Upadacitinib Meets Primary End Point of Phase 2 Trial for Vitiligo and Advances to Phase 3

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AbbVie recently announced that its phase 2b study investigating upadacitinib (Rinvoq) for the treatment of adults with nonsegmental vitiligo met the primary end point of percent change from baseline in the Facial Vitiligo Area Scoring Index (F-VASI) at week 24 with the 11 mg and 22 mg doses versus placebo. Overall, the percent reduction from baseline in F-VASI at week 52 was numerically greater than results at week 24 for all upadacitinib doses, and no new safety signals were found.AbbVie announced at the European Academy of Dermatology and Venerology (EADV) Congress in Berlin, Germany, that based on this data, it will be advancing its clinical trial program of upadacitinib for vitiligo to phase 3.¹

Key findings from the data include:

• At week 24, upadacitinib achieved the primary end point of percent change from baseline in F-VASI with 11 mg and 22 mg doses versus placebo
• Higher response rates were observed with upadacitinib versus placebo in secondary end points, including F-VASI 75 at week 24 with the 11 mg and 22 mg doses and Total Vitiligo Area Scoring Index (T-VASI) 50 at week 24 with the 22 mg dose
• The mean percent reduction from baseline in F-VASI was numerically greater at week 52 than results at week 24 for all upadacitinib dose groups
• Response rates observed for F-VASI 75 and T-VASI 50 at week 52 were numerically greater than those at week 24 for all upadacitinib dose groups

"There is a high unmet need in vitiligo, with no systemic treatment options approved, leaving patients frustrated in seeking options for re-pigmentation of the skin," said Roopal Thakkar, MD, senior vice president of development and regulatory affairs and chief medical officer at AbbVie, in the news release. "We will continue to apply our significant experience in advancing research and driving innovation in treatments for immune-mediated diseases, including underserved diseases with high burden on patients, such as vitiligo."

Treatment-emergent adverse event (TEAE) rates were similar with upadacitinib and placebo in period 1. The most common TEAEs included COVID-19, acne, headache, and nasopharyngitis. Numerically higher rates of serious TEAEs and TEAEs leading to study drug discontinuation were observed in the upadacitinib 22 mg group versus the other groups.

According to the news release, “One death adjudicated as undetermined/unknown cause and deemed by the investigator to have no reasonable possibility of being related to study drug occurred in the upadacitinib 22 mg group (period 1). One adjudicated event of nonfatal ischemic stroke occurred with upadacitinib 11 mg (period 2) in a patient with known cardiovascular risk factors. One event of malignancy (breast cancer) occurred with upadacitinib 11 mg (period 2) in a patient with positive family history of breast cancer.”

During the study, one event of a serious infection, COVID-19 pneumonia, was reported in the upadacitinib 22 mg group. There were no adjudicated events of venous thromboembolism, gastrointestinal perforation, or active tuberculosis.

"Vitiligo impacts millions of people globally, and there is no cure. The disease can have a great impact on patients' physical and mental health, as depigmentation of the skin can be severe," said Thierry Passeron, MD, PhD, professor and chair of the department of dermatology at the Université Côte d'Azur, in the news release. "In vitiligo, it can take time to see optimal skin re-pigmentation during treatment, which makes long-term studies critical to providing valuable insights on a treatment's meaningful impact for patients."

Reference

1. AbbVie announced upadacitinib (Rinvoq) met the primary endpoint in phase 2 clinical trial of vitiligo as program advances to phase 3. AbbVie. News release. October 11, 2023. Accessed October 12, 2023. https://news.abbvie.com/article_display.cfm?article_id=12620#:~:text=NORTH%20CHICAGO%2C%20Ill.%2C%20Oct,week%2024%20with%20the%2011