Pfizer Inc. announced top-line results from Oral Clinical Trials for tofAcitinib in ulceratiVE colitis (OCTAVE) Sustain, the third phase 3 study of Xeljanz (tofacitinib citrate) being investigated in patients with moderately to severely active ulcerative colitis (UC). OCTAVE Sustain is a 52 week study that evaluated oral tofacitinib 5 mg and 10 mg twice daily (BID) as a maintenance treatment in adult patients with moderately to severely active UC who previously completed and achieved clinical response in either the OCTAVE Induction 1 or OCTAVE Induction 2 studies.

Top-line results from the OCTAVE Sustain study showed that the proportion of patients in remission at week 52, the primary efficacy endpoint, was significantly greater in both the tofacitinib 5 and 10 mg BID groups compared to placebo. In OCTAVE Sustain, remission was defined as total Mayo scorea =2, no subscore >1, and rectal bleeding subscore of 0. No new or unexpected safety findings for tofacitinib were observed in the study.

“Ulcerative colitis is a chronic, often debilitating inflammatory condition that can be difficult to treat. There are a limited number of therapies available and patients need additional treatment options” said Michael Corbo, PhD, chief development officer, Inflammation & Immunology, Pfizer, Inc. “These findings, along with the previously released positive induction data from the OCTAVE studies, are encouraging and provide evidence that tofacitinib, if approved, has the potential to be an effective new oral treatment option that both induces and maintains remission. We are proud to advance our clinical development program for tofacitinib as we work to bring a potential new treatment option to patients living with ulcerative colitis.”

OCTAVE Sustain is a phase 3, randomized, double-blind, placebo-controlled, parallel group, multi-center study. A total of 593 patients were randomized to tofacitinib 5 mg BID, tofacitinib 10 mg BID and placebo BID. Detailed analyses of OCTAVE Sustain, including additional efficacy and safety data, will be submitted for presentation at a future scientific meeting.

The OCTAVE global clinical development program includes three phase 3 studies, OCTAVE Induction 1, OCTAVE Induction 2, and OCTAVE Sustain, as well as a long-term extension trial, OCTAVE Open. We expect that these four studies will form the submission package to regulatory authorities for a potential UC indication.

OCTAVE Induction 1 and OCTAVE Induction 2 are two replicate Phase 3 placebo-controlled studies that evaluated induction of remission by oral tofacitinib 10 mg BID in adult patients with moderately to severely active UC. Subjects needed to have failed or been intolerant to UC treatments including corticosteroids, thiopurines or tumor necrosis factor inhibitors (TNFi).

OCTAVE Sustain is a phase 3 placebo-controlled study evaluating oral tofacitinib 5 mg and 10 mg BID as maintenance therapy in adult patients with moderately to severely active UC.

OCTAVE Open is an ongoing open-label extension study designed to assess the safety and tolerability of tofacitinib 5 mg and 10 mg BID in patients who have completed or who have had treatment failure in OCTAVE Sustain or who were non-responders upon completing OCTAVE Induction 1 or 2.

UC is a chronic, often debilitating inflammatory bowel disease that affects millions of people worldwide. It is believed that UC is the result of complex interactions between multiple factors that include the environment, genetic predisposition, immune response, and the gut microbiome in the colon or intestines. It can cause abdominal pain, fever, weight loss and chronic, bloody diarrhea.UC can have an effect on work, family and social activities. In up to one-third of patients with UC, treatment is not completely successful or complications arise. Under these circumstances, surgery to remove the colon (colectomy) may be considered. Even after surgery, certain symptoms of UC may still persist.

Xeljanz/Xeljanz XR (tofacitinib citrate) is a prescription medicine called a Janus kinase (JAK) inhibitor. In the United States, Xeljanz XR 11 mg QD is the first and only once-daily oral JAK inhibitor approved for the treatment of moderate to severe rheumatoid arthritis (RA).

As the developer of Xeljanz/Xeljanz XR, Pfizer is a leader in JAK innovation. Xeljanz is approved in more than 45 countries around the world for the treatment of moderate to severe RA as a second-line therapy after failure of one or more disease-modifying antirheumatic drugs (DMARDs).

Pfizer is committed to advancing the science of JAK inhibition and enhancing understanding of Xeljanz through a robust clinical development program. The efficacy and safety profile of Xeljanz has been studied in approximately 6,200 patients with moderate to severe RA, amounting to more than 19,400 patient-years of drug exposure in the global clinical development program.

Xeljanz (tofacitinib citrate)/Xeljanz XR (tofacitinib citrate) extended-release is a prescription medicine called a Janus kinase (JAK) inhibitor. Xeljanz/Xeljanz XR is used to treat adults with moderately to severely active rheumatoid arthritis in which methotrexate did not work well. Xeljanz/Xeljanz XR may be used as a single agent or in combination with methotrexate (MTX) or other non-biologic disease-modifying antirheumatic drugs (DMARDs). Use of Xeljanz/Xeljanz XR in combination with biologic DMARDs or potent immunosuppressants, such as azathioprine and cyclosporine, is not recommended.

It is not known if Xeljanz/Xeljanz XR is safe and effective in people with hepatitis B or C. Xeljanz/Xeljanz XR is not for people with severe liver problems. It is not known if Xeljanz/Xeljanz XR is safe and effective in children.