“We don’t think pruritus is an issue with norUDCA,” Dr. Trauner said in response to a delegate’s query on the numerically higher rates of itching in the active treatment groups. “Of course, we paid much attention to this, but overall pruritus was extremely mild, it led to no discontinuation of the study drug.” There is an ongoing, similar-dose study in nonalcoholic fatty liver disease where so far 150 patients have been treated and only a single patient so far has reported this side effect.
Based on these “highly encouraging” results, “the planning of the phase III trial in PSC is already ongoing,” Dr. Trauner said. Asked what the primary endpoint may be, he acknowledged that change in serum ALP was not an accepted endpoint in PSC by itself and that the trial would most likely use a combined endpoint of serum ALP and assessment of fibrosis, perhaps noninvasively with FibroScan.
He also noted that quality of life had been assessed in the phase II trial but no significant differences were seen. It could be that, at 12 weeks, the trial was too short to assess this parameter, he suggested, and that would be perhaps something to also address in the phase III trial. There was no change in IBD disease activity, which was important, he said.
Dr. Heiner Wedemeyer of Hannover (Germany) Medical School and who was not involved in the trial also commented on the importance of the study at the press briefing. Dr. Wedemeyer said that he was “extremely excited” by these data. “This is the most awful liver disease that we have because it is so difficult to judge when to transplant the patient,” he noted. This is a very difficult group of patients to handle and “this is the first time in decades that there is some hope on the horizon. This is the first time we see something that may work.”
Dr. Falk Pharma, Frieburg, Germany, sponsored the trial. Dr. Trauner has received honoraria, research grants, and travel support from Dr. Falk Pharma, and is listed as co-inventor on a patent for the medical use of norUDCA. Dr. Trauner has also received honoraria, research grants, or travel support from Albireo, Genfit, Gilead, Intercept, Merck Sharp & Dohme, Novartis, Phenex, and Roche. Dr. Tacke and Dr. Wedemeyer had no relevant disclosures.
