Clinuvel Pharmaceuticals

We are a global biopharmaceutical company committed to developing drugs for the treatment of a range of severe skin disorders.

Clinuvel’s lead compound, SCENESSE® (afamelanotide 16mg), a first-in-class drug targeting erythropoietic protoporphyria (EPP), has completed Phase II and III trials in the US and Europe. In December 2014 the European Commission granted SCENESSE® marketing authorisation under exceptional circumstances for the prevention of phototoxicity in adult patients with EPP. Further trials of SCENESSE® are underway in the pigmentary disorder vitiligo.

Our website has been developed to help share our expertise on the interaction of light and human skin as well as provide a thorough overview of our global drug development programs.

Latest Company Announcements

Discussion on US pathway to marketing approval for SCENESSE® Clinuvel Pharmaceuticals Limited (ASX: CUV; ADR:CLVLY; XETRA:DAX) today announced that it will meet the US Food and Drug Administration (FDA) to discuss the overall development of SCENESSE® (afamelanotide 16 mg) and the filing requirements for a New Drug Application (NDA) for the treatment of adult patients diagnosed with erythropoietic protoporphyria (EPP; absolute light intolerance). BACKGROUND Clinuvel has been in regular dialogue with the FDA throughout the SCENESSE® development program. The FDA’s Office for Orphan Products Development designated SCENESSE® as an orphan drug for EPP in 2008. Orphan drug designation recognises the potential of drugs to address unmet needs in rare disorders in the US1 and entitles sponsor companies to incentives throughout a product’s development, including seven years of market exclusivity in the US upon approval, a 50% tax credit for clinical trial expenditures, and exemptions from marketing application user fees (estimated at around US$2m). Clinuvel has formally met with the FDA on a number of occasions since 2009 to discuss the use of SCENESSE® in EPP and vitiligo. EPP patient representatives and clinical expert physicians have also met separately with the FDA to discuss the impact of EPP and the effects of treatment with SCENESSE® during clinical trials. In December 2014 SCENESSE® was granted marketing authorisation by the European Commission for the prevention of phototoxicity in adults with EPP (EU/1/14/969). FDA TYPE C MEETING In late September Clinuvel representatives will meet in person with the staff of the FDA’s Division of Dermatology and Dental Products (DDDP), part of the Center for Drug Evaluation and Research (CDER). The DDDP will be responsible for the scientific review of the NDA for SCENESSE®, having previously reviewed the Investigational New Drug (IND) application for SCENESSE® in 2009 and subsequent amendments to the IND. Clinuvel will use the Type C meeting, amongst other objectives, to identify the FDA’s current thinking on: -          the eligibility of SCENESSE® (afamelanotide 16 mg) for Accelerated Approval2 (subpart H); -          the requirements for a US post-authorisation Phase IV trial to monitor EPP patients’ treatment with SCENESSE® long term; and -          whether Clinuvel’s annual reporting obligations for SCENESSE® to the EMA would serve the FDA’s requirements. COMMENTARY “The European distribution of SCENESSE® has evolved into a complex and well considered program with a need to satisfy the EMA,” Clinuvel’s acting Chief Scientific Officer, Dr Dennis Wright said. “I expect that this program will be subjected to the FDA’s attention as long term follow up of patients is also a US requirement.” “We will learn whether the current European Risk Management Plan meets the FDA’s criteria. The pool of our data, patient reported outcomes, and physicians’ declarations of effectiveness all add to the strength of the dossier which we have established over more than a decade.” Dr Wright said. “The timing of discussions with FDA is excellent while, in parallel, the European distribution of SCENESSE® has now become reality,” Clinuvel’s Chairman, Mr Stan McLiesh said. “One never really knows how different regulatory agencies act, however the response of patients and physicians on both sides of the Atlantic has been uniformly in favour of the treatment. Given that the FDA would have access to a similar data package as submitted to the EMA, we anticipate similar discussions on risk, benefit, and clinical relevance of SCENESSE®.” - End - 1 The US Orphan Drug Act (1983) defines an orphan indication as one which affects fewer than 200,000 individuals in the USA or more than 200,000 individuals but “for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug”. 2As defined in 21 Code of Federal Regulations 314.510 subpart H and section 506(c) of the Federal Food, Drug and Cosmetic Act as amended by the Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA).   Media enquiries Lachlan Hay, Clinuvel (UK) Ltd. +44 1372 860 765      This email address is being protected from spambots. You need JavaScript enabled to view it. Nick Miles, Cabinet Privé de Conseils s.a. +41 22 321 4540      This email address is being protected from spambots. You need JavaScript enabled to view it. Ted Agne, The Communications Strategy Group Inc. +1 718 631 3117    This email address is being protected from spambots. You need JavaScript enabled to view it. Investor enquiries     This email address is being protected from spambots. You need JavaScript enabled to view it. Forward-Looking Statements This release to the Australian Stock Exchange and press contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause Clinuvel’s actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: that the FDA may require additional studies beyond the studies planned for product candidates or may not provide regulatory clearances, including for SCENESSE®; that the FDA may not provide regulatory approval for any use of SCENESSE® or that the approval may be limited; that Clinuvel may never file an NDA for SCENESSE® regulatory approval in the US; that the Company may not be able to access adequate capital to move its vitiligo programs forward; that the Company may not be able to retain its current pharmaceutical and biotechnology key personnel and knowhow for further development of its product candidates or may not reach favourable agreements with potential pricing and reimbursement agencies. About Clinuvel Pharmaceuticals Limited Clinuvel Pharmaceuticals Ltd (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) is a global biopharmaceutical company focused on developing drugs for the treatment of a range of severe disorders. With its unique expertise in understanding the interaction of light and human skin, the company has identified patient populations with a clinical need for photoprotection and another population with a need for repigmentation. These patient groups range in size from 5,000 to 45 million. Clinuvel’s lead compound, SCENESSE® (afamelanotide 16mg), a first-in-class drug targeting erythropoietic protoporphyria (EPP), has completed Phase II and III trials in the US and Europe, and has been approved by the European Commission for treating adults with EPP. Headquartered in Melbourne, Australia, Clinuvel has operations in Europe, the US and Singapore. For more information go to    http://www.clinuvel.com. About erythropoietic protoporphyria (EPP) EPP is characterised by severe phototoxicity (absolute intolerance to light) of the skin resulting in intolerable reactions, swelling, scarring and a state of distress. During phototoxic episodes patients experience long‐term swelling of the exposed body surfaces such as the face, hands and feet. A severe reaction – triggered by exposure to light – may result in hospitalisation. Patients do not respond to any analgesics or medication and following light exposure are typically unable to function. Due to the known risk to light and UV, patients often lead lifelong an isolated indoor life deprived of normal activities. Clinuvel is an Australian biopharmaceutical company focussed on developing its drug SCENESSE® (afamelanotide 16mg)for a range of clinical disorders with unmet need. Pharmaceutical research and development involves long lead times and significant risks. Therefore, while all reasonable efforts have been made by Clinuvel to ensure that there is a reasonable basis for all statements made in this document that relate to prospective events or developments (forward-looking statements), investors should note the following: actual results may and often will differ materially from these forward-looking statements; no assurances can be given by Clinuvel that any stated objectives, outcomes or timeframes in respect of its development programme for SCENESSE® can or will be achieved; no assurances can be given by Clinuvel that, even if its development programme for SCENESSE® is successful, it will obtain regulatory approval for its pharmaceutical products or that such products, if approved for use, will be successful in the market place. Level 5, 160 Queen Street  T +61 3 9660 4900             www.clinuvel.com Melbourne, Victoria 3000F +61 3 9660 4999 Australia

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 2015-08-26

Breakthrough treatment for orphan genetic disorder presented to the general medical community EXECUTIVE SUMMARY SCENESSE® published as innovative treatment in erythropoietic protoporphyria (EPP) Combined analyses of Phase III studies show significant improvement in the duration of patient sun exposure without phototoxicity New England Journal of Medicine: highest “impact factor” medical journal Pivotal studies led to December 2014 European marketing authorisation Melbourne, Australia and Leatherhead, UK Clinuvel Pharmaceuticals Ltd (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) today announced that results from its pivotal Phase III studies of SCENESSE® (afamelanotide 16mg) in the orphan genetic disorder erythropoietic protoporphyria (EPP) have been published in the New England Journal of Medicine (NEJM, 2015, vol 375: 48-59, publisher’s embargo lifted at 17:00 EST, July 1). COMMENTARY “Despite more than 40 years in the field it was only while participating as a senior physician in trials with afamelanotide that I gained insight into the major restrictions that EPP places on the daily activities of patients,” said Prof Paul Wilson, Emeritus Professor of Internal Medicine, Netherlands Porphyria Centre, Erasmus MC Rotterdam, and senior co-author of the NEJM paper. “The effect of afamelanotide on diminishing these restrictions and on patient quality of life is dramatic – even more dramatic than has been captured by the trial assessment tools – and a great advantage of the drug is that side effects are minimal.” “The approval of afamelanotide by the European Commission following these trial results represents a major breakthrough for EPP patients. As European porphyria physicians we now enter the next phase of making the drug available to our adult patients,” Prof Wilson said. “The trial results show that the afamelanotide treatment transforms the lives of EPP patients, in essence that is the ultimate goal of having a company and academia working on new technology for 10 years,” said Dr Robert J Desnick, Dean for Genetic and Genomic Medicine and Professor and Chairman Emeritus of the Department of Genetics and Genomic Sciences at Mount Sinai School of Medicine, New York, and the corresponding author for the NEJM paper. “I’m optimistic that afamelanotide will be available to US patients in the future and delighted that European patients will soon have access to a much-needed treatment.” “Today’s publication in the NEJM is as big an event for physicians as it is for patients: the most prestigious medical journal acknowledges a cutting-edge treatment for light-intolerant patients. Beyond EPP there is a great deal of potential for afamelanotide and similar melanocortin-based therapies. The immediate and most obvious targets are translationally photosensitive and light-induced disorders where no effective therapeutic options exist,” Dr Desnick said. “To evaluate SCENESSE® as an innovative drug in EPP, we worked closely with the world’s leading EPP experts to develop the clinical trials’ designs and assessment tools to assess the benefits of this treatment for patients,” Clinuvel’s Director Clinical Affairs, Dr Emilie Rodenburger said. “By measuring the symptom free time patients could spend in direct light, we sought to evaluate the impact treatment with SCENESSE® could have on enabling a normal, risk-free life. Despite all the challenges faced, I am very pleased for all patients and physicians that we persevered.” “The lead NEJM authors, Drs Langendonk and Balwani, deserve particular recognition for their initiative and expertise in rare and metabolic disorders,” Dr Rodenburger said. ANALYSES OF CUV029 AND CUV039 IN NEJM The NEJM article, “Afamelanotide for Erythropoietic Protoporphyria”, reviewed results from two Phase III studies, CUV029 (Europe) and CUV039 (US) conducted across 15 specialist centres. Combined analyses of 168 EPP patients showed that SCENESSE® enabled those who received active drug to expose their skin to daylight and sun without incurring characteristic phototoxic burns, with statistically significant longer exposure seen in patients who received the active drug (p=0.04 in the US, p=0.005 in Europe). The quality of life of patients measured by an EPP-specific survey was compared between active and placebo recipients. The group receiving SCENESSE® showed marked improvement of quality of life at various time points (range of p The NEJM authors emphasised difficulties of evaluating a novel treatment in EPP, a disease which had not previously been subject to parallel placebo-controlled randomised clinical trials. Both studies evaluated the safety of SCENESSE®. Treatment-emergent adverse events – those considered related to afamelanotide – were mild or moderate in severity with no serious treatment-related adverse events identified. The NEJM authors concluded that “afamelanotide had an acceptable side-effect profile and improved tolerance to sunlight in patients with erythropoietic protoporphyria”. RELEVANCE OF SCENESSE® IN THE TREATMENT OF EPP EPP is a particularly complex genetic disorder. Patients experience intense fear of incurring phototoxicity due to light exposure; severe dermal burns can result from any exposure to light, resulting in prolonged distress and incapacitation. The visible wavelengths of light cause the characteristic phototoxicity. From childhood onwards EPP patients are forced to isolate themselves from all sun and light exposure. The ingrained behaviour to minimise light exposure dictates EPP patients’ lives. Afamelanotide, the active ingredient in SCENESSE®, acts as an agonist to the melanocortin-1 receptor and is part of the family of drugs known as melanocortins. SCENESSE® delivers 16mg of afamelanotide as a controlled-release implant activating the pathway to stimulate melanin in skin. As a result patients’ skin becomes visibly darker as soon as 48 hours after drug administration. Melanin reduces skin damage from light exposure by acting as an umbrella over cells. Afamelanotide and melanin also act as powerful antioxidants. As previously reported by Clinuvel, the majority of patients who received SCENESSE® have expressed that the drug provided them a freedom to lead a life they had not had before, enabling them to participate in outdoor activities without fear of severe incapacitating symptoms. EUROPEAN MARKET ACCESS SCENESSE® Based on all five EPP studies conducted with afamelanotide, in particular CUV029 and CUV039, SCENESSE® was granted European marketing authorisation on 22 December 2014 for adult EPP patients. The drug will be made available to European EPP patients as soon as the European Medicines Agency’s Pharmacovigilance Risk Assessment Committee has reviewed the Central European EPP Disease Registry. NEW ENGLAND JOURNAL OF MEDICINE (NEJM) The NEJM currently has the highest impact factor (54.42 in 2013/14) of all medical periodicals, an independent measure reflecting the frequency of citations of its publications. The NEJM’s published articles are subject to rigorous peer review. An earlier open-label study of SCENESSE® in EPP patients was published in the NEJM in 2009. CLINUVEL CONTRIBUTION In accordance with the NEJM Ingelfinger Rule, none of the article on both trials in EPP has been previously published in a medical journal. Although both clinical trials were sponsored by Clinuvel Pharmaceuticals, Clinuvel was not involved in the writing or editing of the manuscript. Clinuvel does not financially incentivise physicians or academics to publish its clinical trial results. REFERENCES Langendonk JG et al (2015). Afamelanotide for Erythropoietic Protoporphyria. New Eng J Med. 373(1):48-59. Abstract online at   http://www.nejm.org/doi/full/10.1056/NEJMoa1411481. Harms J, Minder EI & Minder CE (2009). An alpha-melanocyte-stimulating hormone analogue in erythropoietic protoporphyria. New Eng J Med, 360(3):306-307. - End - Media note: Dr Robert J Desnick is available for interview through the Press Office at Mount Sinai School of Medicine, please contact Sid Dinsay via +1 212 241 9200 or This email address is being protected from spambots. You need JavaScript enabled to view it. . Media enquiries Lachlan Hay, Clinuvel (UK) Ltd. +44 1372 860 765     This email address is being protected from spambots. You need JavaScript enabled to view it. Nick Miles, Cabinet Privé de Conseils s.a. +41 22 321 4540     This email address is being protected from spambots. You need JavaScript enabled to view it. Ted Agne, The Communications Strategy Group Inc. +1 718 631 3117    This email address is being protected from spambots. You need JavaScript enabled to view it. Emma Power, Monsoon Media +61 3 9620 3333     This email address is being protected from spambots. You need JavaScript enabled to view it. Investor enquiries    This email address is being protected from spambots. You need JavaScript enabled to view it. About Clinuvel Pharmaceuticals Limited Clinuvel Pharmaceuticals Ltd (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) is a global biopharmaceutical company focused on developing drugs for the treatment of a range of severe disorders. With its unique expertise in understanding the interaction of light and human skin, the company has identified patient populations with a clinical need for photoprotection and another population with a need for repigmentation. These patient groups range in size from 5,000 to 45 million. Clinuvel’s lead compound, SCENESSE® (afamelanotide 16mg), a first-in-class drug targeting erythropoietic protoporphyria (EPP), has completed Phase II and III trials in the US and Europe, and has been approved by the European Commission for treating adults with EPP. Headquartered in Melbourne, Australia, Clinuvel has operations in Europe, the US and Singapore. For more information go to   http://www.clinuvel.com. Clinuvel is an Australian biopharmaceutical company focussed on developing its drug SCENESSE® (afamelanotide 16mg) for a range of clinical disorders with unmet need. Pharmaceutical research and development involves long lead times and significant risks. Therefore, while all reasonable efforts have been made by Clinuvel to ensure that there is a reasonable basis for all statements made in this document that relate to prospective events or developments (forward-looking statements), investors should note the following: actual results may and often will differ materially from these forward-looking statements; no assurances can be given by Clinuvel that any stated objectives, outcomes or timeframes in respect of its development programme for SCENESSE® can or will be achieved; no assurances can be given by Clinuvel that, even if its development programme for SCENESSE® is successful, it will obtain regulatory approval for its pharmaceutical products or that such products, if approved for use, will be successful in the market place. Level 5, 160 Queen Street  T +61 3 9660 4900             www.clinuvel.com Melbourne, Victoria 3000F +61 3 9660 4999 Australia

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 2015-07-01

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Clinuvel Latest News

Clinuvel issues social media warning

August 26, 2015 Clinuvel Pharmaceuticals today issued a warning regarding potentially fraudulent use of social media accounts across a number of different online platforms. As global attention towards the Company’s program for SCENESSE® (afamelanotide 16mg) increases it is an unfortunate reality that creative technologies have been used by individuals purporting to ...

Vitiligo Latest News

SCENESSE® vitiligo study results published in JAMA Dermatology

Results from Clinuvel's first Phase II study of SCENESSE® (afamelanotide 16mg) have been published in the peer-reviewed journal JAMA Dermatology. Access the article abstract Read Clinuvel's release Watch an interview with CUV102 investigator Dr Henry Lim...

Clinuvel Spotlight

Company History

The technology behind Clinuvel’s lead drug, SCENESSE® (afamelanotide 16mg), has been in research and development since the 1980s when a small team of scientists set out to investigate methods of preventing skin cancer in humans. Clinuvel licensed this technology and has spent over A$20million optimising the chemistry of afamelanotide (the active ingredient in SCENESSE...

Clinuvel Blog Posts

In 2010, Clinuvel announced that it would commence a clinical trial of a new drug for vitiligo. In late 2012 the first treatment results...

The biennial Porphyrins & Porphyrias conference is the only international meeting of global key opinion leaders on the group of...

Today, February 28, marks Rare Disease Day: an annual event to help highlight the effects of rare diseases on individuals, families and the...

Clinuvel Webcasts

What can vitiligo patients do? - Lee Thomas and Dr ...

Taking on Vitiligo, a depigmentation disorder of the skin. In 2010, Clinuvel ventured into an area of dermatology where no other pharmaceut...

The future of vitiligo treatment - Lee Thomas and D...

Taking on Vitiligo, a depigmentation disorder of the skin. In 2010, Clinuvel ventured into an area of dermatology where no other pharmaceut...

Vitiligo trials of SCENESSE® - Lee Thomas and Dr Ph...

Taking on Vitiligo, a depigmentation disorder of the skin. In 2010, Clinuvel ventured into an area of dermatology where no other pharmaceut...

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