CLINUVEL receives final EMA scientific advice for pivotal Phase III vitiligo study

“Totality of evidence” approach to evaluate SCENESSE®

EXECUTIVE SUMMARY

• final European scientific advice received for pivotal Phase III vitiligo study
• EMA emphasised its “totality of evidence” approach
• central photographic review and validated disease assessment tools agreed
• CUV107 to compare SCENESSE® with adjunct NB-UVB vs NB-UVB monotherapy
• CUV107 study (n=300) to commence in 2H 2026

MELBOURNE, Australia, April 24, 2026 (GLOBE NEWSWIRE) — CLINUVEL PHARMACEUTICALS LTD today announced that the European Medicines Agency (EMA) has provided final scientific advice on the design of the planned pivotal Phase III CUV107 study and anticipated evaluation of evidence of CLINUVEL’s drug SCENESSE® (afamelanotide) as a systemic vitiligo treatment.

EMA’s Scientific Advice¹

Following over 12 months of interaction, two formal submissions and a Discussion Meeting with the EMA’s Scientific Advice Working Party (SAWP), the Committee for Medicinal Products for Human Use (CHMP), has issued Scientific Advice on CLINUVEL’s program evaluating SCENESSE® as a systemic therapy for adults and adolescents with non-segmental vitiligo.

The EMA proposed to evaluate the efficacy of SCENESSE® in vitiligo based on a regulatory approach of “totality of evidence”, with T-VASI50² the primary endpoint while patient reported outcomes and clinical data from the other vitiligo studies with SCENESSE® will be evaluated for final analyses of efficacy and safety.

Given the importance of patients’ perceptions of visible change in pigmentation, the EMA emphasised that the assessment by vitiligo patients themselves would play an important role in the final evaluation when vitiligo data from CUV107 and previous studies would be submitted for marketing authorisation.

In particular, photographic evidence of change from baseline (CFB) will be used to assess primary and secondary endpoints, T-VASI50² and F-VASI75³, with an array of other related secondary objectives. In total, five patient and physician surveys are integrated in the study to record Patient Reported Outcomes.

The EMA advised that patients of darker skin colours (Fitzpatrick IV-V-VI) would benefit from systemic treatment first, since the visibility of disease – due to the contrast between unaffected skin and vitiligo lesions – is most pronounced in these patient groups.

Commentary

“The dialogue took one year but eventually resulted in a one-off position taken by the Agency, who are now fully across the SCENESSE® program and possible expected outcomes in vitiligo,” said Dr Emilie Rodenburger, CLINUVEL’s Director, Global Clinical Affairs.

“A major step forward was the request by the SAWP to access photographic changes of vitiligo patients receiving treatment, which demonstrated visual improvement in pigmentation.

“The Discussion Meetings provided three take aways: the patient population of darker skin complexion will be the first to benefit from a systemic treatment, the design of the study and, significantly, the ‘totality of evidence’ approach which will be taken during final evaluation of evidence submitted to the EMA. We can only be appreciative of the extensive work the Agency has done to get across our program,” Dr Rodenburger concluded.

¹Scientific Advice provided by the EMA is non-binding but good practice and documented in the final EPAR (European Public Assessment Report) issued at the conclusion of a marketing authorisation evaluation. The EMA provides opportunities during the clinical development program for drug developers to seek formal guidance, with a focus on advising on expected standards to establish pre-clinical and clinical safety and efficacy.

²CLINUVEL will use T-VASI50 – the proportion of patients who achieve 50% or more repigmentation of the total body excluding hands and feet during the study – as the primary endpoint; this will be indicative but not determinative for efficacy.

³ F-VASI75 – the proportion of patients who achieve 75% or more repigmentation of head and neck (excluding lips) and excluding hands and feet during the study – is being used as a secondary endpoint; this will be indicative but not determinative for efficacy.

About vitiligo

Vitiligo is believed to be a multifactorial disorder with immune components in some cases resulting in progressive loss of pigment (melanin) of the skin and associated with a severe impact on quality of life. It affects an estimated 0.5%-2% of the general population, with no approved therapies for patients with extensive depigmentation (affecting >10% of total body surface area).

About CLINUVEL PHARMACEUTICALS LIMITED

CLINUVEL (ASX: CUV; ADR LEVEL I: CLVLY; Börse Frankfurt: UR9) is a global specialty pharmaceutical group focused on developing and commercialising treatments for patients with genetic, metabolic, systemic, and life-threatening, acute disorders, as well as healthcare solutions for specialised populations. As pioneers in photomedicine and the family of melanocortin peptides, CLINUVEL’s research and development has led to innovative treatments for patient populations with a clinical need for systemic photoprotection, assisted DNA repair, repigmentation and acute or life-threatening conditions who lack alternatives.
CLINUVEL’s lead therapy, SCENESSE® (afamelanotide 16mg), is approved for commercial distribution in Europe, the USA, Israel, and Australia as the world’s first systemic photoprotective drug for the prevention of phototoxicity (anaphylactoid reactions and burns) in adult patients with erythropoietic protoporphyria (EPP). Headquartered in Melbourne, Australia, CLINUVEL has operations in Europe, Singapore, and the USA. For more information, please go to https://www.clinuvel.com.

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Mr Malcolm Bull, CLINUVEL PHARMACEUTICALS LTD

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Forward-Looking Statements

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