UR9 Clinuvel Pharmaceuticals Ltd

A new potential therapy for the pigment loss disorder vitiligo will undergo further studies in North America this year. The drug afamelanotide – approved by US and European regulators for a rare light intolerance disorder – is being evaluated by Australian company CLINUVEL as a treatment for vitiligo patients with darker skin types.

Vitiligo causes the progressive loss of skin pigment in lesions (patches) across the body and affects an estimated 45 million individuals worldwide. Pigment loss caused by vitiligo is most pronounced in patients with darker skin types who report the greatest impact of the disease on their quality of life. Treatment options remain limited, with no treatments for repigmentation of vitiligo currently approved by the US Food and Drug Administration (FDA).

“Many vitiligo patients must grapple with a sense of lost identity as their skin visibly and inexplicably changes, forcing them to withdraw from society and having a severe impact on their mental health and quality of life,” CLINUVEL’s Director of North American Operations, Dr Linda Teng said.

“Clinical trials of afamelanotide to date have shown the drug can assist in repigmenting vitiligo patients’ skin, with the most promising results in darker skinned patients. Therefore we have chosen to focus on this patient group, with the highest unmet need, as our clinical program progresses,” Dr Teng said.

Afamelanotide as repigmentation therapy

One of a new class of drugs known as melanocortins, afamelanotide activates melanin, the pigment in skin. The drug is also understood to play a role in maintaining the overall health of skin including melanocytes, the skin cells responsible for melanin production.

“Many experimental vitiligo treatment approaches to date have been ineffective, inconvenient, or cause unpleasant side effects, often seeking to bluntly suppress the immune system to allow for repigmentation,” Dr Teng said. “Afamelanotide treatment is expected to help regulate immune and inflammatory responses in the skin while restoring patients’ pigmentary function: stimulating melanocytes to migrate to affected skin and produce melanin. Preliminary results should be available late this year, with full results in 2023.”

CLINUVEL’s initial studies in vitiligo patients showed that afamelanotide could repigment skin in combination with a light-based therapy, with nearly 100 patients receiving treatment to date and the safety profile of the drug maintained.1,2 The program will now focus on afamelanotide as a monotherapy after approval of the CUV104 study was received from the Institutional Review Board (IRB) at the specialist medical centre conducting the study.

CUV104 will commence in North America during northern summer months. Adult vitiligo patients will receive multiple doses of afamelanotide with expert physicians monitoring repigmentation and safety for six months. The impact of treatment on the patients’ quality of life will be captured using validated clinical tools.

Up to six patients will be enrolled in CUV104, sufficient to meet the statistical needs of a pilot study, provide feedback on protocol designs for larger studies of afamelanotide as a monotherapy, and complete quickly in a clinical environment where COVID-19 restrictions have impacted study enrolment rates.

In total, over 11,000 doses of afamelanotide have been administered to over 1,600 individuals worldwide across clinical, special access and post-authorisation programs.2

Media enquiries: Monsoon Communications, Mr Rudi Michelson, +61 411 402 737, rudim@monsoon.com.au

1 Results from these studies have been published in peer-reviewed journals:

• Lim, H. W. et al. (2015). Afamelanotide and Narrowband UV-B Phototherapy for the Treatment of Vitiligo: A Randomized Multicenter Trial. JAMA Dermatology, 151(1), 42.
• Toh, J. et al. (2020). Afamelanotide implants and narrow-band ultraviolet B phototherapy for the treatment of nonsegmental vitiligo in Asians. Journal of the American Academy of Dermatology, 82(6), 1517–1519.

2 SCENESSE® (afamelanotide 16mg) is approved in the European Union and Australia as an orphan medicinal product for the prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). SCENESSE® is approved in the USA to increase “pain- free” light exposure in adult EPP patients with a history of phototoxicity. Information on the product can be found on CLINUVEL’s website at www.clinuvel.com.

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.

Head of Investor Relations Mr Malcolm Bull, CLINUVEL PHARMACEUTICALS LTD

Investor Enquiries https://www.clinuvel.com/investors/contact-us

Forward-Looking StatementsThis release contains forward-looking statements, which reflect the current beliefs and expectations of CLINUVEL’s management. A complete copy of our forward-looking statements advisory is available on www.clinuvel.com.

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