American Opportunity
Scaling operations in North America
Strategic Context
CLINUVEL is committed to the development of innovative melanocortin-based products to treat patients with unmet medical needs. Central to our business is an engagement with patient populations within the United States of America who may benefit from our technologies, both directly and through healthcare professionals. Since the launch of SCENESSE® (afamelanotide), the Company has taken considerable steps to build a commercial footprint to meet the needs of erythropoietic protoporphyria (EPP) patients. We are steadfast to replicate this approach to vitiligo patients in the near-term.
Treatment of EPP
Prior to April 2020, there was no approved treatment for American patients with the acute light-affected disorder, EPP. CLINUVEL developed SCENESSE® as the world’s first systemic photoprotective drug for the prevention of phototoxicity (anaphylactoid reactions and burns) in adult patients with EPP, with the U.S. Food and Drug Administration (FDA) approving the drug in October 2019. CLINUVEL promptly commenced commercial distribution in April 2020 to satisfy this unmet need and has successfully built a network of Specialty Centers across the U.S. to treat EPP patients. The ascent to this summit required many years of extensive research and building trust from patients and families, patient associations, physicians, regulators and insurers (both private and government), and broader healthcare networks within the U.S.A. who facilitate patient treatment.
CLINUVEL’s early plans were to distribute SCENESSE® through 30 trained and accredited Specialty Centers, strategically located to enable reasonable patient access to treatment. When this milestone was achieved ahead of schedule, the Company kept adding Specialty Centers to the network. As of 30 June 2025, there are 104 centers across North America and we are targeting to reach 120 by the end of 2025. This network provides convenient patient access to SCENESSE® and provides a ready-made network for the treatment of patients with vitiligo. CLINUVEL intends to continue building and strengthening its Specialty Center network to better meet patients’ long-term needs.
Other initiatives in the U.S. have been to establish:
- patient treatment database compliant with the Health Portability and Accountability Act (HIPAA) to ease insurance processes and ensure regularity of treatment;
- unique codes for the drug (J-code) and treatment (CPT Code®) which serve to streamline reimbursement by insurers – these codes can also be used for additional indications;
- a savings program to assist privately-insured patients with the ‘out-of-pocket’ costs of treatment; and
- a central distribution facility from which SCENESSE® is delivered as ordered by Specialty Centers.
The impact of U.S. operations on CLINUVEL’s financial performance has been positive with a rising trend in revenues evident since the 2021 financial year – refer to the Financial Review on pages 54-63 of this report.
The U.S. team, reporting into Palo Alto, has increased under the leadership of Dr Linda Teng, Director of North American Operations. The team consists of clinical specialists and patient relations personnel, plus financial services, legal, and investor relations professionals.
Unmet Need in Vitiligo
Vitiligo is an auto-immune condition which affects around 1% of the world’s population, causing visible loss of pigment in the skin. The impact of vitiligo is most severe in patients with darker skin complexions, specifically those with Fitzpatrick skin types IV, V and VI. SCENESSE® – with adjunct narrowband ultraviolet-B (NB-UVB) phototherapy –is currently being evaluated in a Phase III clinical program as a systemic therapy for skin repigmentation. There is currently no systemic treatment available to repigment the skin and no approved therapy for patients with extensive pigment loss (affecting >10% of body surface area).
Raising awareness
In October 2023, the first Phase III study (CUV105) commenced recruitment. Initial case reports of patients treated have demonstrated considerable repigmentation and that the drug is well tolerated by individual patients. The first case report was released in March 2024 at the American Academy of Dermatology (AAD) Meeting in San Diego.
The AAD Meeting in Orlando, Florida, in March 2025 was the start and one of many important milestones for the Group’s U.S. expansion plans. CLINUVEL introduced its pioneering research and future ambitions to 20,000 physicians, clinicians, academics and industry representatives. Visitors to CLINUVEL’s Pavilion of Photomedicine learned first-hand about the Company and its expertise in photomedicine. They also heard from people living with vitiligo about the impact of the disease and the potential of a systemic repigmentation therapy. An important outcome was an increased awareness of CLINUVEL’s ambitions to prepare a vitiligo franchise in the U.S.. Attendees understood well the potential of afamelanotide to systemically repigment the skin.
Clinical developments
CLINUVEL is advancing its clinical program with the first Phase III clinical study (CUV105) completing recruitment in May 2025. Work towards the final design of CUV107 is in progress. Two studies totaling over 400 patients are considered necessary to provide a compelling dossier to the FDA for consideration of an extension to the approved label of SCENESSE® to treat vitiligo.
The Company has released five case reports from CUV105 patients who have received the afamelanotide (with adjunct NB-UVB) therapy. The visual results are compelling, and the feedback from patients and physicians is positive.
CUV105 Case Reports
Case Study 1
Participant A
Female, 55 years old, Skin Type IV
Diagnosed with vitiligo in 2006, slowly progressive disease activity, no previous episodes of repigmentation, and no family history of vitiligo. Unresponsive to previous vitiligo treatments.
PHYSICIAN’S REPORT: 80-90% repigmentation was seen after Day 140 but near total repigmentation was achieved after continued NB-UVB monotherapy.
Day 0
Baseline
Day 134
7 afamelanotide implants
39 NB-UVB treatments
Day 222*
7 afamelanotide implants
53 NB-UVB treatments
Case Study 2
Participant B
Male, 52 years old, Skin Type IV
Diagnosed with vitiligo in 2023, progressive disease activity, no previous episodes of repigmentation, and no family history of vitiligo. No history of previous vitiligo treatments.
PHYSICIAN’S REPORT: The patient and our team are pleased with the results. Patient reports greater self esteem posttreatment.
Day 0
Baseline
Day 140
7 afamelanotide implants
40 NB-UVB treatments
Day 170*
No further therapy
Case Study 3
Participant C
Male, 56 years old, Skin Type IV
Diagnosed with vitiligo in 1999.
PHYSICIAN’S REPORT: First repigmentation seen around day 42, considerable repigmentation seen by day 106. Patient continued to repigment after conclusion of treatment protocol with no further therapy.
Day 0
Baseline
Day 134
7 afamelanotide implants
40 NB-UVB treatments
Day 308*
No further therapy
Case Study 4
Participant D
Male, 56 years old, Skin Type IV
Diagnosed with vitiligo in 1986.
PHYSICIAN’S REPORT: Due to extensive depigmentation, patient is yet to fully repigment. Patient continued to receive NB-UVB treatment following the study and continued to repigment (not shown).
Day 0
Baseline
Day 140
7 afamelanotide implants
40 NB-UVB treatments
Case Study 5
Participant E
Male, 46 years old, Skin Type V
Diagnosed with vitiligo in 2004.
PHYSICIAN’S REPORT: Images demonstrate repigmentation of vitiliginous lesions on right forearm (top) and lower legs (bottom). The red outlines demonstrates the extent of the initially affected skin.
Day 0
Baseline
Day 140
7 afamelanotide implants
40 NB-UVB treatments
Market potential
The expanding network of Specialty Centers across the U.S. and Canada is treating more EPP patients, while being established with the future treatment of vitiligo patients in mind. This network is scalable at low cost. CLINUVEL’s presence at the AAD Meeting in Orlando in March 2025 generated a pipeline of over 100 new dermatologists interested in joining this network to treat patients. CLINUVEL aims to expand this network to 120 centers by 1 January 2026, sufficient to support initial commercial plans in vitiligo.
Based on data sources and a range of assumptions, CLINUVEL has provided an indicative model of the commercial roll out for vitiligo in the U.S.A., with expected revenues of US$490-570 million in the first two years of distribution.
Market potential
*Total vitiligo population FST IV-V-VI
†7-8 doses afamelanotide >90%, repigmentation 47,448
Initiatives to add scale
The development of the ACTH formulation NEURACTHEL® continues as a key priority with an objective to obtain regulatory approval to distribute it as a branded generic drug in the first instance.
The distribution of PhotoCosmetic products will also add to the scale of CLINUVEL’s business in the U.S.
The U.S. exemplifies the melanocortin house being built by CLINUVEL
The Melanocortin House
Summary
CLINUVEL has invested incrementally in scaling-up its U.S. commercial operations to meet the needs of larger patient populations. Access to a safe and effective repigmentation therapy would be transformative for many vitiligo patients with darker skin complexions (skin types III-VI). The clinical program continues, while the network of Specialty Centers is being built, the unique codes are ready to be used, the case reports to date are promising, and we are working with determination and focus to the day when systemic repigmentation becomes a reality for vitiligo patients.