CLINUVEL Reports Progress on SCENESSE® Vitiligo Trials; CUV105 Topline Data Expected by December 2026

CLINUVEL's SCENESSE® Journey: From EPP Approval to Vitiligo Research

CLINUVEL PHARMACEUTICALS operates globally with headquarters and activities across Europe, North America, and the Asia-Pacific, focusing on innovative therapies for genetic, metabolic, and dermatological conditions. Its core portfolio includes melanocortin peptides, with ongoing programs in photomedicine and vitiligo treatment. The company is dual-listed on the Australian Securities Exchange (ASX:CUV) and Nasdaq (Nasdaq:CUVL), offering investors access in both Australian and North American markets.

SCENESSE® (afamelanotide 16mg), CLINUVEL's flagship product, has achieved regulatory approvals for erythropoietic protoporphyria (EPP) across the US, Europe, Canada, Israel, and Australia. It is the first systemic photoprotective drug approved to prevent phototoxicity, including anaphylactoid reactions and burns, in adult EPP patients. This regulatory experience provides valuable groundwork as CLINUVEL advances SCENESSE® for vitiligo, a disorder marked by loss of skin pigmentation.

CUV105 Trial Design: Assessing SCENESSE® Plus NB-UVB Versus Standard Care

The CUV105 study is CLINUVEL's initial late-stage clinical trial for SCENESSE® in vitiligo, enrolling 210 patients worldwide across multiple sites. It evaluates SCENESSE® combined with adjunct narrowband UVB (NB-UVB) phototherapy to induce repigmentation in patients with Fitzpatrick skin types III to VI over a 20-week period. The primary endpoint employs the validated Vitiligo Area Scoring Index (VASI), supported by centralized photographic analysis to objectively measure pigmentation changes from baseline, ensuring rigorous efficacy assessment.

Participants receive either SCENESSE® plus NB-UVB or NB-UVB monotherapy, the current standard treatment for extensive vitiligo depigmentation. The primary endpoint is the proportion achieving at least 50% total body surface area repigmentation (T-VASI50), while the secondary endpoint targets 75% or greater facial repigmentation (F-VASI75). Central photographic analyses complement these endpoints to strengthen regulatory evidence. Topline results from CUV105 are expected in December 2026.

EMA Review and Upcoming CUV107 Pivotal Phase III Trial

The European Medicines Agency (EMA) reviewed the CUV107 study protocol in April 2026, providing recommendations on study design tailored to darker-skinned patients (Fitzpatrick IV-VI). CLINUVEL and the EMA agreed that the pivotal CUV107 trial will enroll 300 vitiligo patients within this population. This regulatory alignment reduces risks of protocol revisions or additional studies post-completion.

CUV107 is set to start in November 2026 with a 12-month recruitment phase. It will evaluate repigmentation efficacy, durability of pigmentation gains, and safety in the specified patient group. This sequential approach, following CUV105, allows incorporation of earlier findings into the pivotal study design ahead of regulatory submission.

Supporting Clinical Evidence from Case Reports and Phase II Data

Prior to CUV105 completion, clinical evidence supporting SCENESSE® in vitiligo includes 12 published case reports showing visible repigmentation at 20 weeks post-treatment. Although detailed results were not disclosed, these reports consistently indicate pigmentation recovery, informing the rationale for subsequent trials.

Additionally, Phase II CUV102 data demonstrated that repigmentation achieved during treatment was largely maintained during a six-month follow-up without therapy, suggesting durable effects. This durability is clinically meaningful, potentially reducing the need for continuous treatment and supporting regulatory claims of sustained benefit.

Regulatory Strategy: Leveraging EPP Approval to Expedite Vitiligo Pathway

CLINUVEL's Chief Scientific Officer Dr Dennis Wright emphasized that regulatory pathways for SCENESSE® in vitiligo hinge on outcomes from CUV105 and CUV107. He noted visible pigmentation improvements in CUV105 case studies but stressed that full efficacy and safety profiles will be defined by complete trial data. Dr Wright highlighted the advantage of SCENESSE®’s existing marketing authorizations for EPP by EMA and other regulators, which provide familiarity with the drug’s safety and manufacturing, facilitating future vitiligo reviews.

This established regulatory foundation reduces the review burden for vitiligo applications, allowing agencies to concentrate on vitiligo-specific efficacy and safety data. Such precedent may accelerate approval timelines and minimize the need for additional studies.

Innovative Combination Therapy and FDA Considerations

The SCENESSE® vitiligo program employs a novel approach by combining the drug with adjunct narrowband UVB phototherapy rather than monotherapy. Dr Emilie Rodenburger, CLINUVEL's Director of Global Clinical Affairs, noted evolving FDA expectations, including Real Time Clinical Trials and potential acceptance of single pivotal trials for some indications. However, she indicated this single pivotal trial policy likely does not apply to SCENESSE® in vitiligo due to its novel combination treatment paradigm.

This combination differentiates SCENESSE® from traditional monotherapies and likely necessitates robust evidence from multiple studies demonstrating efficacy and safety in the targeted populations. Dr Rodenburger emphasized the goal of submitting comprehensive data to maximize marketing authorization chances and avoid multiple submissions, referencing challenges faced by peer companies.

Targeted Patient Population and Market Implications

The SCENESSE® vitiligo program focuses on patients with darker skin tones, enrolling Fitzpatrick skin types III-VI in CUV105 and IV-VI in CUV107. Vitiligo’s depigmentation is particularly visible and psychologically impactful in these populations. The comparator in CUV105 is NB-UVB monotherapy, the current standard of care for extensive vitiligo in darker-skinned patients.

This focus addresses a significant unmet clinical need and allows precise patient selection for clinical trials and regulatory labeling. Improved repigmentation or reduced treatment duration could substantially enhance quality of life for this group.

Safety and Tolerability Profile

CLINUVEL reports that SCENESSE® combined with NB-UVB phototherapy has demonstrated good tolerability in preclinical and clinical settings. Specific adverse event data from CUV105 remain undisclosed as the trial is ongoing. The safety profile aligns with SCENESSE®’s established tolerability in EPP, supported by extensive clinical and post-marketing experience.

Safety assessments are integral to both CUV105 and the forthcoming CUV107 trial, which will specifically evaluate safety in darker-skinned patients (Fitzpatrick IV-VI). Regulatory agencies will scrutinize any safety signals unique to the vitiligo population or combination therapy.

Development Timeline and Investor Outlook

CLINUVEL has outlined clear timelines for its vitiligo program: CUV105 topline data are expected in December 2026, a critical near-term milestone. The CUV107 pivotal Phase III trial is planned to start in November 2026, with patient recruitment concluding around November 2027. Subsequent data analysis and regulatory filing preparations will extend into 2028 or later.

Investors should view the vitiligo program as a medium- to long-term growth opportunity. CLINUVEL’s dual listing on ASX and Nasdaq offers flexible trading options as clinical milestones approach.

Competitive Landscape and Market Potential

If approved, SCENESSE® would introduce a novel treatment option for vitiligo, currently lacking global approval for this indication. The standard of care for extensive vitiligo in darker-skinned patients remains NB-UVB monotherapy, the comparator in CUV105. Demonstrated superiority of SCENESSE® plus NB-UVB in repigmentation outcomes could provide a significant clinical and commercial advantage.

CLINUVEL benefits from established manufacturing, supply chain, and regulatory expertise from its EPP approvals, positioning it favorably against competitors in the dermatology space. However, the vitiligo treatment market is competitive, with multiple companies pursuing repigmentation therapies. The results of CUV105 and CUV107 will be pivotal in defining SCENESSE®’s market positioning and potential uptake.