Santhera Pharmaceuticals Holding AG · SIX:SANN
Santhera Pharmaceuticals — Shareholder Meeting (May 2015)
28 slides from Santhera Pharmaceuticals's shareholder meeting presentation, May 11, 2015
Slide 1: Progress in all programs
Fortschritte in allen Programmen Progress in all programs Thomas Meier, CEO Ordentliche Generalversammlung Basel, 11. Mai 2015
Slide 2: Pipeline with Raxone/Catena in three indications with high unmet medical need
2 Pipeline with Raxone®/Catena® in three indications with high unmet medical need Leber’s Hereditary Optic Neuropathy (LHON): MAA under review in EU Duchenne Muscular Dystrophy (DMD): Positive Outcome of Phase 3 study Primary progressive MS (ppMS): Phase 2 study in collaboration with NIH Raxone®: tr
Slide 3: Medical need for effective treatment of respiratory illness in DMD
3 Medical need for effective treatment of respiratory illness in DMD Annual Shareholder Meeting 2015
Slide 4: Measures of pulmonary function loss in DMD
4 Measures of pulmonary function loss in DMD DMD patient healthy individual Figures (B) and (C) courtesy of Dr. Oscar H. Mayer, Division of Pulmonology, The Children's Hospital of Philadelphia, USA. Annual Shareholder Meeting 2015
Slide 5: Use of glucocorticoid steroids and assisted ventilation increase life expectancy in DMD
5 Use of glucocorticoid steroids and assisted ventilation increase life expectancy in DMD taken from Goemans et al. (2014) European Neurological Review, 9(1):78–82 Annual Shareholder Meeting 2015
Slide 6: Urgent medical need for patients unable to take glucocorticoid steroids
6 …. but there is an urgent medical need for patients unable to take glucocorticoid steroids § With increasing age, fewer patients tolerate glucocorticoid steroids (side effects) § Loss of respiratory function enters critical stage in early teenage years § There is currently no treatment available f
Slide 7: Santhera's development program with Raxone in DMD - Overview
7 Santhera’s development program with Raxone® in DMD - Overview Phase II, RCT Phase II, open label DELPHI Trial DELPHI-E Trial (N=21) (N=19) 2005 2006 2007 2008 2009 2010 2011 2012 2013 2014 Phase III, RCT DELOS Trial (N=64) Objective: To assess the efficacy of Raxone® compared to placebo in delayin
Slide 8: Publication in The Lancet
8 Additional clinical observations: Publication in The Lancet Kaplan-Meier Analysis: Upper Respiratory Tract Infection Annual Shareholder Meeting 2015
Slide 9: Phase III trial, DELOS – patients and treatment
9 Phase III trial, DELOS – patients and treatment Patients: Randomized treatment: § Age 10-18 years § Raxone® (900 mg/d): N=31 § No selection for mutational status § Placebo: N=33 § Patients had to be off chronic steroids § Mean Age: 14.3 y § 92% of patients were non-ambulatory § Treatment duration:
Slide 10: Raxone delays the loss in PEF%p
10 Raxone® delays the loss in PEF%p ITT population p=0.22 -2.57 71% reduction in loss p<0.001 of PEF%p ® -8.84 Difference 3.90 8.32 7.60 6.27 p-value 0.10 <0.001 0.02 0.03 Annual Shareholder Meeting 2015
Slide 11: Raxone delays the loss in PEF [L/min]
11 Raxone® delays the loss in PEF [L/min] ITT population +1.72 p=0.85 No decline in PEF p=0.004 -26.38 ® Difference 14.1 33.6 32.7 28.1 p-value 0.18 0.002 0.02 0.03 Annual Shareholder Meeting 2015
Slide 12: Raxone delays the loss of respiratory function
12 Raxone® delays the loss of respiratory function Consistency of results favors Raxone® favors Raxone® Annual Shareholder Meeting 2015
Slide 13: Fewer patients on Raxone fell below critical 1 L-threshold in FVC
13 Fewer patients on Raxone® fell below critical 1 L-threshold in FVC Am J Respir Crit Care Med Vol 164. pp 2191–2194, 2001 No. of Patients Raxone® Placebo BL FVC: > 1L but < 1.5 L 6 9 Patients with FVC < 1L during 1y 1 5 The time when FVC falls below 1 L is a strong marker of subsequent mortality (
Slide 14: Fewer patients on Raxone experience respiratory tract disease
14 Fewer patients on Raxone® experience respiratory tract disease ® p = 0.028 hazard ratio = 0.41 (95%CI 0.19-0.91) Cox Proportional Hazards Model Annual Shareholder Meeting 2015
Slide 15: Emerging DMD Treatment Landscape
15 Emerging DMD Treatment Landscape Raxone® Annual Shareholder Meeting 2015
Slide 16: Positioning of Raxone in the treatment of DMD
16 Positioning of Raxone® in the treatment of DMD Clinical: § First and only treatment for DMD patients not using steroids Ø ~40% of DMD patient population is not using steroids (due to side effects) § No restriction to specific mutational or disease status § Oral medication (2 tablets, 3 times/day)
Slide 17: Raxone in Leber's Hereditary Optic Neuropathy (LHON)
17 Raxone® in Leber’s Hereditary Optic Neuropathy (LHON) Leber’s Hereditary Optic Neuropathy (LHON): Marketing Authorization Application (MAA) under review in EU Annual Shareholder Meeting 2015
Slide 18: LHON – an inherited form of blindness
18 LHON – an inherited form of blindness § (Ultra-) orphan disease: Prevalence ~ 2 in 100’000 § Genetic disease with clear diagnosis and family pattern § Predominantly young males in all ethnic groups affected § Rapid loss of central vision by functional loss and degeneration of retinal nerve cells
Slide 19: Leber's Hereditary Optic Neuropathy (LHON)
19 Leber’s Hereditary Optic Neuropathy (LHON) Healthy Vision LHON Vision Annual Shareholder Meeting 2015
Slide 20: 50% of LHON patients treated with Raxone experience clinically relevant recovery of vision
20 50% of LHON patients treated with Raxone® experience clinically relevant recovery of vision Annual Shareholder Meeting 2015
Slide 21: Temporary Authorization in France; CHMP Decision for Europe awaited
21 Temporary Authorization in France CHMP Decision for Europe awaited § Authorization for Temporary Use (ATUc) granted § Marketing Authorization Application (MAA) for Europe filed in May 2014 (validated in June 2014) § Decision by EMA’s CHMP expected in 1H 2015 Annual Shareholder Meeting 2015
Slide 22: European Commercialization Plans
22 European Commercialization Plans § Hired Chief Commercial Officer for Europe and RoW § Commercial presence build-up in 4 FINLAND regional clusters NORWAY F,BeNeLux SWEDEN ESTONIA D,A,CH,CZ LATVIA DENMARK LITHUANIA Southern IRELAND BRITAIN NETH. POLAND UK, Nordic BELG. GERMANY CZ LUX. SLOVAKIA § N
Slide 23: Catena/Raxone in primary progressive MS
23 Catena®/Raxone® in primary progressive MS Primary progressive MS (ppMS): Phase 2 study in collaboration with NIH Annual Shareholder Meeting 2015
Slide 24: Phase II study in ppMS with Raxone
24 Phase II study in ppMS with Raxone® § primary progressive MS Ø affects 10-15% of total MS population Ø no approved treatment available Ø mitochondrial pathology § Phase II study (IPPoMS) in collaboration with NIH ongoing § Study is fully recruited § Santhera has exclusive license to granted use p
Slide 25: Omigapil – in development as potential treatment for Congenital Muscular Dystrophies
25 Omigapil – in development as potential treatment for Congenital Muscular Dystrophies (CMD) Me N CO2H In-licensed from Novartis: . CO2H O Demir et al., Neuropediatrics, 2004 Lampe and Bushby, JMedGenet, 2005 Annual Shareholder Meeting 2015
Slide 26: First ever clinical study in CMD - CALLISTO
26 First ever clinical study in CMD - CALLISTO Phase I trial Objectives: • Pharmacokinetic profile in children with CMD • Safety and tolerability • Feasibility of efficacy assessments Novel liquid formulation Me N CO2H . CO2H O Annual Shareholder Meeting 2015
Slide 27: Summary and Outlook
27 Summary and Outlook § Regulatory Decisions and Pipeline Ø CHMP opinion on MAA for Raxone ® in LHON expected in 1H 2015 Ø Preparation for regulatory filings for DMD ongoing Ø Expand pipeline for neuromuscular and mitochondrial diseases § Commercial Plans Ø Commercial roll-out in Europe initiated Ø
Slide 28: Advancing mitochondrial medicine towards a first treatment in LHON
28 Advancing mitochondrial medicine towards a first treatment in LHON DMD ppMS Thank you for your attention
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