Pfizer Inc.’s experimental rheumatoid arthritis pill tofacitinib eased symptoms of patients with ulcerative colitis, an inflammatory bowel disease that affects 750,000 Americans, a company-sponsored study showed.
Among 194 adults with moderate to severe ulcerative colitis, tofacitinib reduced symptoms such as pain and bleeding in 78 percent of patients given the trial’s highest dose, according to results published in the New England Journal of Medicine. The study was from the second of three stages of human tests needed for regulatory approval.
Ulcerative colitis often starts when patients are in their teens or 20s and causes diarrhea, bloody stool, weight loss and abdominal pain, according to the U.S. National Institutes of Health. About a third of patients don’t respond to two injectable immune-modulating drugs used to treat patients today, said Miguel Regueiro, co-director of the Inflammatory Bowel Disease Center at the University of Pittsburgh School of Medicine.
“These are very exciting data, this would clearly make an impact on treatment,” Regueiro said in a telephone interview.
Pfizer, based in New York, has moved tofacitinib into the final stage of clinical testing for ulcerative colitis and is awaiting a decision by U.S. regulators whether to approve the drug for use in rheumatoid arthritis.
Pfizer shares fell less than 1 percent to $24.02 at the close in New York.
In the study, 78 percent of 49 patients given a 15 milligram dose experienced a reduction in symptoms; 61 percent of 33 patients given a 10 milligram dose improved.
Patients in the trial got Pfizer’s pill for eight weeks, though likely would take it for longer because ulcerative colitis is a chronic disease, said William Sandborn, chief of the division of gastroenterology at the University of California, San Diego, who led the research.
“If it works in induction, it would be reasonable to test it as a longer-term maintenance therapy,” Sandborn said. “We’re anxious to see what the longer-term treatment profile looks like.”
Pfizer is trying to get the drug approved for patients who fail on other therapies, Victoria Davis, a spokeswoman for the company, said in an e-mail. In the study, patients also got doses of 0.5 milligram and 3 milligrams.
Current patients first get mesalamine, an anti-inflammatory drug. For the half of patients for whom the medicine doesn’t work, they can go on injectable immunosuppressant therapies such as Johnson & Johnson’s Remicade, or the steroid prednisone, said Sandborn. Abbott Laboratories, based in Abbott Park, Illinois, is also studying its injection Humira for use in the disease.
Pfizer’s drug doesn’t work through the same biological pathway as the Abbott and J&J medications.
“Having a different mechanism of action is hugely important,” Regueiro said. It would also let patients take the drug in pill form, instead of by injection or infusion. That would help patients keep on their medicine, he said.
Pfizer said it has started three larger, longer phase III studies that will better examine the drug’s safety and side effects at the higher dosages. In rheumatoid arthritis patients, immunosuppressant medicines can cause infections or cancers. Those patients, though, are often older and have other medical conditions, Sandborn said. Younger patients may be able to tolerate a higher dose, he said.
The most common side effects related to infection reported in the ulcerative colitis study included influenza and cold-like symptoms. There was also a dose-dependent increase in LDL and HDL cholesterol that reversed after patients stopped taking the drug. The size of the trial, however, wasn’t large enough to allow for a comprehensive assessment of the drug’s safety and tolerability, researchers said in the study.
Pfizer is still recruiting patients for the phase III trials, and doesn’t have a date on when the studies will be complete or when it might submit to the U.S. Food and Drug Administration to get the drug approved for ulcerative colitis, said Pfizer’s Davis.