Clinuvel FAQ

Clinuvel is aware of the theoretical potential of its drug to treat additional indications. At present, the company’s focus is on the five indications outlined on this website. Running clinical trials in multiple patient populations is an expensive and complex process, fraught with potential risk. To provide the best possible path to market for afamelanotide, Clinuvel has identified five indications and will focus on these at the present time. Any updates to our clinical development will be announced to the ASX and emailed to our subscriber database.

Clinuvel is testing SCENESSE® (afamelanotide) for the five clinical indications outlined above. SCENESSE® has not been tested for the indication Rosacea, and at present no clinical trials are planned for the foreseeable future. By subscribing to our database, you will receive company updates on any clinical trials. Drugs should only be used for specific disorders, after extensive trialing under clinical conditions, and under the supervision of a qualified healthcare practitioner.

Yes, Clinuvel commenced a clinical trial program in the US in mid 2009. In March 2010, the FDA allowed Clinuvel to extend its clinical program and commence a Phase II trial of SCENESSE® (afamelanotide) in erythropoietic protoporphyria (EPP).

Log onto http://www.clinuvel.com/vitiligo

Every clinical trial has inclusion and exclusion criteria which dictate which individuals can participate in a clinical trial. These criteria are determined by the company and/or physicians running the trial and approved by regulatory and ethical authorities.

Clinuvel is yet to announce inclusion and exclusion criteria for the pilot trial of SCENESSE® in vitiligo. It is anticipated that, once approved by regulatory and ethical authorities, the four centers involved in the trial will look to recruit trial participants from their existing patient groups. As a pilot study, the proposed trial will not look to recruit many participants.

Clinuvel does not recruit trial participants directly, as to do so may jeopardize the integrity of data generated in clinical trials. The company cannot respond to individual requests to be involved in the pilot study of SCENESSE® in nonsegmental vitiligo.

Clinuvel advises individuals with vitiligo to talk to their treating physician which is the best appropriate person to talk to for the different treatment options for their vitiligo, including clinical trials.

Once regulatory approval has been granted, further information will be made available on http://www.clinuvel.com/vitiligo on whether individuals can register their interest to participate in trials.

Please note that Clinuvel is no longer seeking 'healthy' volunteers for clinical trials. The company is unable to respond to requests to be included in trials for individuals who have not been diagnosed with one of the outlined clinical indications.

Selection for clinical trials is not undertaken by Clinuvel but by the physicians at the clinical trial sites. You would need to contact these physicians regarding your suitability. It is best to first discuss your personal interest in clinical trials with your treating physician or dermatologist, who is best placed to assess your suitability for a trial.

The clinical trials that Clinuvel is currently conducting for SCENESSE® (afamelanotide) are in specific patient groups and only for those patients diagnosed with one of the outlined clinical indications. As with all clinical studies there are strict inclusion and exclusion criteria for participating patients and only patients with designated diseases who meet these criteria are eligible for entry. By subscribing to our database, you will receive company updates on any clinical trials and the conditions of participation.

Anonymity is a key requirement for trial participation. By contacting Clinuvel, you may disqualify yourself from possible trial participation.

More information on Clinuvel's confirmatory EPP trials is available on the ClinicalTrials.gov website.

More information on Clinuvel’s AK/SCC trials with organ transplant recipients is available on the ClinicalTrials.gov website.

Clinuvel is not currently seeking volunteers for US clinical trials.

In January 2009, SCENESSE® (afamelanotide) was allowed IND status by the Food and Drug Administration (FDA) to commence clinical trials in the USA on humans. In March 2010, the FDA allowed Clinuvel to extend its US program and commence a Phase II trial of Scenesse in EPP. This trial completed in February 2011.

Individuals diagnosed with EPP who are interested in possible future trials are advised to discuss trial candidacy with their dermatologist or physician who can contact Clinuvel directly.

The American Porphyria Foundation also maintains a register of individuals in the US with EPP and maintains close contact with physicians working in the field.

Clinuvel does not recruit patients directly for trials. Anonymity is a key requirement for trial participation. By contacting Clinuvel, you may disqualify yourself from possible trial participation.

The mechanics the vitiligo are still poorly understood. Recent research studies have highlighted numerous α-MSH and MC1R abnormalities in the blood and skin of patients with vitiligo, demonstrating the important role of α-MSH in the disorder.

SCENESSE®, which mimics the effects of the naturally occurring hormone α-MSH, is expected to restore this deficient system, and thus provide efficient therapeutic repigmentation for the patients.

SCENESSE® as an adjunctive therapy with NB-UVB phototherapy may also speed up and assist the repigmentation process initiated by phototherapy and possibly reduce the doses of NB-UVB radiation. Clinuvel has created a video to help explain this process in greater depth

The company has received significant input and encouragement from leading vitiligo experts worldwide for the use of SCENESSE® as a repigmentation therapy and eagerly awaits the chance to assess the drug in nonsegmental vitiligo.

It is anticipated that SCENESSE® (afamelanotide) will be administered by medical practitioners and delivered as a subcutaneous injection of a bioabsorbable implant that is approximately the size of a grain of rice. This type of underneath the skin administration allows the dosage of the drug to be carefully measured by the medical practitioners who prescribes it. Click here for more information.

At present Clinuvel is only looking for trial volunteers in the clinical indications listed on the site. Although we have conducted ‘healthy’ trials from time to time to test the efficacy or safety ofScenesse, we are no longer seeking volunteers for such trials.

In August 2010, Clinuvel announced it intends to commence a pilot Phase II study of SCENESSE® in individuals diagnosed with nonsegmental, or generalised, vitiligo. For more information, please see http://www.clinuvel.com/vitiligo, this short video from Clinuvel explaining how SCENESSE® will be used in nonsegmental vitiligo and the company’s formal announcement on its vitiligo program.

Clinuvel’s focus in clinical trials is ensuring the safety and efficacy of afamelanotide. To date, no serious drug related events have been recorded in published studies using the SCENESSE® (afamelanotide) implant. Clinuvel is continuing to trial the implantable product to prove the safety of its product under clinical conditions and strict supervision by medical experts

Currently SCENESSE® is only available by a physician’s prescription in Italy under a law called 648/96. The 648/96 listing allows Italian physicians to prescribe SCENESSE® to Italian citizens diagnosed with erythropoietic protoporphyria (EPP). For more information on this scheme, see this webpage.

Clinuvel has not tested SCENESSE®  (afamelanotide) for human use under the age of 18. As no clinical trials have been run in children, it is not possible to declare if the drug is suitable for their use. Clinuvel intends to begin pediatric studies in the future to determine if afamelanotide can assist individuals under 18, however no such research currently exists.

No. SCENESSE® (afamelanotide) is often confused with illegal, untested and unregulated chemicals offered online for self-administration.

The confusion lies in that vendors present injectable tanning products as SCENESSE® . In reality both the molecule and formulation of SCENESSE® have been optimised and altered and is not related to counterfeit products offered nowadays as Melanotan I and II.

Peptides are sensitive molecules consisting of particular amino acids which need diligent and pure synthesis. During the decade of development of SCENESSE® various proprietary manufacturing processes have led to the current optimised product.

Clinuvel’s formulation titrates the available quantities of SCENESSE® in the human body whereby maximum and minimum dosing has been carefully evaluated over the years. At picomol quantities, SCENESSE® is targeting specific skin cells to activate the process of biochemical photoprotection. SCENESSE® has no effect on other organs or the brain. SCENESSE® does not cause increased weight loss or libido.

It is a well known phenomenon that the general public ‘falls in love’ with a drug before it reaches approval. However, there is no foundation for the recreational use of SCENESSE® as a self-injectable. Clinuvel’s proprietary drug is being administered by physicians for the purpose of medicinal photoprotection of the skin.

Clinuvel’s pioneering work aims to provide photoprotection, that is to assist those who suffer from three UV related skin disorders and two cancer related treatments. The five clinical indications that Clinuvel is currently testing SCENESSE® (afamelabotide)for are:

• Erythropoietic Protoporphyria (EPP) - absolute sun intolerance, intolerable pain suffered when exposed to light by between one in 200,000 and one in 750,000 people. SCENESSE® is currently in Phase III trials in Australia, Switzerland and Europe for EPP.
• Phototoxicity associated with Photodynamic Therapy (PDT) for cancer – a debilitating photosensitivity to sunlight and artificial light (skin and eyes) that can last up to 90 days after PDT treatment.
• Polymorphous Light Eruption (PLE/PMLE) – likened to sun poisoning. Suffered by 10 -20% of Population. SCENESSE® is currently in Phase III trials in Australia and Europe for PLE.
• Actinic Keratosis (AK), a precursor to skin cancers and Squamous Cell Carcinoma (SCC) - non-melanoma skin cancers - in organ transplant patients. Organ transplant patients who are fair skinned are 65 to 100 times more likely to develop skin cancer on prescribed immune-suppressant medication following their transplant, compared to the general population. SCENESSE® is currently in Phase II trials in Australia and Europe for AK/SCC.
• Solar Urticaria (SU) – (acute) anaphylactic reaction to sun. In July 2009, Clinuvel announced results from Phase II trials in Europe for SU. You can view this announcement here.

For more information on each condition, click here.

Put simply, photoprotection is protection from the damage caused by UV and visible light. Clinuvel’s work aims to provide photoprotection to patients who are most prone to UV and light related disorders of the skin. SCENESSE® (afamelanotide) provides a biological barrier of melanin between UV light and skin, which reflects and refracts damaging light rays. Click here for more information.

Whenever a company or physician (a ‘sponsor’) wishes to assess a drug in a clinical trial, it must seek approval from the governmental departments or agencies which regulate or control new drugs in a given country or jurisdiction, such as the FDA in the US or the TGA in Australia. This process varies from country to country, but generally requires the sponsor to present a clinical study protocol, which outlines the specifics of the study, and provide scientific rational for their planned trial. These documents must show that the necessary regulatory guidelines related to clinical trials are being followed in the trial protocol.

The relevant regulatory agency will review the study protocol and scientific rationale and determine whether the study should proceed. The protocol is also reviewed to ensure it meets Good Clinical Practice or GCP standard. This complete process often requires some negotiation between the sponsor, the physicians involved in the trial, and the regulator, to reach a satisfactory conclusion. Generally this process takes 1-3 months.

During or shortly after the regulatory approval process for the study protocol, the Ethics Committees at individual study sites will reviews the protocol to ensure it meets the ethical standards of the particular institute or hospital conducting the study.

To help with understanding the clinical trial process, Clinuvel has published a three part series on its blog entitled The Drug Development Process to read these posts, see part 1, part 2 and part 3. Clinuvel also maintains relevant information on Pharmaceutical Development on its website.

Clinuvel expects to complete the pilot study of SCENESSE® in vitiligo and publish results from this study during the second quarter of 2011 (1 April-30 June). As a public company listed on the Australian Securities Exchanges (ASX), Clinuvel will first announce its results to the ASX through a formal announcement, before posting them on the company’s website at http://www.clinuvel.com. On this site you can view previous examples of results being released by the company.

Clinuvel is currently relocating its US office following the approval of the expansion of its US clinical program for EPP to mirror that of the EU and Australia.

Pharmaceutical products are legitimately required to be developed according to international regulatory standards and guidelines, whereby evaluation of safety in human use - short and long term - is the most critical parameter for drug approval. Certainly when it comes to novel drugs the regulatory rigor is stronger than in proven molecules and therapies. While Clinuvel maximises its chances to obtain regulatory approval, the company and its stakeholders need to abide by the regulatory timelines and review processes. Patience is a virtue in this process, and no pressure can accelerate this regulatory process.

At present Clinuvel is on track to file for registration for its photoprotective, SCENESSE® (afamelanotide) , in Europe, Australia or Switzerland some time in 2011. Clinuvel is working hard to meet this target, however pharmaceutical development is not a linear process. Ensuring the safety and efficacy of the drug is Clinuvel’s primary concern and until these two endpoints are met, SCENESSE® will continue to be trialed by Clinuvel.

At present it is not possible to speculate on a market date in the USA. By following Clinuvel’s clinical progress through the corporate website and the online mailing list, you will be kept abreast of any updates on this timeline.

Clinuvel intends to commence the pilot trial at four leading vitiligo centers: two in the US and two in Europe. No other sites are being considered for this trial. Once regulatory and ethics approval has been granted, more specific information will be made available on these centers.

Clinuvel is an Australian biopharmaceutical company developing drugs for individuals with a range of dermatological conditions.

Clinuvel has focused primarily on a drug called SCENESSE® (afamelanotide) which is an injectable implant drug roughly the size of a grain of rice. The active ingredient in SCENESSE® is a peptide called afamelanotide. This is an analogue of a naturally occurring hormone called alpha-Melanocyte Stimulating Hormone (α-MSH). In nature, α-MSH is our body’s response to skin damage from ultraviolet (UV) light. α-MSH binds to receptors (specifically melanocortin 1 receptor or MC1R) in the skin on particular skin cells, melanocytes, which in turn activate our skin’s pigment, melanin. Melanin provides skin cells with color as well as a biological barrier to protect them from UV and light. The skin is visibly darkened when melanin production is activated by α-MSH.

Afamelanotide’s chemical structure differs slightly from natural α-MSH: it has been altered to increase the peptide’s half-life (increasing the length of time it can exist in the blood stream from seconds to minutes) and to increase its ability to bind to MC1R on the melanocyte. As a result, afamelanotide is able to mimic the effects of the natural occurring α-MSH, with a longer lasting effect and increased potency.

Clinuvel has refined afamelanotide’s formulation and chemical structure to reach the current drug formulation: a 16mg afamelanotide controlled release implant, SCENESSE®. SCENESSE® has been formulated to achieve the optimal dose for activating melanin production systemically, while minimizing exposure to the drug and thus reducing possible side effects.

SCENESSE® is administered by physicians in the fatty tissue just above the hip. Approximately two days after administration, SCENESSE’s visible effect can be seen: the skin becomes darker. The drug is only present in the body for approximately ten days however the effect of the drug is seen for 60 days. Hence, in current clinical trials, SCENESSE® is administered once every 60 days. To date, nausea and headaches are the primary side effects reported in clinical trials however the drug has been safely tested in approximately 550 individuals with no reports of serious adverse effects.

For the past five years, SCENESSE® has been trialed for a number of UV and light related skin disorders (conditions called photodermatoses). The most advanced of these programs is for an indication called erythropoietic protoporphyria (EPP), a rare metabolic disorder which causes absolute light intolerance. SCENESSE® has been shown to reduce the number and severity of phototoxic reactions in late stage trials of the drug in individuals with EPP and a confirmatory program is underway in the US and Europe. Pending the outcome of this confirmatory program – due to complete in the first quarter of 2011 – Clinuvel expects to file SCENESSE® for Marketing Authorisation Approval (MAA) with European regulators.

In August 2010, Clinuvel announced it would commence a new program for SCENESSE®, trialing the drug for the first time as a repigmentation therapy in individuals diagnosed with nonsegmental vitiligo. The company is currently making submissions for regulatory and ethics approvals to commence a pilot Phase II study in four centers, two each in the US and Europe, which will evaluate SCENESSE’s ability to repigment skin both as a standalone therapy and as an adjunctive therapy with narrow-band ultraviolet B (NB-UVB) light therapy.

To read more about our work, log onto http://www.clinuvel.com

In late 2008, Clinuvel’s drug in development was assigned an International Non-proprietary Name (INN) by the World Health Organisation (WHO). The INN, also known as the generic name, afamelanotide can now be used universally to describe the active pharmaceutical substance Nle4-D-Phe7 alpha-Melanocyte Stimulating Hormone.

SCENESSE® (afamelanotide ) is a photoprotective drug in a development phase and not currently available on the market. It is currently being tested for its safety and efficacy in clinical trials.

In ethical pharmaceutical drug development a rigorous clinical program needs to be followed to comply with the requirements of regulators worldwide. Drug development therefore is not a speedy process. It takes time.

Ensuring the safety and efficacy of SCENESSE® is paramount to the program and Clinuvel is following an intensive clinical trial process in advance of pursuing regulatory approval and commercialisation to make this process as fast as possible.

SCENESSE® (afamelanotide) is a photoprotective drug, it is not a cosmetic tanning product.

It is a well known phenomenon that the general public ‘falls in love’ with a drug before it reaches approval. However, there is no foundation for the recreational use of SCENESSE® as a self-injectable. Clinuvel’s proprietary drug is being administered by physicians for the purpose of medicinal photoprotection of the skin.

Clinuvel believes that SCENESSE® has the potential to improve the quality of life of people worldwide who suffer from light related skin disorders and damage caused by UV radiation. We strongly support government and health care providers in their initiative to increase education about the dangers to UV-radiation and recommend that people follow their advice.