Gilead's $4.3B Liver Drug Effective Up to 2 Years

Gilead Sciences is advancing its liver disease treatment, seladelpar, with the anticipation of an FDA decision by mid-August. The pharmaceutical giant had previously invested $4.3 billion to acquire CymaBay Therapeutics and its promising liver disease drug, seladelpar, which had already shown significant potential in a phase 3 clinical trial.

Seladelpar has been under investigation for its efficacy in treating primary biliary cholangitis (PBC), a chronic liver disease. Gilead has bolstered the case for seladelpar with additional data from various studies, including an open-label trial and a long-term analysis of patient outcomes over a two-year period. The findings were presented at the European Association for the Study of the Liver Congress, providing robust evidence for the drug's durability and efficacy.

The comprehensive dataset combined participants from both phase 3 and earlier trials, totaling more than 300 PBC patients. Among the 99 patients who were treated with seladelpar for 24 months, 70% met the composite response endpoint, which evaluates multiple liver health markers. Additionally, 42% of these patients achieved normalization of alkaline phosphatase (ALP), a key biomarker for liver damage, after 24 months. Similar positive results were observed in 164 patients who completed 12 months of treatment in earlier studies.

One of the notable benefits of seladelpar is its impact on pruritus, or severe itching, a common and debilitating symptom of PBC. The analysis showed that the mean reduction in itch severity slightly decreased from 3.8 points at 12 months to 3.1 points at 24 months. Despite this slight decline, the data still indicate that seladelpar can effectively alleviate itching, a symptom that current treatments struggle to manage effectively. Gilead considers the drug’s impact on pruritus as a significant differentiator from other treatments.

Johanna Mercier, Gilead’s Chief Commercial Officer, emphasized the importance of addressing pruritus in improving patients' quality of life. "This keeps you up at night. It impacts mental health. It impacts how you work. Basically, you want to scratch your skin off,” she remarked at an RBC Capital Markets event in May. Mercier highlighted the potential of seladelpar to make a substantial difference for patients suffering from this severe symptom.

Should the FDA approve seladelpar, it is expected to receive a second-line treatment label specifically for ALP normalization. This would position seladelpar as a treatment option for patients who do not adequately respond to ursodeoxycholic acid, the currently available therapy.

In summary, Gilead Sciences is on the brink of potentially launching a transformative treatment for primary biliary cholangitis with seladelpar. The drug has shown promising long-term results in liver health improvement and pruritus reduction, paving the way for its possible approval and subsequent impact on the lives of PBC patients. The upcoming FDA decision will be crucial in determining the future availability of this innovative therapy.