Pill Shows Promise for Patients With Ulcerative Colitis

A Cedars-Sinai-led clinical trial found that a once-daily oral medication significantly outperformed placebo in treating adults with moderate to severe ulcerative colitis. The results were published in The New England Journal of Medicine.

The medication, icotrokinra, blocks the effects of IL-23, an immune system protein that helps drive damaging inflammation in the intestinal lining. Unlike many advanced therapies for ulcerative colitis that are given by injection or infusion, icotrokinra is taken as a pill.

An estimated 3.1 million U.S. adults have been diagnosed with ulcerative colitis, according to the Centers for Disease Control and Prevention. The chronic, debilitating condition causes inflammation and ulcers in the lining of the large intestine, leading to frequent bloody diarrhea, abdominal pain, malnutrition, anemia and joint pain.

Conducted in 21 countries, the ANTHEM-UC Phase II-B clinical trial randomly assigned participants to receive icotrokinra or a placebo. Almost half of the patients in the study had been on advanced therapies such as biologics, while the rest were on conventional anti-inflammatory medications.

“Icotrokinra was effective even in hard-to-treat patients who had failed previous drug regimens,” said Maria T. Abreu, MD, executive director of the F. Widjaja Inflammatory Bowel Disease Institute and corresponding author of the study. “The fact that the medicine can be taken orally will help us improve the range of options we have for treating patients.”

Earlier this year, the FDA approved icotrokinra for the treatment of plaque psoriasis.

“Patients with ulcerative colitis often need to try several treatments before finding one that controls their disease,” said Gil Melmed, MD, director of Inflammatory Bowel Disease Clinical Research at Cedars-Sinai. “It’s not unusual for medications that work against one immune-related condition to also benefit patients with another. We already know that targeting the IL-23 pathway can be effective in both psoriasis and inflammatory bowel disease.”

Icotrokinra demonstrated benefits at the study’s primary 12-week assessment, with patients maintaining remission through 28 weeks of treatment.

“The drug’s favorable safety profile and precise mechanism of action could make this an important addition to our treatment options for ulcerative colitis, particularly earlier in the disease,” Abreu said. “Hopefully we can study it for Crohn’s disease as well.”

Additional authors include: Britta Siegmund, MD; Lindsey Surace, MD; Ngozi Erondu, MD, PhD; Minhu Chen, MD, PhD; Karen Chachu, MD, PhD; Edouard Louis, MD, PhD; Katsuyoshi Matsuoka, MD, PhD; Jimmy Limdi, MD; Edmund Arthur, PharmD; Nicole Houck, BS; Colleen Marano, PhD; Marion L. Vetter, MD; Bin Zou, MD; Joyce Zhan, PhD; Mary Ellen Frustaci, MAS; Arun K. Kannan, PhD; Grazyna Rydzewska, MD, PhD; Edward V. Loftus Jr., MD; and Vipul Jairath, MBChB, DPhil.

Funding: The study was supported by Johnson & Johnson.

Disclosures: Maria T. Abreu, MD, reports serving as a consultant or advisory board member for AbbVie, Inc., Amgen, Bristol Myers Squibb, Genentech, Johnson & Johnson, Lilly, Pfizer Pharmaceutical, Takeda Pharmaceuticals, and UCB Pharma.

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