Clinuvel Pharmaceuticals Limited · ASX:CUV
Clinuvel Pharmaceuticals — Investor Presentation (July 2011)
26 slides from Clinuvel Pharmaceuticals's investor presentation, Jul 26, 2011
Slide 1: Clinuvel's objective: SCENESSE as a safe therapeutic solution
For personal use only Clinuvel’s objective is to develop the novel drug SCENESSE® as a safe therapeutic solution for select patients who require photoprotection. 1
Slide 2: Forward-looking statements
For personal use only This mandatory statement aims to protect from any forward-looking statements that may be inferred from the presentation and that may differ from the actual development by Clinuvel. 2
Slide 3: Innovation is at the heart of Clinuvel
For personal use only Innovation is at the heart of Clinuvel and is fourfold: We are working with a novel molecule (NCE; afamelanotide), not used before in the clinic and a novel formulation specifically developed to match the desired pharmacological response. Our team identified the most appropriat
Slide 4: Scientific background of SCENESSE technology
For personal use only The scientific project underpinning the SCENESSE® (afamelanotide) technology started through NIH funding to find a melanoma prevention strategy. Although the company is currently not targeting melanoma, ongoing research in the inhibitive properties of SCENESSE® on human melanom
Slide 5: SCENESSE 16mg afamelanotide implant
For personal use only SCENESSE® 16mg afamelanotide implant is Clinuvel’s lead product, administered in a unique (no other current commercially available) formulation. The formulation has been developed to ensure controlled-release of the drug for a defined period of time. Administration and dose reg
Slide 6: Inadequate UV response in Caucasian skin
For personal use only Under physiological conditions, individuals with phototype I and II (Caucasian skin) prove deficient in an adequate UV-response, meaning those who expose themselves long enough are prone to burn the skin easily. Skin burns at young age increases the risk of skin cancers and mel
Slide 7: Cellular response to UV exposure and alpha-MSH
For personal use only After UV (and light) exposure, the incipient DNA damage of melanocyte and keratinocyte sets in motion a cascade of cellular events in order to protect the skin: Second, the keratinocyte starts to release alpha-MSH at high velocity to the neigbouring melanocyte. Third, the alpha
Slide 8: SCENESSE addresses both clinical issues
For personal use only Clinuvel’s SCENESSE® has proven to address both clinical issues in an adequate manner. Those Caucasian patients diagnosed with EPP, PLE and SU with an inadequate eumelanin-pheomelanin ratio and partial loss of receptor-function respond well to the drug and produce eumelanin, of
Slide 9: Long-term toxicology studies
For personal use only Two sets of long-term toxicology studies were conducted. The change of pigmentation of skin (and in animals of fur colour) were consistently observed, with no system organ involvement. 9
Slide 10: Development history and patient experience
For personal use only The first decade, a search was undertaken to find an appropriate dose and formulation of a novel melanocortin. In 2006, new management accelerated development and mapped out a clinical strategy, whereby a new formulation was chosen. To date the drug has been used in over 600 pa
Slide 11: EPP: orphan drug designation and filing plans
For personal use only EPP is a severe, rare metabolic disorder which causes absolute intolerance to light, particularly visible or ‘blue’ light. There are no effective therapies for EPP, which is a lifelong disorder. Light intolerance forces patients to avoid sunlight and has a significant impact up
Slide 12: EPP trials CUV030 and CUV029
For personal use only The protocols CUV030 and CUV029 are near-identical. The overall objective is to demonstrate clinical relevance of the drug under conditions of use. The challenge is to show that patients have been able to change their (since childhood) adapted behaviour. In essence, we are en r
Slide 13: Vitiligo and the INSPIRE program
For personal use only Vitiligo is a pigmentary disorder causing the loss of melanocyte function in skin. This disease has a severe impact upon patient quality of life (Tasmania, Lamb & Bewley, 2010 & Linthorst Homan et al, 2009) and no effective treatments have been identified to date. No drugs have
Slide 14: Vitiligo studies CUV101 and CUV102
For personal use only CUV101 and CUV102 are a multi-centre six-month open label pilot study to evaluate the efficacy of SCENESSE® as a combination therapy with narrowband ultraviolet B (NB-UVB) phototherapy compared to NB-UVB alone. NB-UVB clinically administered thrice per week over 18 months is co
Slide 15: Selecting indications for SCENESSE
For personal use only CUV team analysed the various disorders which were most likely to respond clinically to SCENESSE® Six indications were identified, of which five were selected. Some studies served as proof of concept, while others aimed at proving efficacy and safety. Upon analysing the various
Slide 16: EPP and vitiligo selected for advanced development
For personal use only The five selection criteria resulted in the identification of EPP and vitiligo as disorders warranting advanced clinical and regulatory development of SCENESSE®. For EPP, orphan status was obtained in 2008, and expert centres were identified in EU- AUS-US. The drug would be use
Slide 17: Regulatory strategy
For personal use only The regulatory strategy is aimed at understanding concerns, and by engaging in an active dialogue progressing towards acceptability of scientific concept and clinical benefit. We follow a structured approach. Abbreviations: NME/NCE new molecular entity/new chemical entity COMP
Slide 18: Assessing the regulatory challenge
For personal use only Before embarking on the regulatory path, and in order to understand whether Clinuvel is able to match shareholders’ expectations, our team assessed the challenge on the way up. *Image posted to flickr.com by anaulin. Used under creative commons license. 18
Slide 19: Orphan drug and NME approval rates
For personal use only Looking at orphan drug applications and NMEs, a high percentage of those dossiers which reached application status were actually approved in the past four years. Similar numbers are seen in the US. 19
Slide 20: Clinuvel's business model
For personal use only Clinuvel’s business model is unique in drug development; a necessity of the product with which we work. Over five years the company has developed strong relationships with the expert communities caring for our patients and a number of peer-reviewed papers and conference present
Slide 21: Advantages of the focused model
For personal use only By narrowing in on specific elements of the business, one must weigh the advantages and disadvantages to ensure the end goal can be achieved in the best possible manner. Here, our clinical and regulatory goals are clear and the long term benefits from the model are already bein
Slide 22: Financial overview
For personal use only An overview of the current financials is given. 22
Slide 23: Strategy based on two clinical indications
For personal use only Clinuvel has a clear strategy based around two clinical indications for which there are no effective medical therapies to date. Additional clinical indications (OTR patients and PLE) will be evaluated based on pending clinical results. Numbers are estimated on modest penetratio
Slide 24: First commercial distribution in Italy
For personal use only Clinuvel’s decision to develop treatment for underserved patient populations is correct, the Italian experience underwrites this. The first commercial distribution of SCENESSE® in Italy is a success as gauged by repetitive use and sales of the first year. 24
Slide 25: Upcoming filing and trial results
For personal use only Most significant is the company’s first filing for registration in Q4 of this year, pending positive outcome of the trials CUV030 and CUV029. The Italian scheme has served as a proof of principle for the distribution of the drug, we monitor long-term the safety of the drug as w
Slide 26: Clinuvel Pharmaceuticals
Clinuvel Pharmaceuticals systemic photoprotection of the skin for patient populations unable to expose to UV and light Appreciation to NOMURA team 26 July 2011 Sydney Ph Wolgen, CEO H Agersborg, CSO ASX: CUV XETRA-DAX: UR9 ADR CLVLY 26
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