Gilead, Ipsen Tout Phase III Primary Biliary Cholangitis Data as PDUFA Dates Loom
临床结果并购临床3期
Pictured: 3D image of liver damage/iStock, Mohammed Haneefa Nizamudeen
Gilead Sciences announced Wednesday that its drug candidate seladelpar has shown improvement in a late-stage trial of patients with the rare liver disease primary biliary cholangitis. However, rival Ipsen Biopharmaceuticals could beat Gilead to the U.S. market.
Data from the Phase III ASSURE trial in primary biliary cholangitis (PBC), showed “rapid and sustained” improvement in patients treated with Gilead’s seladelpar, a peroxisome proliferator-activated receptor delta (PPAR) agonist. This included high rates of normalization of liver biomarkers and a clinically meaningful reduction in pruritus, or itch.
According to Gilead, 70% of patients met the composite response endpoint, which includes having alkaline phosphatase (ALP) levels below 1.67x the upper limit of normal (ULN), as well as a decrease in ALP levels of at least 15% and total bilirubin levels at or below the ULN.
In addition, 42% of patients reached ALP normalization at 24 months, a marker of liver disease progression. Patients had also reached “sustained improvement” in pruritus, with a mean reduction of 3.8 and 3.1 points on the numerical rating scale at 12 months and 24 months, respectively.
“The long-term efficacy and safety interim results from ASSURE demonstrate that seladelpar may meaningfully raise the bar in PBC. Seladelpar can help people achieve significant reduction, and in some cases, normalization of liver blood tests,” Palak Trivedi, consultant hepatologist at the University of Birmingham, said in a statement.
Seladelpar was acquired by Gilead in its $4.3 billion acquisition of CymaBay Therapeutics earlier this year. The company’s target action date with the FDA is August 14, 2024, but Ipsen’s candidate may beat Gilead to approval.
Ipsen’s elafibranor, another PPAR agonistPPAR agonist, also on Wednesday reported encouraging long-term results in a late-stage trial, as its PDUFA date approaches on June 10, 2024.
The Phase III ELATIVE trial showed that at week 52, elafibranor led to “greater reductions” in the 5-D itch score and a clinically meaningful reduction in the itch domain of PBC-40, compared to placebo. The candidate also reported an improved quality of life in PBC patients, as measured by the severity of itching, sleep disturbance, and emotional impact of itching.
At week 52, 58% of patients on elafibranor experienced relief from itching compared to 27% on placebo, while 80% on the treatment had no sleep disturbances.
“These long-term data from the Phase III ELATIVE study further demonstrate the potential for elafibranor to provide an effective treatment option for these patients,” Sandra Silvestri, executive vice president and chief medical officer at Ipsen, said in a statement.
Ipsen nabbed elafibranor in 2021 through a strategic partnership with Genfit. It paid €120 million ($130.5 million) upfront and could potentially spend up to €360 million ($391.7 million) in milestones.
Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.
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