Santhera Pharmaceuticals Holding AG · SIX:SANN
Santhera Pharmaceuticals — Investor Presentation (July 2007)
32 slides from Santhera Pharmaceuticals's investor presentation, July 2007
Slide 1: Dedicated to small-molecule therapeutics for neuromuscular diseases
Dedicated to small-molecule therapeutics for neuromuscular diseases Company presentation July 2007
Slide 2: Disclaimer
Disclaimer This presentation is not and under no circumstances is to be construed as a solicitation, offer, or recommendation, to buy or sell securities issued by Santhera. Santhera makes no representation (either express or implied) that the information and opinions expressed in this presentation a
Slide 3: Content
Content 1. Santhera at a glance 2. Business overview 3. Product pipeline 4. Key results 2006 5. News flow & concluding remarks Company presentation, July 2007 slide 3
Slide 4: Santhera at a glance
Santhera at a glance Focus on small-molecule therapies for orphan neuromuscular diseases Lead compound SNT-MC17 with well-established safety profile in filing for EU marketing approval and clinical development in three indications: Friedreich’s Ataxia (FRDA), Duchenne Muscular Dystophy (DMD) and Leb
Slide 5: Key achievements 2007 to date
Key achievements 2007 to date Accelerated MAA filing of SNT-MC17 in FRDA in Europe Amendment of clinical protocol of ongoing Phase III trial in FRDA in Europe Revised and shortened development program for SNT-MC17 in FRDA in US, protocol for Phase III trial currently in SPA process under open IND Or
Slide 6: Content
Content 1. Santhera at a glance 2. Business overview – Medical need – Market opportunity 3. Product pipeline 4. Key results 2006 5. News flow Company presentation, July 2007 slide 6
Slide 7: Over 200 NMDs and movement disorders
Over 200 NMDs and movement disorders Dyskinesia in Parkinson’s Disease (DPD) Movement disorders Huntington’s Disease Spinocerebellar ataxias Friedreich’s Ataxia (FRDA) Sensory-motor deficits Spinal cord injury Charcot-Marie-Tooth neuropathies Amyotrophic lateral sclerosis Motorneuron diseases Guilla
Slide 8: Focus on neuromuscular diseases offers interesting business opportunity
Focus on neuromuscular diseases offers interesting business opportunity High unmet Chronic diseases often leading to life threatening conditions medical need Few if any approved therapies + Genetic Well-understood disease mechanisms disorders New treatment opportunities offered by scientific advance
Slide 9: Focused business strategy
Focused business strategy Research & Grow in key area of expertise through own research as Development well as in-licensing and co-operations Leverage compounds into several indications Out-license non-core development programs Marketing Launch products with own specialty marketing and sales & Sales
Slide 10: Current portfolio
Current portfolio Preclinical Phase I Phase II Phase III Filing SNT-MC17 in Europe Friedreich’s Ataxia SNT-MC17 pivotal study Friedreich’s Ataxia in US SNT-MC17 proof-of-concept Duchenne Muscular Dystrophy study in Europe SNT-MC17 proof-of-concept Leber’s Hereditary Optic Neuropathy study in Europe
Slide 11: Significant market potential of current clinical portfolio
Significant market potential of current clinical portfolio Friedreich’s Ataxia Duchenne Muscular Dystrophy ~ 20,000 patients in Europe and ~ 30,000 patients worldwide North America in total Affects men, diagnosed around Affects primarily Caucasians 3 to 5 years of age Life-long treatment required Li
Slide 12: Content
Content 1. Santhera at a glance 2. Business overview 3. Product pipeline – SNT-MC17 (INN: idebenone) – JP-1730 (INN: fipamezole) – SNT-317 (INN: omigapil) – Key partnerships 4. Key results 2006 5. News flow Company presentation, July 2007 slide 12
Slide 13: Friedreich's Ataxia (FRDA)
Friedreich’s Ataxia (FRDA) Severe genetic disorder: – Degeneration of nerve and muscle tissue – Loss of muscle control – Impaired movements – Muscle wasting – Thickening of heart walls (cardiomyopathy) Caused by a reduced level of frataxin, a protein needed in mitochondria to facilitate energy produ
Slide 14: NIH/Santhera trial in FRDA with SNT-MC17
NIH/Santhera trial in FRDA with SNT-MC17 Study objectives: Establish safety of mid and high dose SNT-MC17 in young FRDA patients over 6 month treatment period Primary endpoint is level of 8OH2’dG – a marker for oxidative stress Secondary endpoints to pilot efficacy on - neurological scores: ICARS, F
Slide 15: NIH/Santhera: International Cooperative Ataxia Rating Scale (ICARS)
NIH/Santhera: International Cooperative Ataxia Rating Scale (ICARS) Patients with baseline ICARS >10 and <54 ANOVA model with effects for treatment and allele category p=0.002 for hypothesis that there is a dose-dependent effect (Jonckheere-test) Mean % changes are raw means 10 ICARS placebo low mid
Slide 16: NIH/Santhera: Activities of Daily Living (ADL)
NIH/Santhera: Activities of Daily Living (ADL) Patients with baseline ICARS >10 and <54 ANOVA model with effects for treatment and allele category Mean % changes are raw means 10 ADL placebo low mid high 5 baseline1 11.74 12.52 13.00 11.27 % change placebo low change1 0.81 0.02 - 1.25 - 1.45 0 P-val
Slide 17: SNT-MC17 in FRDA: accelerated clinical and regulatory development
SNT-MC17 (INN: idebenone) in FRDA: accelerated clinical and regulatory development Well-established safety profile Consistent reporting of reduced cardiac hypertrophy in several published academic studies Medical community widely views SNT-MC17 as efficacious in FRDA Latest clinical data (NIH/Santhe
Slide 18: Compassionate use and temporary registration of idebenone in Europe
Compassionate use and temporary registration of idebenone in Europe ~1:250,00 -1:750,000 In Switzerland: Provisional registration for ~1:100,000 ~1:50,000 ~ 1:30,000 treatment of cardiomyopathy in FRDA patients In France: Compassionate use in FRDA In Italy: Compassionate use in FRDA Other European c
Slide 19: Leveraging SNT-MC17: second and third indication
Leveraging SNT-MC17: second and third indication Duchenne Muscular Dystrophy Leber’s Hereditary Optic Neuropathy Most common form of muscular dystrophy Genetic disease caused by a degeneration of Average onset between 3 and 5 years; average nerve cells in retina leading to rapid loss of life expecta
Slide 20: JP-1730 (fipamezole) in Dyskinesia in Parkinson's Disease
JP-1730 (INN: fipamezole) in Dyskinesia in Parkinson’s Disease (DPD) Chronic use of dopaminergic drugs, the standard PD treatment, causes motor dys- functions such as dyskinesia (chaotic movements) that increase in frequency and severity over time Dyskinesia characterized by periodically occurring c
Slide 21: JP-1730: clinical and regulatory status
JP-1730: clinical and regulatory status Clinical phase I/II studies indicate compound Antidyskinesia responses to be safe and well tolerated 0.5 (n=10) Change in dyskinesia p>0.05 -0.5 (n=10) Strong proof of concept in collaborative NIH score -1.5 p=0.05 Phase IIa demonstrated -2.5 (n=10) p=0.02 (n=
Slide 22: SNT-317 (omigapil) in Congenital Muscular Dystrophy (CMD)
SNT-317 (INN: omigapil) in Congenital Muscular Dystrophy (CMD) Severe muscle weakness at birth (“floppy infant syndrome”) or in early childhood lead to reduced life expectancy Several genetically and clinically different sub-forms (MDC1A, Ullrich CMD and Bethlem myopathy being the most common in the
Slide 23: SNT-317: development and regulatory status
SNT-317: development and regulatory status Percentage of apoptotic myofibers 1.4 Triceps muscle ∗ 1.2 Omigapil binds to GAPDH and acts as inhibitor of 1.0 apoptosis 0.8 In dyW mouse animal model for MDC1A, omigapil ... 0.6 – reduces early mortality and body weight loss 0.4 0.2 – improves muscle hist
Slide 24: Collaborations and partnerships (1)
Collaborations and partnerships (1) Novartis In-licensing of omigapil from Novartis for clinical development in CMD with option to expand to other NMDs Upfront payment and milestone payments upon start of pivotal trial and marketing approval plus royalties on product sales Santhera has full access t
Slide 25: Collaborations and partnerships (2)
Collaborations and partnerships (2) Juvantia Co-operation agreement to conduct Phase IIb trial with JP-1730 in DPD to confirm efficacy and commercial potential of compound Santhera has option to acquire Juvantia by end of 2008 (with potential extension of option term until mid-2009) in exchange for
Slide 26: Content
Content 1. Santhera at a glance 2. Business overview 3. Product pipeline 4. Key results 2006 – Financials – Share information 5. News flow Company presentation, July 2007 slide 26
Slide 27: Strong cash position and tightly managed burn rate
Strong cash position and tightly managed burn rate Financing since 2004: – CHF 86.7 million raised in three private financing rounds – CHF 101.8 million in IPO (greenshoe exercised) Cash and cash equivalents as of December 31, 2006: CHF 125.7 million Gross cash burn from operating and investing acti
Slide 28: Key financial information 2006
Key financial information 2006 Income statement Balance sheet (IFRS, consolidated, in CHF thousands) (IFRS, consolidated, in CHF thousands) Dec 31, Dec 31, 2006 2005 2006 2005 Gross profit 781 13,756 Cash and cash equivalents 125,662 31,268 R&D expenses –17,985 –14,542 Noncurrent assets 34,260 32,99
Slide 29: Shareholder structure
Shareholder structure 89.8% of shares registered; over 720 investors* 96.1% of shares held by institutional investors Pre-IPO investors (61.7%) locked-up until May 2007 (hard lock-up) and November 2007 (soft lock-up) respectively Free float: 38.3% Shareholders by Domicile Largest shareholders Others
Slide 30: Content
Content 1. Santhera at a glance 2. Overview of neuromuscular diseases 3. Product pipeline 4. Key results 2006 5. News flow Company presentation, July 2007 slide 30
Slide 31: Expected milestones and news flow
Expected milestones and news flow Final protocol under SPA for US phase III trial with SNT- H2 2007 MC17 in FRDA MAA filing of SNT-MC17 for FRDA in Europe Start of US Phase III trial with SNT-MC17 in FRDA Results of Phase IIa trial with SNT-MC17 in DMD Start of Phase IIb trial with JP-1730 in DPD Bi
Slide 32: Contacts
Contacts Santhera Pharmaceuticals Holding AG Hammerstrasse 47 CH-4410 Liestal Switzerland www.santhera.com Barbara Heller, CFO Phone +41 (0)61 906 89 54 [email protected] Thomas Staffelbach, VP P&IR Phone +41 (0)61 906 89 47 [email protected] Company presentation, July 2007 s
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