Clinuvel Pharmaceuticals Limited · ASX:CUV
Clinuvel Pharmaceuticals — Investor Presentation (March 2010)
18 slides from Clinuvel Pharmaceuticals's investor presentation, Mar 15, 2010
Slide 1: Medicinal Photoprotection (Rx) - Afamelanotide 1st-in-class
Medicinal Photoprotection (Rx) For personal use only Afamelanotide 1st-in-class, the end of clinical development Clinuvel Pharmaceuticals Ltd Hong Kong, Singapore ASX: CUV Monday 15th –Thursday 18th 2010 XETRA-DAX: UR9 Dr Philippe Wolgen, CEO ADR: CLVLY
Slide 2: Safe harbor
Safe harbor Clinuvel is an Australian biopharmaceutical company focused on developing For personal use only its leading drug candidate, afamelanotide, for a range of UV- related skin disorders resulting from exposure of the skin to harmful UV radiation. Pharmaceutical research and development involv
Slide 3: Financials
Financials • MCAP A$76.4M For personal use only • Reserves (March 1) A$30.8M • Current Ratio 12.6 • Burn rate (monthly) A$1.1M • Shares on issue 303,188,665 • Average trading volumes (ASX:Q1) 378,000 • Registry top 20 hold 40% free float 20%
Slide 4: R&D expenditures [IPO'01-'10]
R&D expenditures [IPO’01-’10] For personal use only • Drug formulation development 21.56M • Clinical & regulatory 24.49M • Operational 26.56M • Total expenditures¹ A$72.6M ¹ on accrual basis acc GAAP
Slide 5: Projections months
Projections 18 months¹ For personal use only • Clinical 21.0M • Operational 6.2M • Marketing, legal 3.0M • Total expenditures A$30.2M ¹ Forecast Q1 2010
Slide 6: Clinuvel's business strategy
Clinuvel’s business strategy For personal use only • EMA regulatory filing for MAA EPP 2010 FDA, TGA EPP 2011 • Preparation EU commercial distribution afamelanotide¹: 1. market in orphan diseases (ODD) 2. EU pricing & reimbursement assessment completed 3. access programs enabling early use 4. 3-4% E
Slide 7: Development milestones
Development milestones 2010 Q1 Q2 Q3 Q4 ‘011 For personal use only US confirmatory PK study results CUV028 FDA approval start EPP (US) CUV030 [6 months] EPP Ph III confirmatory trial start (EU) CUV029 [6 months] PLE Ph III trial starts (EU) CUV032 [4 months] EPP Ph III final results (EU/AUS) CUV017
Slide 8: Polylactide resorbable implant formulation
Polylactide resorbable implant formulation For personal use only • biocompatible polymer √ - biodegradable/safety known • unique dissolution rate √ • stability √ - intended shelf-life >2 yrs • ease s.c. administration √
Slide 9: Strong IP
Strong IP • Proprietary drug product, IP For personal use only – composition of matter [2014] – plasma concentration/bioavailability – pharmacogenomic – methods of use [photodermatoses/skin cancer/dermoprotection] • Continuous filings PCT/EPO • Orphan drug designation EU and US: 10–7 yrs exclusivity
Slide 10: Pharmacological basis
Pharmacological basis <selective & specific melanocortin agonist to modulate UV response of the skin> For personal use only • Melanocortin-1 receptor (MC1R) deficiency → 7 allelic variations > 75% Caucasians • Endogenous paracrine α-MSH ineffective (t½ = short) → insufficient dermal melanocytic cAMP
Slide 11: Afamelanotide
Afamelanotide optimized linear α-MSH For personal use only • MC1R–specific in skin, New Molecular Entity • Kd/Ki = MC1R binding • t½ = 6 * longer, rapid peptide degradation • Controlled-release formulation • 1st in-line Rx in porphyrias (EPP/CEP) - prophylactic treatment λ/UV • Preclinical safety pr
Slide 12: Afamelanotide in porphyria
Afamelanotide in porphyria < metabolic disorder of heme synthesis, Ch 18q21.3 low expression FECH allele, 1:175,000> For personal use only • Tetrapyrrole ring in porphyria is excitable @ λ> 408nm • Porphyrin accumulation in skin • Acute phototoxicity due to ROS to endothelium • Lack effective therap
Slide 13: Afamelanotide clinical data
Afamelanotide clinical data For personal use only • Afamelanotide tested in 106 EPP patients (Phase II+ III), > 350 doses over more than 3 years • Preliminary results EPP Phase III CUV017 [n = 101] I reduction in severity p=0.03 [CI 95%] II active-placebo p=0.006 [CI 95%] • Clinical significance: le
Slide 14: Afamelanotide clinical development
Afamelanotide clinical development Patients to date EPP efficacy Additional safety trials For personal use only Liquid 1,700 doses in 137 patients Pharmacodynamics I p = 0.008 Phase II PLE I Implant 1,069 doses in 371 patients 81 patients MD: p < 0.01 18 patients MD: p < 0.05 Currently 113 patients
Slide 15: Translational use afamelanotide
Translational use afamelanotide For personal use only
Slide 16: FDA New Drug Approvals
FDA New Drug Approvals For personal use only ’06 ’07 ’08 ‘09 Analgesics 2 2 3 6 Cardiovascular 1 2 2 6 CNS 3 5 1 2006: 27 Dermatology 3 2 2 21 NDAs 2007: 47 GI 1 6 2 18 NDAs Haematology 2 3 4 Metabolics 8 24 7 10 Misc 1 1 2 1 Oncology 7 7 5 4 Ophthalmology 1 2 4 3 2008: 42 21 NDAs 2009: 54 26 NDAs R
Slide 17: Key facts afamelanotide
Key facts afamelanotide world’s first preventative drug to photo protect skin (Rx) For personal use only • Basis for development afamelanotide 16mg - in EPP high clinical need - lack of effective therapy in small population (ODD) • Longitudinal safety in > 2,700 doses is excellent • 100% adoption by
Slide 18: Medicinal photoprotection (Rx)
Medicinal photoprotection (Rx) prophylactic s.c. administration per 60 days For personal use only Clinical severity is pivotal factor in CUV’s program I cutaneous symptoms in EPP & other photodermatoses II (pre)malignant actinic lesions in organ transplant recipients Day 1 Day 30
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