‘Encouraging’ ELATIVE data for elafibranor may spark race for primary biliary cholangitis
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Key takeaways:
- More than half of patients with primary biliary cholangitis who received elafibranor 80 mg achieved a cholestasis response.
- Topline ELATIVE trial results may trigger race to PBC market between Intercept and Ipsen/Genfit.
Topline findings from the phase 3 ELATIVE trial showed that elafibranor improved cholestasis response in patients with primary biliary cholangitis, Ipsen and Genfit announced.
Elafibranor, an investigational dual peroxisome activated receptor agonist, was granted breakthrough therapy designation by the FDA in 2019 for adults with primary biliary cholangitis disease who are intolerant, or have had an inadequate response, to ursodeoxycholic acid (UDCA).
Given the recent setback for rival Intercept’s Ocaliva in pre-cirrhotic liver fibrosis due to nonalcoholic steatohepatitis, both Intercept and Ipsen/Genfit have redoubled their efforts toward a second-line treatment for PBC; this phase 3 data could position elafibranor as a strong competitor in the race to market.
“These are encouraging results that suggest elafibranor could be an effective treatment to prevent progression of PBC in patients who have received UDCA,” Howard Mayer, executive vice president and head of research and development for Ipsen, said in a press release. “It has a good safety profile and was well-tolerated, and could provide an important new therapeutic option for long-term treatment of patients with this debilitating condition.”
He added: “We are excited about the potential of this investigational treatment and Ipsen now intends to discuss these results with regulatory agencies and plans to move forward with regulatory submissions to the U.S. Food and Drug Administration and the European Medicines Agency.”
In the ELATIVE phase 3 trial, a 52-week double-blind, placebo-controlled trial, patients with PBC (n=161) were randomized 2:1 to receive either elafibranor 80 mg once daily or placebo. Patients who exhibited an inadequate response to UDCA would continue to receive UDCA in combination with elafibranor or placebo, while patients unable to tolerate UDCA would receive only elafibranor or placebo.
According to topline results, the trial met its primary endpoint, with 51% of patients who received elafibranor 80 mg achieving a cholestasis response vs. 4% of patients who received placebo (P <.0001). The researchers defined cholestasis response as alkaline phosphatase (ALP) <1.67 x upper limit of normal (ULN), an ALP decrease 15% and total bilirubin ULN at 52 weeks.
The researchers also determined that the trial met its secondary endpoint – normalization of ALP at 52 weeks — with statistically significant improvements for elafibranor vs. placebo. In addition, the researchers noted “a trend for pruritus improvement” with a greater reduction in PBC Worst Itch numerical rating scale score for patients who received elafaibranor; however, this did not reach statistical significance.
Elafaibranor demonstrated a safety and tolerability profile that is consistent with previous studies, according to researchers.
“We are pleased by these results because PBC remains a disease where significant unmet medical needs exist,” Pascal Prigent, CEO of Genfit, said in the release. “This long-awaited trial outcome is therefore good news for patients and for health care professionals who need more options to improve the clinical management of patients with PBC. It is also a gratifying recognition of the quality of our team’s work and of Genfit’s ability to innovate and deliver tangible results.”
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