CLINUVEL progresses innovative DNA Repair Program

MELBOURNE, Australia, Sept. 10, 2020 (GLOBE NEWSWIRE) -- CLINUVEL PHARMACEUTICALS LTD today announced the progression of its drug SCENESSE^® (afamelanotide 16mg) to treat the disease xeroderma pigmentosum (XP). The aim of the development program is to confirm the drug’s ability to regenerate DNA of skin exposed to ultraviolet (UV) damage.¹

Skin incurs DNA damage following UV exposure. Non-ionising light penetrates the nucleus of skin cells and causes distortions to the DNA helix known as photoproducts. If left unrepaired, these chemical changes to DNA may replicate as mutations, leading to irreversible damage (photoaging) and further progress to skin cancer, including melanoma.

Human biology contains complex mechanisms to protect itself from UV damage and restore cellular DNA to its original state. People of fair-skinned complexion are known to have defects in these UV protective and DNA regenerative processes, increasing their long-term risk of skin cancer.

Having commercialised SCENESSE^® in Europe and the USA for the rare genetic disorder porphyria (EPP), CLINUVEL is expanding its clinical research, aiming to confirm how intervention with the drug enhances elimination of photoproducts and regeneration of DNA.

“In SCENESSE^® we have a hormone acting on various organs and receptors, but most of all known to protect human DNA,” CLINUVEL’s Chief Scientific Officer, Dr Dennis Wright said. “Our task is to confirm how DNA regeneration occurs within genetically affected patients and healthy subjects.”

Following decades of clinical experience with SCENESSE^®, CLINUVEL is now accelerating a comprehensive DNA Repair Program by first treating patients with the rare genetic disorder xeroderma pigmentosum (XP) who have the most extreme deficiencies in their DNA repair processes, leading to a 10,000-fold increase in their risk of skin cancer.

“Tragically, XP provides an extreme model of what happens to our skin if UV-induced damage is left unrepaired,” Dr Wright said. “From a young age, XP patients are incapable of responding to DNA damage caused by UV exposure and experience disfiguring and aggressive skin cancers.

“After two decades of clinical research, I’m delighted that our team can now focus on the XP patients who are severely affected by UV radiation leaving them a short life expectancy. We will facilitate treatment for the first patient in the next few weeks,” Dr Wright said.

The first clinical results from the DNA repair program are expected to be reported in 2021.

¹ SCENESSE^® (afamelanotide 16mg) is approved in the European Union 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 .

Note to editors: a technical release has been lodged with the ASX. CLINUVEL CEO Dr Philippe Wolgen is available for interview through Monsoon Communications.

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

About CLINUVEL PHARMACEUTICALS LIMITED

CLINUVEL PHARMACEUTICALS LTD (ASX: CUV; NASDAQ INTERNATIONAL DESIGNATION ADR: CLVLY; XETRA-DAX: UR9) is a global biopharmaceutical company focused on developing and delivering treatments for patients with a range of severe genetic, skin, and systemic disorders. As pioneers in photomedicine and understanding the interaction of light and human biology, CLINUVEL’s research and development initially has led to innovative treatments for patient populations with a clinical need for photoprotection and repigmentation. These patient groups range in size from 5,000 to 45 million worldwide. CLINUVEL’s lead compound, SCENESSE^® (afamelanotide 16mg), was approved by the European Commission in 2014 and the US Food and Drug Administration in 2019 for the prevention of phototoxicity (anaphylactoid reactions and burns) in adult patients with erythropoietic protoporphyria (EPP). More information on EPP can be found at . CLINUVEL is advancing its portfolio of melanocortins, among which is PRÉNUMBRA^® for the treatment of several critical disorders. Headquartered in Melbourne, Australia, CLINUVEL has operations in Europe, Singapore, and the USA. For more information please go to .

SCENESSE^® and PRÉNUMBRA^® are registered trademarks 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 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); our ability to achieve expected safety and efficacy results 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, 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^® 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 based products; decisions by regulatory authorities regarding approval of our products as well as their decisions regarding label claims; any failure 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 2019 Annual Report and 2020 Preliminary Final 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 the forecasts and estimates is available on request. Past performance is not an indicator of future performance.

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