Clinuvel Newsletter May 2011 - Clinuvel Pharmaceuticals

Clinuvel Communiqué
MAY 2011
CONTENTS
CEO’S OVERVIEW
CEO’s Overview 1
Confirmatory EPP studies complete in the US & Europe 2
INSPIRE vitiligo program update 2
Guest bloggers and new video releases 3
Share price and financials 3
CLINUVEL REPRESENTATION
AT SCIENTIFIC & FINANCIAL EVENTS
RECENT:
• 29th Annual JP Morgan Healthcare Conference – San Francisco, US
(January 11-14)
• Clinuvel’s third Annual Scientific and Clinical Excellence Meeting
(ASCEM III) – Luzern, Switzerland (January 23-24)
• 4th Annual Sachs Associates European Life Science CEO Forum –
Zürich, Switzerland (January 25-26)
• 69th Annual Meeting of the American Academy of Dermatology –
New Orleans, US (February 4-8)
• BIO-Europe Spring 2011 – Milan, Italy (March 14-16)
• Porphyrias and Porphyrins 2011 – Cardiff, Wales (April 11-14)
• Generational Dermatology Summit – New York, US (April 29-May 1)
At a final stage of preparing Clinuvel’s regulatory filing in Europe,
many activities take place in-house to optimise a successful outcome.
To arrive at this stage is truly remarkable as we took less than five
years of development since starting the program in erythropoietic
protoporphyria (EPP).
In this newsletter, I shall take you back to the sequence of events the
past quarter.
We welcomed Eli Ishag as new Board member, providing the
Board additional commercial input at this stage of preparing the
commercialization of SCENESSE® (afamelanotide).
In January we organized the third ACSEM in Luzern, where world’s
leading clinicians, scientists and experts in the fields of alpha-MSH,
cell biology and dermal carcinogenesis attended. This meeting
has become a forum of excellence where the latest developments
and insights are being discussed and presented. Additionally, our
scientific teams exchange views on the application of afamelanotide
in the clinic. It is essential to Clinuvel’s existence to obtain regular
and unbiased feedback on the use of SCENESSE®.
This quarter we learned about the progressive adoption of
SCENESSE® by Italian EPP patients, whereby we recorded for the
first time sales of the drug in a European country. Feedback is
excellent, and patients and physicians report an increase demand for
SCENESSE®. Italy serves as a good benchmark for years to come.
UPCOMING:
• World Congress of Dermatology – Seoul, Korea (May 24-29)
• World Orphan Drug Summit – Frankfurt, Germany (May 31-June 1)
• XXIst International Pigment Cell Conference – Bordeaux, France
(September 21-24)
• 20th Congress of the European Association of Dermatology and
Venereology (EADV) – Lisbon, Portugal (October 20-24)
After discussions with FDA in January and February, we altered
the vitiligo program (INSPIRE) with separate European and
US trial protocols (CUV101 and CUV102). The US trial, which
commenced recruitment last week, will see patients administered
four afamelanotide doses to ‘active’ cohorts over six months. EU
centers, awaiting Ethics approval for a further amendment, will
administer six afamelanotide doses over six months. The combination
of SCENESSE® with narrowband UVB has set high expectations
(Continued on Page 2)

Clinuvel Communiqué May 2011
among the medical and patient community as witnessed by the high
online demand for information and we will provide further updates as
approvals warrant.
The highlight of the past weeks was undoubtedly our submission of
documentation to the European Medicines Agency (EMA) and
its respective committee COMP. Early May Clinuvel was invited to
attend a meeting in London to discuss the dossier on SCENESSE® in
the use of EPP. As you will know by now, the outcome was positive
and Clinuvel was given positive feedback on the quality of its dossier,
whereby the results of our confirmatory EPP studies (CUV029 and
CUV030) would be the final determinants of efficacy. After five
years of starting this program, the outcome summarises a collective
achievement from the Clinuvel teams across Australia, Europe and the
US. At times, and much to my personal frustration, to have to await
lengthy regulatory procedures the clock seemed to be ticking slower
than desired, however against industry benchmarks to be able to file
within five years of commencement is a great moment. Pending the
outcome of these clinical results, Clinuvel aims to lodge its European
filing before January 1 2012.
We are now awaiting the final results of CUV029 and CUV030
anticipated the next weeks to months. This will ultimately show us
whether SCENESSE® is effective in EU and US patients to prevent or
reduce their symptoms. Additionally, we will release PLE (CUV032)
results which will serve as safety data to our filing. An interim
analysis on CUV011, actinic keratosis and squamous cell carcinoma in
immunocompromised transplant patients, is under way and expected
this year.
The demand for our drug is high. We are all aware of this and our
relentless commitment is to demonstrate that SCENESSE® deserves
a position in the pharmaceutical list of reimbursable drugs. Clinical
response has been excellent; the challenge remains to obtain approval
in Europe first, followed by the US (FDA). Clinuvel has entered a final
stage, a unique position where not many companies have arrived.
I thank you for your patience and ongoing support, drug
development requires a long term view from all of us, but will be
rewarding for patients and investors.
Philippe Wolgen
CONFIRMATORY EPP STUDIES COMPLETE IN THE US &
EUROPE
Results from CUV029 and CUV030 are due in the coming months
and the company expects them to add to the first dossier for a
marketing authorisation application with the European Medicines
Agency. Discussions are still ongoing with the FDA to set the next
steps for Clinuvel’s US EPP program.
INSPIRE VITILIGO PROGRAM UPDATE
Vitiligo is a disease which causes white or off-white depigmented
skin lesions to appear on different parts of the body due to a loss of
melanin (pigment) production. This disorder may spread over time
and cause patients significant psychological and emotional distress.
In March, Clinuvel announced that the US Food and Drug
Administration (FDA) and the Italian Medicines Agency (AIFA)
had allowed the company to commence a pilot Phase II study of
SCENESSE® (afamelanotide) in patients diagnosed with nonsegmental
vitiligo, the most common form of the disease which affects over 45
million individuals globally.
The International SCENESSE® Pilot Repigmentation Evaluation
(INSPIRE) program will evaluate the efficacy of SCENESSE® as
a combination therapy with narrowband ultraviolet B (NB-UVB)
phototherapy compared to NB-UVB alone. The goal of the trial is to
determine whether SCENESSE® will reduce the total dose of radiation
(NB-UVB) and the time required to reactivate skin pigment producing
cells in vitiliginous lesions.
Following further discussions with regulators, Clinuvel took the
decision to run parallel protocols in Europe and the US (CUV101 and
CUV102 respectively). These protocols will provide results on the
safety and efficacy of the drug when given every 28 days alongside
six months’ NB-UVB phototherapy (CUV101) and when dosing
commences two months into a six month NB-UVB treatment regime
(CUV102). Each study site will recruit approximately 20 patients, with
50% of the patients treated with NB-UVB alone, and 50% treated in
combination with SCENESSE®.
Further amendments have been made to broaden the potential
patient population, a reflection of feedback from the patient and
medical communities asking for the inclusion of patients who have
undergone previous treatment for their vitiligo. These amendments
have lead to delays in the INSPIRE program, but are expected to
generate quality clinical data from which we can evaluate SCENESSE’s
potential as a vitiligo therapy.
The company announced in February that all patient visits for the
first Phase II study of SCENESSE® (afamelanotide) to be conducted
in the US had been completed. The study (CUV030) – a six month,
randomised, placebo-controlled trial – was designed to further
evaluate the safety and efficacy of SCENESSE® in reducing the
number and severity of phototoxic skin reactions in patients with the
rare light intolerance disorder erythropoietic protoporphyria (EPP).
The protocol of CUV030 closely followed that of a confirmatory
Phase III study being conducted in European sites (CUV029). All
patient visits for the CUV030 and CUV029 have completed and data
collection and management is under way.
In the last week, our first US site has commenced recruitment for
CUV102. Two further US sites are expected to commence recruitment
shortly. European protocol amendments are currently being evaluated
by the relevant regulatory authorities with the first patients expected
to commence across the three sites (France, Italyand Switzerland) in
June.
For more information on Clinuvel’s vitiligo program, log onto http://
www.clinuvel.com/vitiligo
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Clinuvel Communiqué May 2011
GUEST BLOGGERS AND NEW VIDEO RELEASES
Over recent months the team has invited several individuals to
share their experiences of erythropoietic protoporphyria (EPP),
polymorphous light eruption (PLE) and vitiligo on the company’s blog.
You can read all of these posts at http://www.clinuvel.com/en/blog/
guest/
To improve awareness of EPP and vitiligo online, the company has
released two new videos since April.
The first Burning in the Shadows is the latest in a series of videos
featuring Mikey, an Australian man with EPP. Mikey explains there is
no escape from light when outside for those with EPP and that even
in the shadows he has been in agony from reflected light alone.
The second video, Understanding vitiligo, explores established and
recent theories that explain what causes vitiligo, a disease which is
still not fully understood by the medical community.
To view these videos, log on to the Clinuvel website or our Youtube
channel at http://www.youtube.com/photoprotection.
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Clinuvel is listed on XETRA (UR9) and
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