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.”

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¹ Ezzedine, K., Grimes, P. E., Meurant, J. M., Seneschal, J., Léauté-Labrèze, C., Ballanger, F., Jouary, T., Taïeb, C., & Taïeb, A. (2015). Living with vitiligo: results from a national survey indicate differences between skin phototypes. The British journal of dermatology, 173(2), 607–609. https://doi.org/10.1111/bjd.13839

² Public meeting on patient-focused drug development for Vitiligo. U.S. Food and Drug Administration. (n.d.). https://www.fda.gov/drugs/news-events-human-drugs/public-meeting-patient-focused-drug-development-vitiligo-03082021-03082021

³ Lim, H. W., Grimes, P. E., Agbai, O., Hamzavi, I., Henderson, M., Haddican, M., Linkner, R. V., & Lebwohl, M. (2015). Afamelanotide and narrowband UV-B phototherapy for the treatment of vitiligo: a randomized multicenter trial. JAMA dermatology, 151(1), 42–50. https://doi.org/10.1001/jamadermatol.2014.1875.

About CLINUVEL PHARMACEUTICALS LIMITED

CLINUVEL (ASX: CUV; ADR LEVEL 1: CLVLY; XETRA-DAX: 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 the general population. 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, 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.

SCENESSE^®, PRÉNUMBRA^®, and NEURACTHEL^® are registered trademarks of CLINUVEL.

Authorised for ASX release by the Board of Directors of CLINUVEL PHARMACEUTICALS LTD

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Forward-Looking Statements

This release contains forward-looking statements, which reflect the current beliefs and expectations of CLINUVEL’s management. Statements may involve a number of known and unknown risks that could cause our future results, performance or achievements to differ significantly from those expressed or implied by such forward-looking statements. Important factors that could cause or contribute to such differences include risks relating to: our ability to develop and commercialise pharmaceutical products; the COVID-19 pandemic and/or other world, regional or national events affecting the supply chain for a protracted period of time, including our ability to develop, manufacture, market and sell biopharmaceutical products; competition for our products, especially SCENESSE^® (afamelanotide 16mg), PRÉNUMBRA^® or NEURACTHEL^®; our ability to achieve expected safety and efficacy results in a timely manner through our innovative R&D efforts; the effectiveness of our patents and other protections for innovative products, particularly in view of national and regional variations in patent laws; our potential exposure to product liability claims to the extent not covered by insurance; increased government scrutiny in either Australia, the U.S., Europe, Israel, China and Japan of our agreements with third parties and suppliers; our exposure to currency fluctuations and restrictions as well as credit risks; the effects of reforms in healthcare regulation and pharmaceutical pricing and reimbursement; that the Company may incur unexpected delays in the outsourced manufacturing of SCENESSE^®, PRÉNUMBRA^® or NEURACTHEL^® which may lead to it being unable to supply its commercial markets and/or clinical trial programs; any failures to comply with any government payment system (i.e. Medicare) reporting and payment obligations; uncertainties surrounding the legislative and regulatory pathways for the registration and approval of biotechnology and consumer based products; decisions by regulatory authorities regarding approval of our products as well as their decisions regarding label claims; our ability to retain or attract key personnel and managerial talent; the impact of broader change within the pharmaceutical industry and related industries; potential changes to tax liabilities or legislation; environmental risks; and other factors that have been discussed in our 2021 Annual Report. Forward-looking statements speak only as of the date on which they are made, and the Company undertakes no obligation, outside of those required under applicable laws or relevant listing rules of the Australian Securities Exchange, to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise. More information on preliminary and uncertain forecasts and estimates is available on request, whereby it is stated that past performance is not an indicator of future performance.

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