MELBOURNE, Australia–(Business Wire)–
A new Australian drug has been shown to assist in protecting patients with a
rare genetic disorder that makes their skin toxic to light and UV, and
previously forced them to live in the shadows.
Results released today from a Phase III trial of the drug SCENESSE, conducted by
Melbourne-based Clinuvel Pharmaceuticals, have shown that the drug has the
ability to reduce and prevent painful phototoxic reactions experienced by
patients with erythropoietic protoporphyria (EPP).
The 12 month European and Australian study of SCENESSE (generic name
afamelanotide) is the first large scale study of a preventative drug for
patients with EPP, dubbed an ‘orphan’ disease due to its rarity and severity.
Clinuvel had to develop new study methodology with global EPP experts to be able
to successfully evaluate SCENESSE as a protective treatment.
EPP is characterised by intolerable pain: patients` skin burns, blisters and
scars when exposed to normal levels of light and sun. The disease is incurable
and affects patients for life. As a result, EPP patients are forced to live
indoors or to ‘shadow jump’ when outside; living so as not to expose their skin
to a ray of light. It is estimated that 10,000 individuals worldwide are
afflicted with EPP.
“Porphyria is a very severe disease causing great pain to patients when their
skin, even briefly, is exposed to light, therefore changing their Quality of
Life’ said Dr Gianfranco Biolcati, world expert in the disease and head of the
Italian Porphyria Centre at San Gallicano Dermatology Institute – IFO – in Rome
where 22 patients underwent treatment with SCENESSE in the trial. “Until now
there has been no effective treatment for EPP.”
Analysis of results from the Clinuvel study showed that SCENESSE treatment
significantly reduced the average daily pain severity scores experienced by EPP
patients compared to placebo.
Further results suggested that treatment with SCENESSE allowed patients to
expose their skin to sunlight and spend more time outdoors; previously unheard
of in EPP. Importantly, SCENESSE was well tolerated by all patients with no
serious safety issues identified. Complete results from the study will be
presented at the 19th Congress of the European Association for Dermatology and
Venereology in Gothenburg, Sweden, in October.
“Since commencing treatment with SCENESSE, we have seen marked improvements in
EPP patients` abilities to lead normal lives, without fearing pain during
exposure to the sun,” Dr Biolcati said. “As a physician, living this experience
is, for me, a great satisfaction and reward.”
“We have shown mathematically what we already learned from anecdotal reports
from our clinics: SCENESSE has great potential to help those patients with a
genuine medical need for protection from UV and light,” said Dr Hank Agersborg,
Clinuvel`s Chief Scientific Officer. “Perhaps more importantly, we have again
seen that the drug is safe for these patients longer-term.”
Clinuvel`s CEO, Dr Philippe Wolgen, said that the results were very positive for
EPP patients and the company`s overall program for SCENESSE.
“We are confident of being able to present an adequate dossier for regulatory
review. Board and management will convene around the table the in the coming
weeks to determine the optimum timing of filing,” Dr Wolgen said.
“The past four and half years this team has shown diligence and prudence in the
way it works and approaches complex issues, and the same approach will be
applied in the European and US filing process to ensure we stand the best chance
of success for EPP patients and our investors.” Dr Wolgen said.
- End –
Editor`s note: a complete release on the results from this study, including
statistical significance figures can be found at http://www.clinuvel.com
About SCENESSE (afamelanotide)
SCENESSE is a first-in-class therapeutic being developed by Clinuvel, with the
generic name (or INN) afamelanotide. An analogue of α-MSH, SCENESSE is a linear
peptide which activates the skin to release eumelanin, the dark pigment which is
known to have photoprotective properties (providing skin protection against
light and UV radiation). SCENESSE is administered underneath the skin as a
dissolvable implant approximately the size of a grain of rice. SCENESSE has been
approved under law 648/96 in Italy, allowing it to be prescribed to patients
prior to formal approval in Europe; a program initiated by the Italian Porphyria
Centre at San Gallicano Hospital, Rome.
SCENESSE is a registered trademark of Clinuvel Pharmaceuticals Ltd. For more
information see scenesse.com.
About Erythropoietic Protoporphyria (EPP)
Porphyrias are a group of inherited disorders with enzymatic deficiency in the
blood synthesis pathway (also called porphyrin pathway). They are broadly
classified as erythropoietic porphyrias based on the site of the overproduction
and main accumulation of porphyrin. They manifest with either skin problems,
neurological complications or gastro-intestinal problems (occasionally all).
EPP is a rare genetic disease found in people with fair skin. It is
characterised by severe phototoxicity (or intolerance to light) of the skin
resulting in intolerable pain, swelling, and scarring, usually of the hands and
face. The pain experienced and expressed by EPP patients when their skin is
exposed to light is reported as intolerable. EPP patients are often forced to
remain indoors, severely affecting their quality of life.
About Clinuvel Pharmaceuticals Limited
Clinuvel Pharmaceuticals Ltd is a leading and innovative Australian company
focused on the development of SCENESSE (afamelanotide), its proprietary
first-in-class photoprotective drug. Clinuvel has identified five groups of
patients with a clinical need for photoprotection. Currently, Clinuvel is in its
final stages to complete testing of SCENESSE in Phase II and III trials in
Australia, Europe and the United States. Clinuvel`s ongoing focus is to
demonstrate the safety and efficacy of SCENESSE. Pending positive clinical
results, Clinuvel aims to file SCENESSE for its first market approval for the
orphan indication porphyria (EPP).
Clinuvel is currently testing SCENESSE in five clinical indications:
Indication Description Clinical Trial Status
Erythropoietic Protoporphyria Absolute sun/UV intolerance Phase III trial full reported July 2010
(EPP) Confirmatory Phase III trial approved August 2009
Actinic Keratosis (AK) and Skin cancer in transplant patients Phase II trial
Squamous Cell Carcinoma (SCC) in Organ Transplant Recipients (OTRs) started October 2007
Polymorphic Light Eruption Severe sun/UV poisoning Phase III trial preliminary results reported December 2009
(PLE / PMLE)
Solar Urticaria Acute anaphylactic reaction to sun/UV Phase II trial results
(SU) reported July 2009*
Photodynamic Therapy (PDT) – Phototoxicity following cancer treatment Phase II trial results
systemic reported December 2009*
*Program deferred February 2010.
Phase I and II human clinical trials using SCENESSE have demonstrated that the
drug is well tolerated and no significant safety concerns have been identified
to date. Following successful conclusion of the development program, Clinuvel
will work closely with global regulators to facilitate marketing approval of
SCENESSE. For more information see clinuvel.com.
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
* 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
* 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
Australia: Lachlan Hay, Head of Global Network and Communications, T: +61 3 9660
Europe: Daniela Schaefer, Head of Business Operations Europe, T: +41 44 253 75
Copyright Business Wire 2010