitoring endpoints including ALP and GGT improvement. The document thus ties the core innovation to oral administration of seladelpar and salts for cholestatic disease treatment with biochemical monitoring using ALP and GGT.
Claims Coverage
The partial content identifies one independent claim, directed to a method of treating primary sclerosing cholangitis with seladelpar or a salt thereof at a specified daily dose range calculated as seladelpar. The dependent claims add oral administration, specific daily dose levels, dosing-frequency constraints, and a specific salt form.
Seladelpar for treating primary sclerosing cholangitis at a specified daily amount
A method of treating primary sclerosing cholangitis by administering a therapeutically effective amount of seladelpar or a salt thereof, where the amount is between 0.5 mg/day and 2 mg/day when calculated as seladelpar.
Oral administration
The method administers the compound orally.
Specified daily dose levels
The method administers the compound at 0.5 mg/day, 1 mg/day, or 2 mg/day.
Once per day dosing frequency
The method administers the compound once per day.
Dosing frequency range from once per week to every other day
The method administers the compound between once per week and every other day.
Seladelpar L-lysine dihydrate salt form
The method uses seladelpar L-lysine dihydrate salt.
Overall, the claim set coverage centers on administering seladelpar or salts for primary sclerosing cholangitis using a therapeutically effective amount specified as 0.5 mg/day to 2 mg/day, calculated as seladelpar, with dependent claim refinements specifying oral administration, allowable daily dose levels, dosing-frequency constraints, and an example salt form.
Stated Advantages
Rapid, potent ALP reductions in the Phase 2 high-dose PBC trial.
High responder rates based on ALP response in the Phase 2 high-dose PBC trial.
ALP decreases in the low-dose PBC trial.
Absence of transaminase/pruritus safety signals in the low-dose PBC trial.
ALP and GGT improvement as monitoring endpoints in the PSC trial design.
Documented Applications
Treatment of primary sclerosing cholangitis using oral seladelpar or pharmaceutically acceptable salts, with ALP and GGT used as monitoring endpoints in described trial design.
Treatment of primary biliary cholangitis (PBC), including summary of Phase 2 high-dose and low-dose clinical trial evaluations using oral seladelpar as L-lysine dihydrate salt and ALP-focused outcomes.
The broader document context addresses intrahepatic cholestatic diseases, including primary biliary cholangitis (PBC), primary sclerosing cholangitis (PSC), progressive familial intrahepatic cholestasis (PFIC), and Alagille syndrome (AS).
Interested in licensing this patent?