Clinuvel Pharmaceuticals Limited · ASX:CUV
Clinuvel Pharmaceuticals — Investor Presentation (February 2016)
25 slides from Clinuvel Pharmaceuticals's investor presentation, Feb 8, 2016
Slide 1: Presentation to international porphyria patient group
Presentation to international porphyria patient group Melbourne, Australia and Leatherhead, UK, February 8, 2016 Clinuvel Pharmaceuticals Ltd (ASX: CUV; XETRA‐DAX: UR9; ADR: CLVLY) presented to an international porphyria patient meeting, held in Rotterdam, the Netherlands, on February 6, 2016. The p
Slide 2: Clinuvel will not discuss the product profile during this meeting
In line with European Guidelines on Medical Advertising and UK Advertising Standards for Medicinal Products, Clinuvel will not discuss the profile of the product SCENESSE® (afamelanotide 16mg) during this meeting. The product is approved to prevent phototoxicity in adult erythropoietic protoporphyri
Slide 3: Forward looking statements are not aimed at soliciting interest in the company
The possible forward looking statements in this discussion are not aimed at inviting or soliciting interest in the company. 2
Slide 4: Discussion points relevant to physicians, experts and EPP patients
Today’s discussion points are relevant to physicians, experts in the treatment of the disease, and EPP patients and their families. 3
Slide 5: First discussion point: characterisation of the disorder
The first discussion point is aimed at characterisation of the disorder. 4
Slide 6: Stigmas patients have reported over the years
The stigmas patients have reported to our teams over the years are found in an abundance of letters, emails and from phone conversations (left). The labelling of the disease by bystanders, ‘outsiders’, differs from the description patients seem to give themselves (right). 5
Slide 7: Key questions in developing a drug for EPP in 2005
In 2005 there were a number of key questions in developing a drug for EPP, however the hypothesis of SCENESSE® was answered after the first study in 2006. The responses from patients and physicians has been stronger than expected. 6
Slide 8: Understanding the suffering of a patient
In general one can not fully understand the suffering of a patient until one experiences the ordeal, symptoms and disease her/himself. This will always be the chasm between drug companies and patients. A discussion on human’s discriminatory senses and especially EPP‐patients’ ability to discern “vis
Slide 9: Eight characteristics of EPP
Over the years eight characteristics of EPP have been identified by our teams and experts, and all of them make the disease complex to evaluate. In addition, as there are no appropriate scientific tools to measure the characteristic symptoms, the Clinuvel program led to optimum synthesis of a molecu
Slide 10: Diagram developed for regulatory discussions on EPP
As part of the regulatory discussions and filings, this diagram was developed and is discussed today. EPP patients are self‐limiting their ability to expose due to the characteristics of the disorder. A number of behavioural components make EPP different from other light induced disorders, rare dise
Slide 11: The next topic is the paediatric development
The next topic is the paediatric development. 10
Slide 12: Estimated paediatric development time and cost
The estimated time of development is approximately 36 months, costing millions of EUROs including product development, clinical trials, regulatory filings, follow up of patients. The regulatory views will largely determine further timelines. The successful completion of the development of the paedia
Slide 13: The third topic is risk management
The third topic is risk management. 12
Slide 14: Protocolised treatment of EPP in Europe and pharmacovigilance
The main eight points are part of the protocolised treatment of EPP in Europe, also expected in other parts of the world. The overall commitment for any developer of novel drugs is to emphasise PHARMACOVIGILANCE, the ability to be extra vigilant for its patients long term; Clinuvel undertakes this.
Slide 15: Initial roll-out focus on countries with known patient populations
Clinuvel’s initial roll‐out focus is on those countries with known patient populations. The company’s distribution centre is Leatherhead, UK. 14
Slide 16: Country-by-country assessment of the product within the EU
Within the EU each country is assessing the product on a number of criteria, including: benefit, cost to society, number of patients expected and risk of off‐label use. 15
Slide 17: European EPP Disease Registry (EEDR)
Under European marketing authorisation, Clinuvel is establishing and maintaining an EPP‐specific Disease Registry (EEDR). The ownership of the medical data will remain with the individual patients and reside in each medical centre, however the pseudonymised medical data related to EPP will be made a
Slide 18: EMA timelines for reporting data from the European program
Set timelines have been established by EMA for Clinuvel to report data from its European program. The timelines given here provide a sample 12 month reporting requirement. 17
Slide 19: Pharmacovigilance data flow
The data flow established for pharmacovigilance is complex. At each point of these processes, there are controls in place and all these controls form part of a pharmacovigilance management system. Each ‘station’ on the chart requires independent auditing of processes, proper management and adequate
Slide 20: A summary is provided
SCENESSE® (afamelanotide 16mg) will be available to all known EU countries first EPP Expert Centres only Product = High Specialty Technology Controlled and planned distribution Ongoing explanation of EPP required _g Clintvel Asummary is provided. 19
Slide 21: Seeing and observing are distinct
SEEING AND OBSERVING ARE DISTINCT IT REQUIRES DECADES TO FULLY ‘UNDERSTAND’ EPP P Cintvel 20
Slide 22: EPP patients are continuously weighing up exposure versus isolation
EPP PATIENTS ARE CONTINUOUSLY WEIGHING UP EXPOSURE VERSUS ISOLATION RISK VERSUS CONSEQUENCES SHADE VERSUS LIGHT P Cintvel 21
Slide 23: Clinuvel hopes to provide light for EPP patients
In the renaissance artists were trying to capture light in shadows on canvas. Clinuvel hopes to provide light for EPP patients who have led a life on the fringe and in darkness. 22
Slide 24: More than a decade devoted to one drug in one disorder
Clinuvel has devoted more than a decade to one drug in one disorder, the response of patients and physicians has made this journey worthwhile. 23
Slide 25: Thank you for the invitation
Thank you for the invitation. 24
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