Pfizer Inc. (NYSE: PFE) today announced positive topline results from two Phase 3 trials evaluating the efficacy and safety of LITFULO once daily in patients with both active and stable nonsegmental vitiligo (NSV) and who had a broad range of disease severity. The TRANQUILLO study included patients aged 12 years and older, while TRANQUILLO 2 enrolled adults only. Across the studies, both the 50 and 100 milligram doses of LITFULO delivered significant, clinically meaningful improvements over placebo on co-primary endpoints for the facial and total body Vitiligo Area Scoring Index, or VASI. Based on these results, Pfizer intends to submit global regulatory filings for LITFULO as a potential new oral systemic therapy for NSV for adults.
"LITFULO was internally discovered by Pfizer and has a unique mechanism of action targeting TEC family kinases and JAK3, giving rise to its distinct clinical profile," said Michael Vincent, M.D., Ph.D., Chief Inflammation & Immunology Officer, Pfizer. "The positive results from the TRANQUILLO pivotal studies in nonsegmental vitiligo build on our deep expertise in dermatology and on our experience with LITFULO in severe alopecia areata, including its established efficacy and well-characterized safety profile. These findings give us confidence that, if approved, LITFULO could become a new oral systemic treatment option for adults living with NSV, significantly improving and potentially maintaining facial and total body repigmentation."
NSV is a chronic autoimmune disease in which the immune system attacks melanin-producing cells, leading to a loss of pigment and resulting in white macules and patches on the skin/hair over time.1 NSV can appear at any age and in all skin types, causing depigmentation anywhere on the skin, often affecting highly visible areas such as the face, neck, and hands.2 While vitiligo is frequently treated as a cosmetic condition, it is a serious autoimmune disease that can have a considerable impact on patients' lives well beyond the physical symptoms.2,3 Because NSV is a chronic disease, systemic treatment options may be needed to help patients restore skin pigmentation and maintain repigmentation over time.4
Across both studies, significantly more patients treated with LITFULO achieved F-VASI75 (≥75% improvement in Facial Vitiligo Area Scoring Index) and T-VASI50 (≥50% improvement in Total Vitiligo Area Scoring Index) from baseline at Week 52 compared with placebo. In the U.S., the co-primary endpoints were the proportion of patients with F-VASI75 and the proportion of patients with T-VASI50 at Week 52. In countries outside of the U.S., F-VASI75 at Week 52 was the primary endpoint and T-VASI50 at Week 52 was a key secondary endpoint.
The safety profile of LITFULO in NSV was consistent with the established safety profile in alopecia areata. No new safety signals were observed. Overall, the proportion of patients with treatment‑emergent adverse events (TEAEs) was similar across all treatment groups.
"Living with nonsegmental vitiligo can have a profound impact on patients' daily lives. Many patients experience frustration due to the unpredictable nature of nonsegmental vitiligo as well as the limitations of currently available treatments and are seeking additional options that can address the underlying disease,"2 said Iltefat Hamzavi, M.D., Senior Staff Physician, Department of Dermatology, Henry Ford Health and Hamzavi Dermatology Specialists. "In these trials, LITFULO (ritlecitinib) demonstrated significant improvements in both facial and total body repigmentation, with the findings reinforcing the potential of LITFULO to help support a new treatment paradigm with systemic therapies for patients for whom disease burden warrants more than localized treatment alone."
Additional results from TRANQUILLO and TRANQUILLO 2 will be presented at a future medical meeting.
LITFULO was approved in 2023 by the U.S. Food and Drug Administration (FDA) for the treatment of severe alopecia areata in adults and adolescents 12 years and older. Pfizer plans to initiate a pivotal study evaluating LITFULO for the treatment of moderate alopecia areata in 2026.
About the TRANQUILLO Clinical Trial Program
The Phase 3 TRANQUILLO clinical development program consists of two pivotal trials, TRANQUILLO and TRANQUILLO 2, as well as a long-term extension trial, TRANQUILLO LTE, for participants who completed treatment in the parent study, TRANQUILLO. TRANQUILLO and TRANQUILLO LTE included adults and adolescents, while TRANQUILLO 2 enrolled adults only. The program evaluated the efficacy and safety of LITFULO in people with active or stable NSV, with a broad range of disease severity and facial involvement:
- TRANQUILLO evaluated 50 mg LITFULO once-daily in 607 adults and adolescents.
- TRANQUILLO 2 evaluated 50 or 100 mg LITFULO once-daily in 1,567 adults; comparisons involving the 50 mg dose were exploratory and descriptive in nature.
The two trials evaluated 2,174 patients in total with NSV across 271 sites worldwide.
For both trials, the co-primary endpoints in the U.S. were:
- The proportion of patients with ≥75% improvement in F-VASI from baseline (F-VASI75) at Week 52.
- The proportion of patients with ≥50% improvement in T-VASI from baseline (T-VASI50) at Week 52.
