CLINUVEL Progresses DNA Repair Program

CLINUVEL PHARMACEUTICALS LTD today announced that it has received the necessary regulatory and ethics committee approvals to commence a new study in patients with the rare DNA repair disorder Xeroderma Pigmentosum (XP).

The Phase II study (CUV156) will evaluate the safety of SCENESSE^® (afamelanotide 16mg)¹ in XP-C patients, as well as the drug’s ability to assist reparative processes following ultraviolet (UV) provoked DNA damage of the skin. Following the lifting of pandemic restrictions in hospitals, the first XP-C patients are expected to be screened by the end of October.

“We have used the time to add more parameters to this trial, and patients and clinicians are very excited to start this innovative study, evaluating afamelanotide as a treatment for Xeroderma Pigmentosum,” CLINUVEL’s Head of Clinical Operations, Dr Pilar Bilbao said. “XP is a severely life-limiting disorder without approved treatments. The regulatory authorities and ethics committees have been understandably cautious to subject these patients to interventional studies but have given their clearance to commence this program.

“Following the first XP patient treated safely in 2020, SCENESSE^® will be evaluated in XP patients as part of our DNA repair program. It has been a long time in the making to treat this group of patients.”

UV Damage Places XP Patients at Extreme Skin Cancer Risk

UV radiation damages the nucleus of skin cells, and causes defects of the DNA helix known as “photoproducts”. If left unrepaired, these chemical DNA changes may replicate as mutations, leading to irreversible damage (photoaging) and progression to skin cancers, including melanoma.

Due to eight distinct genetic defects severely impairing their DNA repair mechanisms, XP patients are at the most extreme risk of skin cancers from an early age and have a life expectancy of around 30 years. Most patients are lifelong shielded from all UV light exposure.

CUV156 Clinical Study

The CUV156 study aims to evaluate the effect of SCENESSE^® administered every two weeks to six patients with the XP-C complementation. CLINUVEL will assess whether SCENESSE^® increases the quantum of UV light needed to cause DNA damage of skin cells, as well as the extent of skin repair before and after treatment. The CUV156 study will be conducted in a single European expert clinic.

CUV156 is the first in a set of studies planned as part of the DNA repair program. The CUV150 study was initially planned to be conducted in early 2021, but the delays incurred by the hospital due to COVID-19 offered the opportunity to improve study design and add more analytical parameters, following consultation with XP clinical experts.

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¹ 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.

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

Annex I: Following ASX Best Practice

Name of study

A Proof of Concept Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Subcutaneous Implants of Afamelanotide in Patients with Xeroderma Pigmentosum (CUV156).

Primary objective

Evaluate the impact of afamelanotide on minimal erythema dose (MED) in patients with XP.

Secondary objectives

Explore the impact of afamelanotide on UV-induced DNA damage and repair capacity in patients with XP.
Evaluate the safety and tolerability of afamelanotide in patients with XP.
Evaluate the impact of afamelanotide on the skin disease severity of patients with XP.
Evaluate the impact of afamelanotide on the skin melanin density of patients with XP.
Evaluate the impact of afamelanotide on the quality of life of patients with XP.

Blinding status

Open label.

Product development status

Good Manufacturing Practice (GMP) Standard.

Treatment method and dose levels

Six SCENESSE® (afamelanotide 16 mg) implants

Number of trial subjects

Up to six XP patients.

Subject selection criteria

To be eligible to enter the study, volunteers must meet the following inclusion criteria:

• Adult males and females with genetically confirmed diagnosis of XP-C aged between 18 and 75 years (inclusive).
• Able to understand and provide written Informed Consent prior to the performance of any study-specific procedure.
• Willing and able to comply with the conditions specified in the protocol and study procedures, in the opinion of the Investigator.
• Free of significant abnormal findings (including severe hepatic disease, hepatic impairment and renal impairment) as determined during the screening procedure by medical history and vital signs.

Further safety related inclusion and exclusion criteria apply.

Trial location

One European XP expert clinic.

Duration of trial

Up to nine months.

Trial standard

In compliance with Good Clinical Practice (GCP) and ICH guidelines.

Annex 2: About CLINUVEL PHARMACEUTICALS LIMITED

CLINUVEL PHARMACEUTICALS LTD (ASX: CUV; NASDAQ INTERNATIONAL DESIGNATION ADR: CLVLY; XETRA-DAX: UR9) is a global and diversified biopharmaceutical company focused on developing and commercialising treatments for patients with genetic, metabolic, and life-threatening disorders, as well as healthcare solutions for the general population. As pioneers in photomedicine and understanding the interaction of light and human biology, CLINUVEL’s research and development has led to innovative treatments for patient populations with a clinical need for systemic photoprotection, DNA repair and acute or life-threatening conditions. 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, the US Food and Drug Administration in 2019 and the Australian Therapeutic Goods Administration in 2020 for the prevention of phototoxicity (anaphylactoid reactions and burns) in adult patients with erythropoietic protoporphyria (EPP). More information on EPP can be found at http://www.epp.care. Headquartered in Melbourne, Australia, CLINUVEL has operations in Europe, Singapore and the USA. For more information please go to http://www.clinuvel.com.

SCENESSE^® and PRÉNUMBRA^® are registered trademarks of CLINUVEL PHARMACEUTICALS LTD.

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

Head of Investor Relations

Mr Malcolm Bull, CLINUVEL PHARMACEUTICALS LTD

Investor Enquiries

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

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 2020 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 the forecasts and estimates is available on request. Past performance is not an indicator of future performance.

www.clinuvel.com
Level 11
535 Bourke Street
Melbourne – Victoria, Australia, 3000
T +61 3 9660 4900
F +61 3 9660 4999

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