Santhera Pharmaceuticals Holding AG · SIX:SANN
Santhera Pharmaceuticals — Shareholder Meeting (April 2017)
25 slides from Santhera Pharmaceuticals's shareholder meeting presentation, Apr 4, 2017
Slide 1: Advancing Mitochondrial Medicine
Advancing Mitochondrial Medicine Thomas Meier, CEO Ordentliche Generalversammlung Basel, 4. April 2017
Slide 2: First full year as commercial company in EU
2 First full year as commercial company in EU • Sales in 2016: CHF 19 million • 2016: Raxone® sold in 15 countries, sales primarily in FR and DE • Sales target for 2017: CHF 21-23 million Santhera ASM │ 4 April 2017
Slide 3: Successful European Launch of Raxone
3 Successful European Launch of Raxone® • Raxone®: EU approval for patients with LHON (first approved medication) • Commercial presence in 4 regional clusters (western Europe) • Distribution partner Ewopharma for eastern European countries • Headcount commercial team: ~ 40 Santhera ASM │ 4 April 201
Slide 4: Leber's Hereditary Optic Neuropathy (LHON): the clinical presentation
4 Leber’s Hereditary Optic Neuropathy (LHON): the clinical presentation Days, weeks or few months Santhera ASM │ 4 April 2017
Slide 5: Santhera's LHON Ambassador: Fabrizio Sottile
5 Santhera’s LHON Ambassador: Fabrizio Sottile • Age: 24 years • Diagnosed with LHON at age 17 • Differential diagnosis took over 5 months (misdiagnosed as MS and brain tumor) • Treated with idebenone • Has now stable preserved vision (45% left eye and 10-15% right eye) • Medal winner in swimming ch
Slide 6: Clinical efficacy of Raxone in LHON
6 Clinical efficacy of Raxone® in LHON Santhera ASM │ 4 April 2017
Slide 7: Progress with Raxone in DMD
7 Progress with Raxone® in DMD • Filing of Marketing Authorization Application for Raxone® for the treatment of Duchenne Muscular Dystrophy (DMD) in EU and Switzerland • Start of new Phase 3 trial SIDEROS in DMD patients on concomitant glucocorticoid treatment • Orphan drug designation granted for A
Slide 8: Positioning of Raxone as treatment of DMD
8 Positioning of Raxone® as treatment of DMD Santhera ASM │ 4 April 2017
Slide 9: Respiratory complications in patients with DMD
9 Respiratory complications in patients with DMD Santhera ASM │ 4 April 2017
Slide 10: Respiratory complications in patients with DMD
10 Respiratory complications in patients with DMD Santhera ASM │ 4 April 2017
Slide 11: Respiratory complications and early morbidity
11 Respiratory complications and early morbidity Santhera ASM │ 4 April 2017
Slide 12: The medical need …. raising awareness
12 The medical need …. raising awareness Santhera ASM │ 4 April 2017
Slide 13: Development program with Raxone in DMD
13 Development program with Raxone® in DMD Phase 3 Phase 2 Phase 3 GC: glucocorticoid steroids; PPM study: prospectively planned matching study Santhera ASM │ 4 April 2017
Slide 14: Understanding the clinical value of Raxone in the treatment of DMD
14 Understanding the clinical value of Raxone® in the treatment of DMD Santhera ASM │ 4 April 2017
Slide 15: Initial target population for Raxone in DMD
15 Initial target population for Raxone® in DMD Patients with respiratory function decline currently not using glucocorticoids patients 8 years and older not using glucocorticoids represent 40% of total DMD population Santhera ASM │ 4 April 2017
Slide 16: SIDEROS – a new trial with Raxone in patients with DMD using concomitant GCs
16 SIDEROS – a new trial with Raxone® in patients with DMD using concomitant glucocorticoids (GCs) Phase 3 Phase 2 Phase 3 GC: glucocorticoid steroids; PPM study: prospectively planned matching study Santhera ASM │ 4 April 2017
Slide 17: The SIDEROS trial
17 The SIDEROS trial To assess the efficacy of idebenone (Raxone®) compared to placebo, in slowing the loss of respiratory function in patients with DMD receiving glucocorticoids (GCs) Santhera ASM │ 4 April 2017
Slide 18: Finding patients for SIDEROS
18 Finding patients for SIDEROS Santhera ASM │ 4 April 2017
Slide 19: Near-term regulatory objective
19 Near-term regulatory objective • Based on DELOS data obtain early approval for DMD patients not using steroids The intended indication is for patients in whom respiratory function has started to decline and who are currently not taking glucocorticoids. The indication would include patients who pr
Slide 20: Management targets US opportunity
20 Management targets US opportunity Todd Bazemore Chief Operating Officer of Santhera Pharmaceuticals (USA), Inc. Objectives for U.S. team for 2017: • Intensify interactions with clinical experts, patients advocacy groups and FDA • Develop regulatory strategy for early approval of Raxone® in DMD •
Slide 21: Progress with omigapil in congenital muscular dystrophy (CMD)
21 Progress with omigapil in congenital muscular dystrophy (CMD) • FDA granted Fast Track Designation for omigapil for the treatment of CMD • Received FDA Grant in support of the ongoing CALLISTO trial with omigapil The CALLISTO trial with omigapil in patients with CMD • Conducted at the US National
Slide 22: Santhera Pipeline
22 22 Santhera Pipeline LHON DMD PPMS CMD Santhera ASM │ 4 April 2017
Slide 23: Future opportunities for Santhera in advancing mitochondrial medicine
23 Future opportunities for Santhera in advancing mitochondrial medicine Source: Foundation for Mitochondrial Medicine Santhera ASM │ 4 April 2017
Slide 24: Outlook 2017
24 Outlook 2017 • Continue commercial roll-out in EU for Raxone® as treatment of LHON • EU: Obtain Marketing Authorization for Raxone® as treatment of DMD • US: Evaluate possibility for Accelerated Approval for Raxone® in DMD with FDA • Complete enrolment of SIDEROS trial in DMD • Complete CALLISTO
Slide 25: Advancing Mitochondrial Medicine
Advancing Mitochondrial Medicine
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