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发表于 2019-7-16 20:22:43
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SCENESSE
WORLD’S FIRST SYSTEMIC PHOTOPROTECTIVE
Philippe Wolgen CEO
CLINUVEL PHARMACEUTICALS LTD FROM CONCEPT TO COMMERCIALIZATION
JEFFERIES HEALTHCARE CONFERENCE NEW YORK 6 JUNE 2019
ASX: CUV Nasdaq Int’l: CLVLY Xetra-Dax: UR9
SAFE HARBOUR STATEMENT 2019
This release contains forward-looking statements, which reflect the current beliefs and expectati** of CLINUVEL’s management. Statements may involve a number of known and unknown risks that could cause our future results, performance or achievements to differ significantly from those expressed or implied by such forward-looking statements. Important factors that could cause or contribute to such differences include: risks relating to: our ability to develop, obtain regulatory approvals in major markets and risk to commercialise pharmaceutical products, including our ability to develop, manufacture, market and sell one or more biopharmaceutical products while competition for our products may occur especially SCENESSE (afamelanotide 16mg); our ability to achieve expected safety and efficacy results through our innovative R&D efforts; the risk of proving effectiveness of the product for our patents and other protecti** for innovative products, particularly in view of national and regional variati** in patent laws; our potential exposure to product liability claims to the extent not covered by insurance(s); increased ** scrutiny in either Australia, the U.S., Europe and Japan of our agreements with third parties and suppliers; our exposure to currency fluctuati** and restricti** as well as credit risks; the effects of reforms in healthcare regulation and pharmaceutical pricing and reimbursement; that the Company may incur unexpected delays in the outsourced manufacturing of SCENESSE which may lead to it being unable to supply its commercial markets and/or clinical trial programs; any failures to comply with any ** payment system (i.e. Medicare) reporting and payment obligati**; uncertainties surrounding the legislative and regulatory pathways for the registration and approval of biotechnology based products; decisi** by regulatory authorities regarding approval of our products as well as their decisi** regarding label claims; any failure to retain or attract key personnel and managerial talent; the impact of broader change within the pharmaceutical industry and related industries; potential changes to tax liabilities or legislation; environmental risks; and other factors that have been discussed in our 2018 Annual Report. Forward-looking statements speak only as of the date on which they are made and the Company undertakes no obligation, outside of those required under applicable laws or relevant listing rules of the Australian Securities Exchange, to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise. More information on the forecasts and estimates is available on request. Past performance of the company is not an indicator of future performance.
SCENESSE (afamelanotide 16mg)
new molecular entity (NME) = synthetic 13 aminoacid peptide (API) novel controlled-release subcutaneous injectable implant (polymer) dose frequency 60 days
first-in-class therapy in erythropoietic protoporphyria (EPP)
world’s first systemic photo protective
Alpha-MSH:
1. anti-oxidative
2. melanogenic (activates epidermal melanin production) 3. anti-inflammatory
4. DNA reparative, photo protective
CLINUVEL – translational use melanocortins
EPP
[COMPLETED]
Proof of concept - porphyrias (EPP, CEP) 1991- 2019
pandermal protection, visible light (>408nm)
Photo protection, DNA repair
Systemic repigmentation (total body)
1st pharma addressing loss of pigmentation
positive early safety/efficacy data
Systemic & topical photoprotection
2nd generation melanocortins
additional indicati**
complementary product lines – OTC
S A F E T Y
Additional indication
Vitiligo
[Ph II]
Metabolic Diseases Photo protection
CLINUVEL REGISTER
IPO 2001 Australian Securities Exchange
48,960,633 ordinary shares
Geographic distribution
ASX-300: VWDA
CUV [07 SEP 2018] 204,808 [0.42% of OSC]
35.00 30.00 25.00 20.00 15.00 10.00
5.00 0.00
JAN 2016 – JUN 2019
VWDA : 39,238 204,808
0.09% 0.42%
Australia Europe
North America Asia Pacfic
R&D 2005-2016
- cost-management
- equity financing
- never below critical cash
- financial proof of principle 2010
COMMERCIAL 2016
- cost-management - cash positivity
- profitability
- debt-free
- first dividend FY18
FINANCIALS
$30.00
$20.00
$10.00
$-
-$10.00
-$20.00
CAGR 29%
79%60 87% 40
58% 20 0
-20
-40
FY05 FY06 FY07 FY08 FY09 FY10 FY11 FY12 FY13 FY14 FY15 FY16 FY17 FY18
Total expenses Total Revenue EPS (A$ cents) Net cash provided by/(used in) operating activities
*CUV continues quarterly reporting
**Cash flow statement will reflect seasonal fluctuati** due to cyclical treatment period
A$M
EPS A$ cents
Product Pipeline
Programme – SCENESSE (afamelanotide 16mg)
SCENESSE in adult EPP patients (Europe)
SCENESSE in adult EPP patients (USA) SCENESSE in adult EPP patients (Australia, Japan)
SCENESSE in adult vitiligo patients (global)
SCENESSE in adult variegate porphyria patients (Europe)
Programme – next generation products
SCENESSE ENFANCE (paediatric formulation) CUV9900
VLRX001
OTC product line
Phase I
Phase III
Pre- clinical
Phase II
Approved
Erythropoietic protoporphyria (EPP)
Phototoxic reaction in an EPP patient. FECH deficiency 18q21, part of the heme metabolic pathway Image courtesy of the KE family.
Ill-characterized, poorly understood disease
Absolute intolerance to blue/green/UVA/UVB light
(i) Phototoxicity – incapacitating anaphylactoid reacti**,
burns
(ii) High unmet medical need
Isolated patients living reclusive existence
SCENESSE IN ERYTHROPOIETIC PROTOPORHYRIA (EPP)
EMA – MA 2014: standard of care for EPP in European Union, Switzerland
rare genetic metabolic disorder, 1:140,000
17 countries with known EPP patients
Commercial roll out, focused specialized team
disciplines across clinical, regulatory, pharmacovigilance, QA, communicati**, market access cost effective business model is being replicated in USA
University medical centers exclusively (reference centers)
Equitable treatment of payors
Uniform pricing strategy globally
Health economic impact: US$96,000 per patient [calendar year]
SCENESSE IN VITILIGO
Clinical Objectives
1. repigmentation face/head/neck 2. follicular resp**e
3. safety profile
Ph II CUV102 n=54
- extent of repigmentation
- time to repigmentation - face
- JAMA Dermatology 2013, 2015
- maintenance of pigmentation at day 336
Ph II CUV103 n=18 [2018]
- repigmentation
total body, head, neck at day 196 VASI (p<0.001)
- maintenance of pigmentation at day 280
VASI (p<0.025) (p=0.01)
[2013]
SCENESSE IN VITILIGO
Day 0 Baseline
Day 55
After 15 NB-UVB treatments, 1 implant
Day 111
After 27 NB-UVB treatments, 3 implants
Day 176
After 40 NB-UVB treatments, 4 implants
SUMMARY – CLINUVEL PHARMACEUTICALS
SCENESSE innovative medical concept – world’s first systemic photoprotective drug (Rx)
Commercialized orphan medicinal product (NME) in Europe
Erythropoietic protoporphyria (EPP) – rare genetic metabolic disorder
Generating Real World Evidence under “PASS”
94% treatment continuation in EU/CH (as per 1 April 2019)
SCENESSE - NDA for prevention EPP
Profitability FY2017, FY2018:
Growth
PDUFA date 6 October 2019
maiden dividend 2018
1. translational use melanocortins 2. M&A, in-licensing
“Afamelanotide has radically changed the way I approach my daily life... This medicine has freed me from the debilitating c**equences of EPP and from fear of suffering them” Swiss EPP patient
CLINUVEL PHARMACEUTICALS LTD
JEFFERIES HEALTHCARE CONFERENCE NEW YORK 6 JUNE 2019
GRATITUDE TO CLINUVEL GLOBAL TEAMS FOR 14 YEARS OF TENACITY
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发表于 2019-6-10 10:42:11
