CymaBay Therapeutics Achieves Liver Breakthrough: Seladelpar Hits Bullseye in Phase III Trial
In a groundbreaking development, CymaBay Therapeutics announced a stunning victory for its liver disease champion, seladelpar, as it conquered both primary and secondary endpoints in the Phase III RESPONSE trial. This clinical-stage biopharma’s quest was to combat primary biliary cholangitis (PBC), a condition that relentlessly erodes the liver’s bile ducts.
Picture a year-long, double-blind odyssey featuring 193 PBC patients who had either faced the shortcomings or suffered the side effects of the standard PBC treatment, ursodeoxycholic acid. The primary composite endpoint starred serum alkaline phosphatase (ALP), a critical marker for liver damage and bone disorders.
In an electrifying revelation, CymaBay declared that an astonishing 61.7% of patients triumphed in reaching this milestone, leaving the mere 20% of placebo takers in the dust. But that wasn’t all! The drama unfolded further as the study gracefully achieved a second primary endpoint related to ALP normalization and another crucially linked to itching.
As the applause reverberated, seladelpar also clinched safety accolades, with CymaBay proudly announcing, “no treatment-related serious adverse events in the study.” They went on to describe seladelpar’s “favorable and consistent” tolerability profile, a testament to its potential.
CymaBay’s CEO, Sujal Shah, chimed in with optimism, noting that many PBC patients grapple with relentless itching and the looming threat of a life-altering liver transplant. He expressed hope that these groundbreaking results might revolutionize the treatment landscape for these patients.
Gideon Hirschfield, a leading figure in autoimmune liver disease research, joined the chorus of praise, emphasizing the “potential for an efficacious and safe new therapy” that not only improves liver health but, for a substantial portion of patients, normalizes these critical measures. Hirschfield noted that seladelpar’s impact on itch relief could transform the quality of life for countless PBC patients.
The grand finale: CymaBay hinted at regulatory discussions and plans to seek approval from the U.S. Food and Drug Administration, the Medicines and Healthcare products Regulatory Agency, and the European Medicines Agency, leveraging the stellar Phase III RESPONSE trial results.
Despite the monumental news, investors initially played it cool, with share prices showing a modest uptick of just under 8% in premarket trading. However, market analysts at William Blair were brimming with enthusiasm, dubbing the results a “hat trick.” They anticipate seladelpar emerging as the top-tier second-line agent in PBC treatment, urging investors to seize the opportunity.
With the ongoing Phase III IDEAL study on the horizon, seladelpar may soon reshape the treatment landscape for a broader spectrum of PBC patients, ensuring this captivating medical journey is far from over.