Announcement • Jul 13
Clinuvel Pharmaceuticals Limited Receives Health Canada Notice of Compliance for Scenesse for Prevention of Phototoxicity in Adult Patients with Erythropoietic Protoporphyria Clinuvel Pharmaceuticals Limited’s novel drug SCENESSE (afamelanotide) has been granted a Notice of Compliance (NOC) by Health Canada for the prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). The NOC acknowledges the compliance of the SCENESSE new drug submission with Section C.08.002 and C.08.005 of the Canadian Food and Drug Regulations and grants Clinuvel Pharmaceuticals Limited the right to market SCENESSE in Canada. SCENESSE is the only treatment for EPP to have received marketing authorisation – recognising the drug’s clinical benefit, safety and quality – from any regulatory body worldwide. The Canadian approval follows marketing authorisations in Europe (2014), the U.S.A. (2019) and Australia (2020). Clinuvel Pharmaceuticals Limited has built a network of Specialty Centers across North America to treat EPP patients following the drug’s approval by the U.S. Food and Drug Administration in 2019. Five Centers are already trained and accredited in Canada and have treated EPP patients with SCENESSE under special access arrangements. EPP is a rare metabolic disorder which causes debilitating phototoxic reactions and burns following brief exposure to visible light, particularly sunlight. It is estimated to affect 5,000-10,000 individuals globally, including approximately 1 in 140,000 individuals in Canada. SCENESSE contains 16mg of the peptide afamelanotide, an analogue of a naturally occurring hormone which stimulates the production of melanin in skin. The drug provides a photoprotective effect, preventing the penetration of light through skin as well as acting as a strong antioxidant. Over 21,000 doses of SCENESSE have been administered to EPP patients worldwide, with the longest treated patients receiving up to 20 years of continuous therapy under expert care. SCENESSE (afamelanotide), is approved for commercial distribution in Europe, the U.S.A., Canada, 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). The drug has been administered over 21,000 times to EPP patients across clinical trials, compassionate and special access programs, and marketing authorizations. SCENESSE is only prescribed and administered by trained and accredited Specialty Centers (North America) and EPP Expert Centres (Europe), with the subcutaneous implantation occurring in an outpatient setting. SCENESSE has maintained a positive safety profile for up to two decades in EPP patients. Most commonly reported adverse events in clinical trials were headaches, nausea, implant site reactions, oropharyngeal pain, cough, fatigue, skin hyperpigmentation, dizziness, melanocytic nevus, respiratory tract infection, somnolence, non-acute porphyria and skin irritation. Announcement • Apr 24
Clinuvel Pharmaceuticals Limited Receives Final European Scientific Advice for Pivotal Phase III Vitiligo Study Clinuvel Pharmaceuticals Limited announced that the European Medicines Agency has provided final scientific advice on the design of the planned pivotal Phase III CUV107 study and anticipated evaluation of evidence of Clinuvel’s drug SCENESSE (afamelanotide) as a systemic vitiligo treatment. Following over 12 months of interaction, two formal submissions and a Discussion Meeting with the European Medicines Agency’s Scientific Advice Working Party, the Committee for Medicinal Products for Human Use has issued Scientific Advice on Clinuvel’s program evaluating SCENESSE as a systemic therapy for adults and adolescents with non-segmental vitiligo. The European Medicines Agency proposed to evaluate the efficacy of SCENESSE in vitiligo based on a regulatory approach of “totality of evidence”, with T-VASI50 the primary endpoint while patient reported outcomes and clinical data from the other vitiligo studies with SCENESSE will be evaluated for final analyses of efficacy and safety. Given the importance of patients’ perceptions of visible change in pigmentation, the European Medicines Agency emphasised that the assessment by vitiligo patients themselves would play an important role in the final evaluation when vitiligo data from CUV107 and previous studies would be submitted for marketing authorisation. Photographic evidence of change from baseline will be used to assess primary and secondary endpoints, T-VASI50 and F-VASI75, with an array of other related secondary objectives. Five patient and physician surveys are integrated in the study to record Patient Reported Outcomes. The European Medicines Agency advised that patients of darker skin colours (Fitzpatrick IV-V-VI) would benefit from systemic treatment first, since the visibility of disease – due to the contrast between unaffected skin and vitiligo lesions – is most pronounced in these patient groups. Scientific Advice provided by the European Medicines Agency is non-binding but good practice and documented in the final European Public Assessment Report issued at the conclusion of a marketing authorisation evaluation. Clinuvel will use T-VASI50 - the proportion of patients who achieve 50% or more repigmentation of the total body excluding hands and feet during the study – as the primary endpoint; this will be indicative but not determinative for efficacy. F-VASI75 - the proportion of patients who achieve 75% or more repigmentation of head and neck (excluding lips) and excluding hands and feet during the study – is being used as a secondary endpoint; this will be indicative but not determinative for efficacy. Vitiligo is believed to be a multifactorial disorder with immune components in some cases resulting in progressive loss of pigment (melanin) of the skin and associated with a severe impact on quality of life. It affects an estimated 0.5%-2% of the general population, with no approved therapies for patients with extensive depigmentation (affecting >10% of total body surface area). Announcement • Feb 19
Clinuvel Pharmaceuticals Limited to Report First Half, 2026 Results on Feb 26, 2026 Clinuvel Pharmaceuticals Limited announced that they will report first half, 2026 results on Feb 26, 2026 Announcement • Sep 03
Clinuvel Pharmaceuticals Limited, Annual General Meeting, Oct 17, 2025 Clinuvel Pharmaceuticals Limited, Annual General Meeting, Oct 17, 2025. Announcement • Aug 29
Clinuvel Pharmaceuticals Limited announces Annual dividend, payable on September 19, 2025 Clinuvel Pharmaceuticals Limited announced Annual dividend of AUD 0.0500 per share payable on September 19, 2025, ex-date on September 04, 2025 and record date on September 05, 2025. Announcement • Aug 21
Clinuvel Pharmaceuticals Limited to Report Fiscal Year 2025 Results on Aug 28, 2025 Clinuvel Pharmaceuticals Limited announced that they will report fiscal year 2025 results on Aug 28, 2025 Announcement • Jun 13
Clinuvel Pharmaceuticals Limited Announces Philippe Wolgen Return to the Business Full Time as Managing Director Clinuvel Pharmaceuticals Limited announced that Dr. Philippe Wolgen has returned to the business full time in his role as Managing Director and will focus in the short-term on advancing specific product development and corporate projects. Lachlan Hay will continue in the role of Acting Chief Executive Officer until the end of September 2025. Announcement • May 07
CLINUVEL Recruitment 200 Patients in Phase III Vitiligo Trial CUV105 CLINUVEL has met its recruitment target in its phase III trial (CUV105) of SCENESSE®? (afamelanotide 16mg) in vitiligo, with more than 200 patients enrolled. The last patient to enter the study is scheduled to complete screening in May 2025. First results from the study are expected in the second half of 2026. SCENESSE®? is being evaluated as a systemic repigmentation therapy for vitiligo patients, with a clinical focus on adolescent (12 years and above) and adult patients with darker skin types (Fitzpatrick III-VI). CUV105 is a randomised multi-centre Phase III trial being conducted in 37 study sites across three continents. Four previously released case studies - patients with skin type IV and varying disease duration - demonstrated repigmentation of vitiliginous lesions on the face or back after four weeks of commencing treatment with afamelanotide, and that some patients experience additional spontaneous repigmentation following the conclusion of the treatment protocol. A fifth case study shared, demonstrates repigmentation of lesions on the arms and legs in a skin type V patient with a 20-year history of the vitiligo. All case study patients have reported satisfaction with the treatment results and that afamelanotide was well tolerated with adjunct NB-UVB. Above: the fifth case study to be released from CUV105, a 46-year-old male with skin type V treated with seven SCENESSE®? implants and 40 NB-UVB sessions. Announcement • Feb 20
Clinuvel Pharmaceuticals Limited to Report First Half, 2025 Results on Feb 27, 2025 Clinuvel Pharmaceuticals Limited announced that they will report first half, 2025 results on Feb 27, 2025 Announcement • Oct 01
CLINUVEL Files Canadian New Drug Submission for SCENESSE® in EPP CLINUVEL has filed a New Drug Submission (NDS) to Health Canada, seeking approval for its novel photoprotective therapy SCENESSE® (afamelanotide) for the prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). If approved, SCENESSE® would be the first treatment for Canadian EPP patients. Health Canada review process: Health Canada’s Health Products and Food Branch (HPFB) reviews and approves medications for use in Canada, assessing the safety and efficacy of the products in the proposed indication, as well as the drug’s quality. Following a formal dossier validation period, the HPFB may complete the review of a new drug candidate within 300 days. Canadian Special Access Program ongoing: In 2023 CLINUVEL announced that the first Canadian EPP patient had received treatment with SCENESSE® under Canada’s Special Access Program (SAP). The SAP allows individual physicians to facilitate access to treatment for patients who have serious or life-threatening conditions and lack therapeutic alternatives. Patient treatment under the SAP continued without interruption prior to Health Canada’s review of the NDS. All Canadian patients treated under the SAP have received insurance coverage to support their treatment access. Two Canadian Specialty Centers have been trained and accredited to treat EPP patients with SCENESSE®. Further potential Canadian treatment centres have been identified to enable prompt treatment access pending regulatory and pricing approvals. To date, CLINUVEL has trained and accredited 85 Specialty Centers across North America. EPP affects approximately 1:140,000 individuals, with an estimated 280 EPP patients in Canada. SCENESSE® in EPP: systemic photoprotection: EPP is a rare genetic disorder which causes phototoxicity, debilitating reactions and burns following light exposure. CLINUVEL has spent nearly two decades developing SCENESSE® as the first treatment for EPP. The drug, administered as a controlled-release injectable implant every 60 days, stimulates the production of melanin in skin, protecting skin cells from visible and ultraviolet light (photoprotection) and acting as a strong antioxidant. Clinical and long-term post-marketing studies of SCENESSE® have shown that it can prevent and reduce the severity of phototoxic reactions, as well as improving patients’ quality of life. The drug has been approved for adults by the European Medicines Agency, US Food and Drug Administration (FDA), and regulatory authorities in Australia and Israel. To date, over 16,000 doses of SCENESSE® have been administered to EPP patients worldwide. Announcement • Sep 13
Clinuvel Pharmaceuticals Limited, Annual General Meeting, Oct 16, 2024 Clinuvel Pharmaceuticals Limited, Annual General Meeting, Oct 16, 2024. Location: the events centre at collins square, tower 2, level 5, 727 collins street, vic 3008, melbourne Australia Announcement • Aug 19
Clinuvel Pharmaceuticals Limited to Report Fiscal Year 2024 Results on Aug 29, 2024 Clinuvel Pharmaceuticals Limited announced that they will report fiscal year 2024 results on Aug 29, 2024 Announcement • Jun 28
Clinuvel Pharmaceuticals Limited Announces Change of Company Secretary Clinuvel Pharmaceuticals Limited advised Mr. Peter Vaughan will be appointed Company Secretary, effective 28 June 2024 until 6 August 2024 when a permanent Company Secretary is expected to start employment, with Mr. Darren Keamy stepping down from the role on28 June 2024. Announcement • Jun 19
CLINUVEL Announces A Novel Clinical Program CLINUVEL announced a novel clinical program evaluating afamelanotide as a treatment in early-stage Parkinson’s Disease (PD or Parkinson’s) in fair-skinned patients. The program objectives are to determine whether afamelanotide – through melanocortin-1 receptor (MC1R) activation – is able to lower a-synuclein (a toxin) in blood levels in PD patients, and positively affect neurons of the midbrain. MC1R is known to be a key receptor in brain and skin cells. In large studies, it was found that fair-skinned patients have a higher risk of PD associated with a malfunctioning MC1R. Since afamelanotide is known to optimise the function of the MC1R, it is hypothesised that the drug treatment would have a positive effect in PD by lowering a-synuclein, as recently demonstrated in preclinical studies.²¯³ Afamelanotide is marketed in Europe and the USA as SCENESSE® for patients diagnosed with erythropoietic protoporphyria (EPP). Evidence for use of afamelanotide in Parkinson’s Disease: Worldwide, individuals born with red hair and fair skin are found to have a loss of function of MC1Rs – expressed on skin and brain cells – and to carry an increased risk of Parkinson’s and melanoma. In various Parkinson’s models it has been illustrated that an MC1R-binding drug – such as afamelanotide – enables cellular protection against a-synuclein, a toxic substance found in the neurons of PD patients. In preclinical models, the use of afamelanotide has been shown to improve neurodegenerative conditions. Following decades of human use, it has been well established that afamelanotide strongly binds to MC1R and optimises cellular functions (signalling) through pharmacological activation. The CUV901 study is the first human study evaluating the effect of afamelanotide in PD as a therapeutic option. Study design – CUV901: The Phase IIa CUV901 study will evaluate six fair-skinned patients with early symptoms of PD who are not yet receiving medicinal therapy. The first objectives of the open-label study are to focus on the safety of afamelanotide, while determining a-synuclein in blood, and assessing visual changes in the midbrain. Secondary endpoints are to assess cognitive functions. The study design has obtained ethics and regulatory approval. Patients between 40 and 85 years old will receive 11 doses of 0.08 mg per kilogram body weight of afamelanotide on each day of drug administration, over a study duration of 56 days. The first patients are expected to enroll before the end of 2024. Announcement • Feb 16
Clinuvel Pharmaceuticals Limited to Report First Half, 2024 Results on Feb 22, 2024 Clinuvel Pharmaceuticals Limited announced that they will report first half, 2024 results on Feb 22, 2024 Announcement • Sep 29
Clinuvel Pharmaceuticals Limited Announces Board Changes Clinuvel Pharmaceuticals Limited announced that Non-Executive Director Sir Andrew Likierman, who has been a member of the Board of Directorssince April 2022, will resign from his position at the conclusion of the AGM to focus on his various academic and consultancy activities. Mr. Willem Blijdorp has announced he will step down as Chairperson of the Board of Directorswith effect from of 1 January 2024, pending his re-election as a Non-Executive Director at theCompany's AGM. The Board has commenced a review process, after which a new Chairpersonwill be announced. Announcement • Sep 13
Clinuvel Pharmaceuticals Limited, Annual General Meeting, Oct 31, 2023 Clinuvel Pharmaceuticals Limited, Annual General Meeting, Oct 31, 2023, at 10:00 AUS Eastern Standard Time. Announcement • Aug 30
Clinuvel Pharmaceuticals Limited Announces Dividend for the Full Year Ended June 30, 2023, Payable on September 20, 2023 Clinuvel Pharmaceuticals Limited announced ordinary fully paid dividend of AUD 0.05000000 per share for the full year ended June 30, 2023. Ex-date is September 5, 2023. Record date is September 6, 2023. Payment date is September 20, 2023. Announcement • Aug 28
Clinuvel Pharmaceuticals Limited to Report Fiscal Year 2023 Results on Aug 29, 2023 Clinuvel Pharmaceuticals Limited announced that they will report fiscal year 2023 results on Aug 29, 2023 Announcement • Feb 15
Clinuvel Pharmaceuticals Ltd Announces the Completion of Manufacture of the First cGMP Batch of PRÉNUMBRA® Instant Clinuvel Pharmaceuticals Ltd. announced the completion of manufacture of the first cGMP batch of PRÉNUMBRA® Instant, an immediate-release liquid injectable presentation of afamelanotide. The product will first be evaluated in patients who have suffered arterial ischaemic stroke (AIS). Pathway to market: The next stage is for manufacturing processes to be scaled up, followed by manufacturing validation work generating data for the regulatory dossiers to be submitted in the United States and European Union. Since data from preclinical studies and long-term clinical use of the SCENESSE® (afamelanotide 16mg) implant are available, a full development of PRÉNUMBRA® Instant is not required. Rapid validation of the specifications and batch release testing of PRÉNUMBRA® Instant is possible due to the extensive existing SCENESSE® dossier. The American and European pathway to marketing approval for PRÉNUMBRA Instant. PRÉNUMBRA® Instant, new dosage form CLINUVEL has completed the manufacture of clinical trial supplies of PRÉNUMBRA® Instant under Good Manufacturing Practice (cGMP) via an exclusive arrangement with a contract manufacturing organization. The use of the fast-releasing formulation of afamelanotide provides flexibility in acute care, allowing physicians to adapt the patient's dose according to individual needs. Specialized neurology centres have been selected and engaged as clinical trial sites for CLINUVEL's second study of afamelanotide in stroke patients (CUV803), which will be the first to prescribe the PRÉNUMBRA® Instant formulation. CUV803 will commence following regulatory and ethics consent. CLINUVEL's melanocortin portfolio: Having commercialised the first afamelanotide formulation - the 16mg SCENESSE® controlled-release implant - CLINUVEL has retained extensive expertise on the therapeutic potential of the drug, plus the broader melanocortin family. The development of melanocortins and their various formulations is part of the Company's strong focus, risk management, and diversification. Thereby, the red line running through R&D is to lead clinical development based on populations in highest need of treatment or care. Announcement • Feb 02
Clinuvel Pharmaceuticals Limited Announces First Positive Results from Study Evaluating the Protective Effects of Afamelanotide on Skin Exposed to Ultraviolet Radiation Clinuvel Pharmaceuticals Limited announced the first positive results from a study evaluating the protective effects of afamelanotide on skin exposed to ultraviolet (UV) radiation (CUV151). The study, conducted at Salford Royal Hospital, Manchester, showed that systemic treatment with afamelanotide decreased the UV-erythema dose-response following ultraviolet radiation (UVR), indicative of reducing the first signs of UV-induced DNA damage. Analyses of biopsies that were taken during the study, assessing the drug's influence on DNA repair capacity, are pending. Study design CUV151: The objective of the study was to assess the impact of afamelanotide on UV-induced DNA-damage and repair capacity in healthy volunteers with fair skin types (Fitzpatrick I-III)¹ by measuring: changes in UVR erythema dose response following treatment with afamelanotide; changes in minimal erythema dose (MED) following treatment with afamelanotide - an increased MED indicates a greater resistance to the mutagenic effects of UVR; the amount of DNA damage, and DNA damage repair following exposure to controlled doses of UVR. Manchester University is a renowned specialist centre which undertakes advanced clinical research in skin cancer, and its affiliate hospital (Salford Royal) was chosen for the study. Nine healthy adult volunteers were administered UVR by a solar simulator under controlled clinical conditions. Evaluation of the skin occurred before and after UVR. Results CUV151: From a safety perspective, afamelanotide was well tolerated with two patients reporting mild headache and one patient experiencing mild nausea. It was found that following UV irradiation, the UV-erythema (‘provoked sunburn damage’) dose response was reduced (p=0.018). The observed decrease of the UV dose-response indicates the reduction of DNA damage incurred following afamelanotide treatment. Consistent with earlier studies, skin melanin density increased following treatment with SCENESSE® (p<0.05). Relevance of the results: Scientific research has long focused on the identification of principal skin cancer risk factors, among which are DNA damage defects incurred due to solar radiation. Short exposure to UVR, and evidently a first sunburn, causes loss of DNA integrity through the formation of helical breaks, and the formation of single-strand dimers (cyclobutane pyrimidine dimers, CPDs), thereby increasing the risk of skin cancer(s). The scope of this study is limited to erythemal UVR and direct DNA damage. Following the use of solar-simulated UVR, reduction of UV dose-response, and increased melanisation shows the potential of systemically2 used afamelanotide to significantly reduce solar skin damage, erythema, and therefore first DNA lesions. These results in healthy subjects confirm the earlier results of study CUV156 in xeroderma pigmentosum (XP) patients, who suffer from the highest risk of developing skin cancers due to a defect in DNA-repair mechanisms (‘Afamelanotide Reduces DNA Photodamage in Xeroderma Pigmentosum’, 16 January 2023). The results in CUV156 showed a reduction in CPDs following dosing of afamelanotide. As seen in previous studies, in CUV151 it was confirmed that afamelanotide increases human skin pigmentation, which is strongly associated with photoprotection against systemic oxidative stress caused by solar radiation, and which may further reflect the drug's and melanin's antioxidative properties. Announcement • Jan 23
Clinuvel Pharmaceuticals Limited Announces Neuracthel Manufacturing Processes Advance Clinuvel Pharmaceuticals Limited announced an update on its commercial development of the analogue adrenocorticotropic hormone (ACTH), part of CLINUVEL's portfolio of melanocortin products. The Company will launch a range of ACTH products under the trade names NEURACTHEL® Instant and NEURACTHEL® Modified-release for patients with neurological, endocrinological, and degenerative disorders, with plans to file a regulatory drug master file (DMF) for ACTH in the second half of 2023. CLINUVEL is currently pursuing the development of three melanocortin-based pharmaceutical product lines globally: NEURACTHEL®, PRÉNUMBRA®, and SCENESSE®. Working closely with exclusive manufacturing partners, CLINUVEL completed method and process development work throughout H2 2022, including the establishment of critical process parameters serving the commercial product manufacturing. In the coming months work is planned for on production of the ACTH drug substance under current Good Manufacturing Practices (cGMP) standards at commercial scale, characterisation of the drug substance, and overall evaluation stability. Data from validation batches support the filing of a DMF with global regulatory authorities laying the foundation for the submission of marketing authorisation applications. Announcement • Jan 17
Clinuvel Pharmaceuticals Limited Shares the First Results of Phase II Study Evaluating Afamelanotide in Patients with Xeroderma Pigmentosum Clinuvel Pharmaceuticals Limited shared the first results of a phase II study (CUV156) evaluating afamelanotide in patients with xeroderma pigmentosum (XP), a genetic disease characterised by a defect in DNA skin repair (NER1 defect). Analyses showed a decrease in ultraviolet (UV) light-induced DNA skin damage following treatment. This study constitutes the first time globally that permission was granted to expose XPC patients to a novel systemic therapy. Despite living in fully shielded and isolated conditions, this population suffers from frequent skin cancer(s), resulting in a median life expectancy of 30 years. INTERIM RESULTS CUV156: The CUV156 study is conducted over ten weeks, with a six month follow up. Patients are administered six doses of afamelanotide as well as controlled UVB radiation on unexposed areas of the skin (buttocks), with DNA markers evaluated. UVB serves to evaluate tolerance (MED) to the point of inducing DNA skin lesions, cyclobutane pyrimidine dimers (CPDs), characteristic for photodamage. Skin biopsies are taken of UVB irradiated and non-irradiated anatomical sites before and after treatment and analysed through immunohistochemical staining (IHC; microscopic analyses). In the three patients, a reduction of CPDs was found, most specifically in the deeper layer of the skin (basal layer of epidermis). In two patients, the skin specimens showed an increase in p53 expression, indicating activation of natural defence mechanisms. P53 serves as a biological marker in man for suppressing tumour formation. In three patients, H2AX (a DNA marker5) showed an increase, indicative of the activation of cellular repair mechanisms of the skin. All three patients showed reduced erythema when increasing UVB dosing, whereby two showed an increase in MED, indicating the ability to tolerate higher UV doses without incurring `skin burns'. In all patients, an increase in melanin density (MD6) was seen, suggestive for the formation of skin pigmentation acting as a physical UV barrier. Overall, clinical assessment by the treating physicians was that afamelanotide provided effective systemic photoprotection in XPC patients. These first positive results justify further progression of the CUV156 study in XPC and the ongoing CUV152 study in XPV patients. CLINICAL RELEVANCE OF THE FIRST RESULTS: Due to inherited defects in the DNA repair process, the XP population is globally known to experience the highest risk of skin cancer development. A therapy providing systemic photoprotection, reducing photodamage, and assisting DNA skin repair nucleotide excision repair (NER) and base excision repair (BER) would be of high value to these patients. This therapeutic approach bears relevance for a wider population at higher risk of skin cancers. Melanocortin therapy would potentially benefit those affected by medical conditions, active in high-risk environments (reflective surfaces, high altitude, high UV intensity), or whose genetic make-up (those with non-pigmented skin, blue eyes, and fair hair colour) places them at higher risk of incurring solar damage. MODE OF ACTION AFAMELANOTIDE: Afamelanotide belongs to the family of proopiomelanocortins which exhibit a number of documented and published properties, such as the activation of melanin, the optimisation of cellular response (signalling) to UV skin damage, the assistance in DNA damage, the reduction of oxidative damage, reduction in oncosis (swelling), and decrease in inflammatory processes.