CLINUVEL progresses vitiligo study

Executive Summary:

• Institutional Review Board (IRB) approves CUV104 study
• Afamelanotide evaluated as monotherapy in vitiligo patients
• CUV104 study to focus on patients of darker skin complexion (Fitzpatrick IV-VI)

CLINUVEL today announced that it will start treatment of vitiligo patients with its drug afamelanotide under a new study protocol (CUV104). The Company has received positive review from the Institutional Review Board (IRB) to commence the CUV104 study, which will be conducted at a North-American vitiligo expert centre.

Up to six adult vitiligo patients with darker skin complexions (Fitzpatrick Skin Types IV-VI) will be enrolled in the CUV104 study, evaluating patients’ response to afamelanotide as a monotherapy over six months.

“We are thrilled to start this study, as we have seen the efficacy of afamelanotide in vitiligo in earlier studies and now we are all anxious to learn the effect of the drug as a monotherapy,” CLINUVEL’s Director of North American Operations, Dr Linda Teng said. “Visible and beneficial effects from the drug as a single therapy would be a leap forward for these patients, as our hormonal solution would be the most biological answer to a stigmatising disorder.”

Impacts of Vitiligo on sense of identity

Vitiligo is a depigmentation disorder characterised by the chronic and progressive loss of functional epidermal melanocytes (the melanin producing cells), which often leads to disfiguration and psychosocial distress. The precise cause of vitiligo remains unknown.

A high disease burden is experienced by patients with Fitzpatrick Skin Types IV-VI (darker skin), where the contrasting loss of skin colour can lead to a profound sense of loss of identity.¹ On 8 March 2021, the FDA hosted a patient-focused vitiligo meeting for the first time, when testimonies were given of the impact of vitiligo in patients of darker skin colour, and its effect on their quality of life.² Going forward, the FDA stated that it will take into account quality of life and impact of disease in new drug evaluations.

Clinuvel’s program in Vitiligo

The earlier studies CUV102 (US) and CUV103 (Singapore) have shown that afamelanotide treatment, in combination with narrowband ultraviolet B (NB-UVB) therapy, can achieve faster and deeper repigmentation than NB-UVB as monotherapy, currently the most common treatment in vitiligo.³

CUV104 study

The CUV104 study is an open label, phase II study assessing both the safety and changes in pigmentation in generalised vitiligo following administration of afamelanotide as monotherapy. The study will be conducted during the summer months in a North-American centre that specialises in vitiligo, leucotrichia, and pigmentation disorders.

The primary objective of CUV104 is to evaluate afamelanotide’s ability to repigment facial lesions (patches). The face and neck are regarded by vitiligo patients as the most distinct body part, and loss of pigmentation affects communication and normal social interaction. Six eligible patients will be enrolled in a six-month study, which includes a three-month follow-up period.

The study objectives will be evaluated with validated tools, including the Vitiligo Area Scoring Index (VASI), Patient Global Assessment (PtGA), Vitiligo Noticeability Scale (VNS), and the Vitiligo Quality of Life index (VitiQoL).

The study design is a result of scientific review by the FDA, and collaboration with vitiligo expert clinicians.

“We have fundamentally supported the concept of providing an effective therapy acting through the bloodstream reaching all melanocytes, as opposed to a localised or topical therapy that requires frequent applications for patients,” Dr Teng said. “In scientific discussions over the years, we have received positive encouragement both from expert physicians and regulatory authorities to use our lead hormone analogue in vitiligo. Part of the novelty of our approach lies in the treatment of patients who suffer from the psychosocial impact due to loss of their distinct skin colour.”