Tofacitinib Meets Phase III Endpoints in Two Ulcerative Colitis Trials

Tofacitinib (Xeljans/Pfizer), an oral ulcerative colitis (UC) drug, successfully met key endpoints in two Phase III studies enrolling adult patients with moderate to severe active UC.

The results were presented at the European Crohn’s and Colitis Organization meeting held recently in Amsterdam.

In the oral clinical trials for tofacitinib in ulcerative colitis Octave 1 and Octave 2, adults were given oral tofacitinib 10 mg twice daily. In the first study, after eight weeks, 18.5% of patients (or 88 patients) were in remission compared to just 8.2% (or 10) patients.

The company reported that the results of the second study were consistent: the tofacitinib patients reached remission at a rate of 16.6% (71 patients) compared to 3.6% of placebo patients (4 patients).

“We are encouraged by the results from the Octave Induction studies, as they showed that in these trials oral tofacitinib reduced the symptoms of moderate to severe ulcerative colitis and induced remission of the disease,” study investigator Geert D’Haens, MD, PhD, explained in a news release. “What’s more, these results were seen in patients who were previously treated with tumor necrosis factor inhibitors (TNFis) as well as those who had not received treatment with TNFis. And, improvements in symptoms were observed as early as week 2, the first time point it was measured.”

The researchers also reported that more tofacitinib patients receiving 10 mg twice daily achieved mucosal healing by week eight compared to the placebo patients — a secondary endpoint in both of the two Octave studies.

In the first study, about a third of the tofacitinib 10 mg twice daily patients achieved mucosal healing compared to 15% of the placebo patients by week eight. In the second study, 28% of tofacitinib patients reached mucosal healing compared to 11% of the placebo patients.

The adverse and serious adverse events reported by the researchers were similar in both Octave studies.

For the first study, tofacitinib patients saw adverse and serious adverse events at rates of 56.5 and 3.4%, respectively, while placebo patients in that study saw adverse and serious adverse events at rates of 59.8 and 4.1%, respectively.

In the second study, tofacitinib patients saw adverse and serious adverse events at rates of 54.1% and 4.2%, respectively, while the placebo patients in that study saw adverse and serious adverse events at rates of 52.7 and 8%, respectively.

The most common adverse event that led to discontinuation was UC flare, but discontinuation rates were under 5% among the tofacitinib patients in both studies.