Seladelpar

The PBC drug market sees two new FDA-approved therapies, Iqirvo and Livdelzi, alongside an FDA advisory committee's unfavorable vote on Ocaliva's long-term safety and efficacy.

The European Commission approves Ipsen's IQIRVO (elafibranor) for primary biliary cholangitis, the first new therapy in the EU in a decade. Other companies, including CymaBay, Zydus, and GlaxoSmithKline, are preparing to enter the market. The approval is based on the Phase III ELATIVE trial, showing a 47% clinical benefit of IQIRVO combined with UDCA. Additionally, Gilead Sciences' LIVDELZI (seladelpar) received FDA accelerated approval for PBC.

COUR Pharmaceuticals' phase 2a study of CNP-104 for primary biliary cholangitis (PBC) shows potential as a disease-modifying treatment, addressing the root cause of PBC by inducing tolerance to pathogenic T-cells, with positive results in safety, tolerability, and efficacy.

Biotech sector growth driven by personalized medicine, FDA approvals, and Generative AI in drug discovery. Illumina, Alnylam, and Gilead Sciences highlighted for potential returns due to robust pipelines and recent FDA approvals. Market optimism and economic value projections contribute to biotech's bright future.

After a July surge in FDA approvals for gastroenterology, including the first blood test for colorectal cancer, the agency saw a summer lull with only three GI approvals. Notable approvals included Skyrizi for UC and Crohn’s, a rapid HCV test for point-of-care use, and a third ustekinumab biosimilar.

FDA grants Gilead Sciences' Livdelzi (seladelpar) accelerated approval for treating PBC in adults intolerant or unresponsive to UDCA. Livdelzi, a PPAR delta agonist, showed significant improvement in liver disease markers and reduced pruritus in the RESPONSE study, with potential for continued approval pending further trials.

FDA advisors will assess obeticholic acid's (Ocaliva) clinical benefit in PBC, despite null post-approval evidence. Intercept Pharmaceuticals seeks an indication for reducing hepatic decompensation, liver transplant, and death in PBC patients with compensated cirrhosis, using the drug with or without UDCA. FDA reviewers doubt the drug's efficacy for PBC patients intolerant or unresponsive to UDCA, citing failed postmarketing trials. The advisory committee will vote on whether the drug's benefits outweigh risks in non-contraindicated PBC patients.

Livdelzi® (seladelpar), a PPARδ agonist, treats primary biliary cholangitis (PBC) in adults, either with UDCA or as a monotherapy for UDCA-intolerant patients. Gilead Sciences acquired CymaBay Therapeutics, expanding its liver portfolio with seladelpar in 2024. The FDA granted accelerated approval in August 2024, based on the Phase III RESPONSE study, showing 62% of Livdelzi recipients achieved the primary endpoint of composite biochemical response, compared to 20% on placebo. Livdelzi also normalized ALP levels and reduced pruritus significantly. The drug is available in 10mg capsules and is under review by the EMA and UK MHRA.

Gilead Sciences discusses its focus areas in virology, oncology, and inflammation/immunology, highlighting successful data in HIV prevention with lenacapavir. The company also mentions progress in its liver franchise and oncology portfolio, including the potential launch of a BCMA cell therapy. Gilead expects growth driven by new product launches and anticipates sharing more data later this year.

Avenzo Therapeutics and Gilead Sciences partner to assess a combination treatment regimen of AVZO-021 (CDK2 inhibitor) and Trodelvy (Trop-2 ADC) for HR+, HER2- breast cancer. Gilead supplies Trodelvy, while Avenzo conducts and sponsors the study, maintaining all rights for their assets.