RINVOQ (Upadacitinib) Meets Primary Endpoint in Phase 2 Clinical Trial of Vitiligo, Program Advances to Phase 3
At week 24, upadacitinib achieved the primary endpoint of percent change from baseline in F-VASI with 11 mg and 22 mg doses versus placebo.
Abbvie's upadacitinib (RINVOQ) met the primary endpoint of percent change from baseline in Facial Vitiligo Area Scoring Index (F-VASI) at week 24 with 11 mg and 22 mg doses versus placebo in adults with non-segmental vitiligo (NSV), according to data presented at the European Academy of Dermatology and Venerology (EADV) Congress in Berlin, Germany.
No new safety signals were identified beyond the known safety profile for upadacitinib.
The use of upadacitinib in vitiligo is not approved and its safety and efficacy have not been evaluated by regulatory authorities. Based on these data, AbbVie is advancing its clinical program of upadacitinib in vitiligo to Phase 3.
At week 24, upadacitinib achieved the primary endpoint of percent change from baseline (%CFB) in F-VASI with 11 mg and 22 mg doses versus placebo. Higher response rates were also observed with upadacitinib versus placebo in secondary endpoints, including F-VASI 75 (≥75% reduction from baseline in F-VASI) at week 24 with the 11 mg and 22 mg doses and Total Vitiligo Area Scoring Index (T-VASI) 50 (≥50% reduction from baseline in T-VASI) 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. In addition, 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.
"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," says Thierry Passeron, MD, PhD, a professor and the chair of the Department of dermatology at the Université Côte d'Azur in Nice, France, in a 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."
No New Safety Signals
Treatment-emergent adverse event (TEAE) rates were generally similar with upadacitinib and placebo in period 1 (most common TEAEs: 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. One death adjudicated as undetermined/unknown cause and deemed by the investigator to have no reasonable possibility of being related to the 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 a positive family history of breast cancer. Throughout the study, a single event of 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.
About the Phase 2 Study in Vitiligo
The 52-week, Phase 2b multicenter, randomized, double-blind, placebo-controlled study comprises two periods. Enrolled patients were aged 18–66 years with NSV, a Facial Vitiligo Area Scoring Index (F-VASI) of 1.1, and a Total Vitiligo Area Scoring Index (T-VASI) of 22, both mean scores at baseline. In period one, 185 patients (18 to 65 years old) were randomly assigned to once-daily upadacitinib 22 mg (N=43), upadacitinib 11 mg (N=47), upadacitinib 6 mg (N=49), or placebo (N=46) for 24 weeks of treatment. 166 patients (89.7%) continued to a 28-week blinded extension (period two). In period two, patients receiving upadacitinib during period one continued their respective regimens (upadacitinib 22 mg, N=33; upadacitinib 11 mg, N=45; upadacitinib 6 mg, N=45); patients who received placebo in period one were pre-assigned to receive either upadacitinib 11 mg (N=21) or upadacitinib 22 mg (N=22) in period 2.