210-Patient SCENESSE Vitiligo Study to Report in December

Vitiligo Trial Progress and Key Endpoints

Clinuvel Pharmaceuticals (ASX:CUV, Nasdaq: CUVL) is on track to deliver topline results from its CUV105 vitiligo study by December 2026. This Phase II trial, involving 210 patients globally, evaluates SCENESSE® (afamelanotide) in combination with adjuvant narrowband ultraviolet B (NB-UVB) phototherapy versus NB-UVB monotherapy, the current standard of care. The trial specifically targets patients with darker skin types (Fitzpatrick III-VI), a group often underserved in vitiligo research.

The primary endpoint focuses on the proportion of patients achieving at least 50% repigmentation across total body surface area (T-VASI50), with a secondary endpoint measuring at least 75% facial repigmentation (F-VASI75). Centralised photographic analysis provides an objective measure of pigmentation changes, complementing clinical scoring.

Regulatory Milestones and Phase III Launch

Following the CUV105 results, Clinuvel plans to initiate the pivotal Phase III CUV107 trial in November 2026. The European Medicines Agency (EMA) reviewed and advised on the CUV107 protocol earlier this year, endorsing a design enrolling 300 patients with Fitzpatrick skin types IV to VI. This trial will assess not only repigmentation efficacy but also the durability of response and safety over a longer term.

The EMA's input underscores the agency's interest in a robust evidence package, reflecting a regulatory approach that prioritises comprehensive data over expedited but potentially fragmented submissions. Clinuvel’s Chief Scientific Officer, Dr Dennis Wright, highlighted that while early case reports and Phase II data show promising repigmentation, full results from these studies will determine the drug’s future label and market potential.

Clinical Context and Market Positioning

SCENESSE® is currently approved for erythropoietic protoporphyria (EPP), a rare phototoxicity disorder, with marketing authorisations in multiple regions including the US, Europe, and Australia. Its repurposing for vitiligo represents a significant expansion opportunity, particularly for patients with darker skin tones who face greater challenges with existing therapies.

Earlier Phase II data (CUV102) showed that repigmentation achieved during treatment was largely maintained after six months without therapy, suggesting potential lasting benefits. Physicians have also published 12 case reports indicating visible pigmentation gains at 20 weeks of SCENESSE® treatment combined with NB-UVB.

Clinuvel’s regulatory strategy reflects evolving expectations, especially from the US FDA, which is increasingly emphasising real-time clinical trials and streamlined pivotal studies. However, given SCENESSE®’s novel combination approach with phototherapy, Clinuvel anticipates maintaining a more traditional regulatory pathway to ensure robust data and avoid multiple resubmissions.