Santhera Pharmaceuticals Holding AG · SIX:SANN
Santhera Pharmaceuticals — Merger or Acquisition (December 2018)
16 slides from Santhera Pharmaceuticals's merger presentation, Dec 11, 2018
Slide 1: Santhera to Acquire Option from Idorsia for Exclusive Sub-License to Vamorolone
Santhera to Acquire Option from Idorsia for Exclusive Sub-License to First-in-class Dissociative Steroid Vamorolone Presentation at Extraordinary Shareholder Meeting, 11 December 2018 Thomas Meier, PhD CEO
Slide 2: Disclaimer
Disclaimer This presentation is not and under no circumstances to be construed as a solicitation, offer, or recommendation, to buy or sell securities issued by Santhera Pharmaceuticals Holding AG. Santhera Pharmaceuticals Holding AG makes no representation (either express or implied) that the inform
Slide 3: Vamorolone – a breakthrough treatment for patients with DMD
Vamorolone – a breakthrough treatment for patients with Duchenne muscular dystrophy (DMD)
Slide 4: Vamorolone: a transformational opportunity for Santhera
Vamorolone: a transformational opportunity for Santhera • Glucocorticoids (GCs) are recognized standard of care in children and adolescent patients with DMD • High-dose GCs have severe systemic side-effects preventing lifelong treatment • Regulators and patients/families seek better tolerable altern
Slide 5: Urgent medical need for new treatments in DMD
Urgent medical need for new treatments in DMD • Glucocorticoids (GCs) are recognized standard of care in children and adolescent patients • Therapeutic goal of GCs is to preserve upper and lower limb strength/function and ambulation • EMFLAZA (deflazacort) approved and marketed in the US by PTC Ther
Slide 6: Vamorolone – discovered and developed by
Vamorolone – discovered and developed by Eric Hoffman, PhD John McCall, PhD Kanneboyina Nagaraju, PhD President and CEO Chairman of the Board Treasurer, Secretary Vice President of Chemistry Vice President of Research 6 Extraordinary Shareholder Meeting l 11 Dec 2018
Slide 7: Vamorolone
Vamorolone • First-in-class dissociative steroidal anti-inflammatory drug • Close analog to prednisone, a standard glucocorticoid (GC) • Different pharmacological properties allow dissociation of beneficial effects from GC-class side effects Source: www.reveragen.com 7 Extraordinary Shareholder Meet
Slide 8: Vamorolone – revolutionary mode of action
Vamorolone – revolutionary mode of action Source: www.reveragen.com 8 Extraordinary Shareholder Meeting l 11 Dec 2018
Slide 9: Vamorolone – current efficacy and safety data
Vamorolone – current efficacy and safety data Effects of vamorolone in animal model for DMD: • Retains GC-type anti-inflammatory efficacy and reduces dystrophy, improves muscle strength and motor function • Superior safety profile with respect to loss of stunting growth, bone symptoms, cardiac side
Slide 10: Vamorolone – pivotal Phase IIb trial (VBP15-004), ongoing
Vamorolone – pivotal Phase IIb trial (VBP15-004), ongoing The Vision-DMD trial Design Phase IIb randomized, double-blind, parallel group, placebo- and active-controlled study with double-blind extension Participants 120 ambulant boys ages 4 to <7 years, not taking steroids Randomization 1:1:1:1 rand
Slide 11: Estimated time to market, protection and sales potential
Estimated time to market, protection and sales potential NDA: new drug application; MAA: marketing authorization application Protection and Regulatory Status Competitive Positioning and Sales Potential • Orphan drug protection: USA (7y) and EU (10y) • Vamorolone to become standard of care • Method o
Slide 12: Excellent strategic fit – Vamorolone complements Santhera's DMD program
Excellent strategic fit – Vamorolone complements Santhera’s DMD program and strengthens its pipeline
Slide 13: Pipeline synergies between vamorolone and idebenone
Pipeline synergies between vamorolone and idebenone • Combination of vamorolone and idebenone addresses medical need of DMD patients at all disease stages • Vamorolone and idebenone could be used in all patients (not restricted to certain mutations) • Combination therapy to be evaluated vamorolone D
Slide 14: Targeted time to market for Santhera's DMD franchise
Targeted time to market for Santhera’s DMD franchise 14 Extraordinary Shareholder Meeting l 11 Dec 2018
Slide 15: Santhera's pro forma pipeline post-transaction
Santhera’s pro forma pipeline post-transaction Santhera Pipeline Drug Preclin. Phase I PoC Pivotal Filing Market Neuro-ophthalmological diseases Leber’s hereditary optic neuropathy Idebenone Raxone® Neuromuscular diseases Duchenne muscular dystrophy (GC non- users) Idebenone completed Duchenne muscu
Slide 16: Thank you for your attention
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