Pilot study to evaluate drug in pigmentary disorder affecting more than 45 million globally
Clinuvel Pharmaceuticals Limited (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) today announced that it will begin a pilot trial (CUV031) with its first-in-class drug SCENESSE® (afamelanotide) as a repigmentation therapy in the pigmentary disorder vitiligo. The trial will focus on patients diagnosed with nonsegmental vitiligo (NSV), the most common form of the disorder which affects approximately 45 million people globally.
During an investigators’ meeting on 21 August in New York, Clinuvel reached agreement with four of the world’s most prominent and expert centres specialising in vitiligo. Consensus was reached on the protocol and study design. The trial is scheduled to start in October, subject to obtaining the necessary European and US regulatory approvals.
SCENESSE® as repigmentation therapy in nonsegmental vitiligo
SCENESSE® is a first-in-class and innovative drug being developed exclusively by Clinuvel. The drug has been tested in approximately 550 patients in regulated clinical trials. SCENESSE® acts by increasing melanin levels in the skin attempting to shield against UV radiation (UVR) and sunlight. The drug is delivered by a small subcutaneous implant approximately the size of a grain of rice. Increased pigmentation of the skin begins to appear two days after implantation and lasts for up to two months.
This trial will evaluate SCENESSE’s ability to repigment areas of skin with and without the use of narrowband ultraviolet B (NB-UVB) phototherapy.
Phototherapy, mainly narrow band UVB (NB-UVB), has emerged as a mainstay of repigmentation treatment in individuals affected by vitiligo. NB-UVB utilises a localised light source to activate melanin in vitiliginous lesions of the skin. This therapy is known to effectively suppress the local immune response and accelerate the maturity of melanocytes in the area around hair follicles, which act as melanocyte reservoirs. This process leads to activation of melanin (pigment). Data from this pilot study will give Clinuvel insight into whether SCENESSE® as monotherapy is preferable to NB-UVB alone or whether the two treatments combined provide a superior clinical result.
Clinuvel's Chief Scientific Officer, Dr Hank Agersborg said:
“In this study we seek to evaluate SCENESSE® in perhaps in the most logical indication of all, nonsegmental vitiligo, a disease in which there is a reduction in functional activity of melanocytes. There is great expectation from the leading scientific experts in the field that SCENESSE® could actually better the currently inadequate treatment. The two US and two European centres selected are among the most advanced in the treatment of vitiligo.”
Clinuvel's CEO, Dr Philippe Wolgen said:
“The breakthrough in our thinking is as meaningful as the choice we made to make SCENESSE® available in vitiligo.
“About two years ago the first long-term results from phototherapy in vitiligo were seen. Frequent sessions of NB-UVB showed that repopulation of pigment producing cells either from the periphery or from the roots of the hairs in the skin was possible. This alone was a breakthrough, as previously it had always been thought that repigmentation of vitiligo was impossible, now we know that partial repigmentation is clinically possible with the assistance of narrowband UVB.
“Naturally, these academic developments piqued our interest, and we soon understood that the re-emergence of melanocytes in vitiligo would offer the pharmacological basis for SCENESSE® to assist, and possibly reduce, the doses of phototherapy.
“It is again clear that SCENESSE® attracts attention from the clinical and academic communities worldwide and, after years of clinicians petitioning for the drug to be used in vitiligo, Clinuvel has now found sufficient evidence to support the use of the drug in vitiligo. For patients and shareholders, today’s announcement marks one of the most exciting directions for Clinuvel.”
Vitiligo is a common skin disorder in which the pigment producing cells of the skin (melanocytes) are absent or inadequate. As a result, lighter depigmented patches of skin (target lesions) appear in different parts of the body due the lack of melanin (pigment). The exact cause of vitiligo is unknown, but it is generally recognised that an autoimmune component plays a part in this disease. Between 0.1-2% of the global population is affected by vitiligo and it affects all races. Vitiligo causes significant psychological and emotional distress.
Vitiligo is traditionally separated into two clinical forms: nonsegmental, or generalised, vitiligo (NSV) and segmental vitiligo (SV), which present with distinctive clinical features and natural histories.
NSV is the most common form of the disease, accounting for 72-95% of the cases. The vitiliginous lesions are usually symmetrically distributed and new patches may appear throughout the patient’s life. The disease is progressive with flare-ups. NSV is frequently associated with personal or family history of auto-immunity.
SV is characterised by a unilateral distribution that may totally or partially match a dermatome (area of skin with innervation from a single spinal nerve) and has an earlier onset and a rapid spread. It may account for 30% of childhood cases. Auto-immune association is rare with SV.
Vitiligo therapy is intended to arrest depigmentation or provide repigmentation of depigmented lesions. Many treatment options exist but many clinical challenges persist. Not all patients respond to available therapies and relapse is common.
Clinuvel's clinical focus for SCENESSE®
Nonsegmental vitiligo (NSV) will be the sixth indication to be clinically evaluated by Clinuvel for the novel drug SCENESSE®.
The company continues to advance its program for the rare light intolerance erythropoietic protoporphyria (EPP) in Phase II and III confirmatory trials in the USA and Europe. respectively. Positive results were announced from the first Phase III study of SCENESSE® in EPP in July 2010. Further trials continue to evaluate SCENESSE® as a photoprotective drug in Europe for patient with severe polymorphic light eruption and in Europe and Australia as a preventative for certain skin cancers in immune suppressed organ transplant recipients.
Pending ongoing safety and efficacy in trials, the company expects to file SCENESSE® for marketing authorisation approval (MAA) for EPP in Europe by 2011.
The company's program, including the pilot NSV trial, is fully costed and funded to its first MAA.
Clinuvel expects that, should the pilot trial NSV prove successful, the company will further pursue NSV in a larger patient cohort. It is anticipated that results from the pilot trial will be known by Q2 2011.
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Clinuvel is an Australian biopharmaceutical company focussed on developing its photoprotective drug, SCENESSE® (afamelanotide) for a range of UV-related skin disorders resulting from exposure of the skin to harmful UV radiation. 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