Santhera Pharmaceuticals Holding AG · SIX:SANN
Santhera Pharmaceuticals — Investor Presentation (March 2022)
28 slides from Santhera Pharmaceuticals's investor presentation, March 2022
Slide 1: Santhera Pharmaceuticals Corporate Presentation
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases Corporate Presentation MAR 2022
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: Santhera Pharmaceuticals Corporate Snapshot
SIX Swiss Exchange listed company (SANN) 1 Global headquarters near Basel (Switzerland) with internationally experienced leadership team North American headquarters in Boston (USA) with team growing under new leadership US NDA filing for vamorolone in Duchenne muscular dystrophy started in Q1-2022 2
Slide 4: Santhera pipeline offers an attractive investment opportunity
Santhera pipeline offers an attractive investment opportunity Two assets with broad therapeutic potential and opportunities beyond current active programs Lonodelestat targeting Vamorolone foundational therapy in Duchenne MD inflammation in cystic fibrosis • Positive pivotal data in Phase 2b trial •
Slide 5: Pipeline offers promising therapeutic options in rare disease areas
Pipeline offers promising therapeutic options in rare disease areas Rolling NDA filing in DMD started in Q1-2022 for lead asset vamorolone Molecule Indication IND Ph 1 PoC Pivotal Filing Market Milestones and remarks Q2-21: Positive pivotal data Duchenne VISION-DMD Q4-21: Successful study completion
Slide 6: Vamorolone in Duchenne muscular dystrophy and potentially other inflammatory disorders
Vamorolone in Duchenne muscular dystrophy and potentially other inflammatory disorders 6
Slide 7: DMD offers attractive opportunity in well-defined orphan disease market
DMD offers attractive opportunity in well-defined orphan disease market DMD market with few current treatment options, projected to be worth Small teams needed to cover DMD Centers HCPs > USD 4 billion by 2023* entire market in US ~160 ~450 EU and US • Approx. 30,000 – 35,000 patients in US and EU4+
Slide 8: Corticosteroids delay disease progression by 2–3 years
Corticosteroids delay disease progression by 2 – 3 years4,6 Established endpoints and consistent evidence base from several clinical studies age (years) Corticosteroids are a standard of care 0 5 10 15 20 25 30 Impaired ability to hop, run, jump, rise from floor • DMD progression is sequential, non-
Slide 9: Corticosteroid treatment is associated with well-defined toxicities
Corticosteroid treatment is associated with well-defined toxicities …up to 65% of patients discontinue treatment early due to adverse events3-5 37% Growth failure/delay2,3 65% Weight gain3 37% Behavioral changes3 31% Excessive hair growth2,3 30% Bone changes or fractures2,3 19% Cataracts2 55% Cushin
Slide 10: Corticosteroid use is limited due to known side effect profile
Corticosteroid use is limited due to known side effect profile Use of corticosteroids in DMD is high, particularly in ambulatory patients but declines with age4 Reasons for Discontinuing Steroid Treatment4 Problems with side effects Not enough benefits Did not like use of long term medication 65.2 C
Slide 11: Vamorolone retains benefits of steroids with fewer side effects
Vamorolone retains benefits of steroids with fewer side effects1-3 Glucocorticoid Receptor Ligand Binding Domain Like corticosteroids4-5 • inhibition of NF-кB pro-inflammatory transcription factors Retained efficacy due to potent anti-inflammatory action Vamorolone Unlike corticosteroids4-5 • Not a
Slide 12: Tested in a comprehensive clinical development program
Tested in a comprehensive clinical development program 200 patient-years exposure in 160 DMD boys treated with vamorolone for up to 2.5 years1 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024 Ph 1 (N=86) Ph 2a (N=48) Pivotal VISION-DMD (N=121) age 4 – <7 years NDA (US)* MAA (EU) Phase 2a Long Term
Slide 13: Pivotal VISION-DMD: Study design
Pivotal VISION-DMD: Study design Randomized, double-blind, placebo and active control trial in 121 steroid-naive patients, aged 4 – <7 yrs Primary Analysis 5 weeks 24 weeks 4 weeks 20 weeks • FDA & EMA: Primary analysis vs placebo at 24 weeks • Long-term safety and switch from prednisone to vamorolo
Slide 14: Primary endpoint met with high statistical significance at 24 weeks
Primary endpoint met with high statistical significance at 24 weeks Secondary endpoints with statistical significance and clinically relevant treatment differences (4 out of 5) Rank Endpoint Comparison vs placebo Difference P-value Primary TTSTAND velocity vam 6mg/kg/d 0.06 rises/s 0.002 TTSTAND vel
Slide 15: Primary endpoint met with clinically relevant treatment difference
Primary endpoint met with clinically relevant treatment difference Treatment difference of 0.05 rises/sec predictive of delay of 2 – 3 years in time to loss of ambulation1 TTSTAND velocity (rises/sec)2 0.08 0.06 Change from Baseline (SEM) 0.04 0.02 p=0.002 0.00 23% improvement in time to rise after
Slide 16: No significant difference for vamorolone vs prednisone across endpoints
No significant difference for vamorolone vs prednisone across endpoints Percentual change from baseline comparing groups at week 24 35 PLA VAM 2 VAM 6 PDN Percentual change from baseline 30 25 20 15 10 5 0 -5 -10 TTSTAND velocity 6MWT distance NSAA score TTRW velocity PDN: Prednisone 0.75 mg/kg/d; V
Slide 17: No loss of efficacy when switching from prednisone to vamorolone
No loss of efficacy when switching from prednisone to vamorolone Durable treatment effect maintained over 48 weeks with vamorolone 6 mg/kg/d1 TTSTAND velocity (rises/sec) 1 0.08 Period 1 Period 2 • During treatment period 1, patients on vamorolone 6 mg/kg/d showed same change 0.06 in TTSTAND velocit
Slide 18: Adverse events decreased after switching from prednisone to vamorolone
Adverse events decreased after switching from prednisone to vamorolone Number of new or worsening adverse events (AE) or adverse events of special interest (AESI) Analysis in population of 30 patients on prednisone switching to either vamorolone 2 or 6 mg/kg/d with 15 patients per dose i.e. same 30
Slide 19: Vamorolone allows for normal bone development and growth
Vamorolone allows for normal bone development and growth Comparison to prednisone upon switching to vamorolone and analyses of long-term use Switching from prednisone to vamorolone recovers Long-term use of vamorolone did not stunt growth normal growth trajectory unlike other corticosteroids used in
Slide 20: Summary of key findings from VISION-DMD pivotal trial
Summary of key findings from VISION-DMD pivotal trial Placebo controlled treatment followed by randomized vamorolone treatment at 2 doses Treatment Period 1 Treatment Period 2 Baseline to week 24 Week 24 to week 48 Placebo controlled Vamorolone treatment Vamorolone at 2 doses vs placebo and predniso
Slide 21: Vamorolone program in DMD rolling NDA started
Vamorolone program in DMD rolling NDA started Pre-NDA meeting with FDA confirmed filing based on current data package for efficacy and safety FDA filing started in Q1-2022* with planned EMA filing in Q3-2022 based on pivotal placebo-controlled VISION-DMD study • Positive primary (p<0.002, TTSTAND ve
Slide 22: Lonodelestat in cystic fibrosis and potentially other inflammatory pulmonary disorders
Lonodelestat in cystic fibrosis and potentially other inflammatory pulmonary disorders 22
Slide 23: Cystic fibrosis is a rare genetic lung disorder with unmet medical need
Cystic fibrosis is a rare genetic lung disorder with unmet medical need Genetics Cause Patients Symptoms Medical need Autosomal recessive Mutations in the CF More than 80,000 Persistent lung No approved disorder diagnosed at transmembrane patients in US and infections, chronic treatment specifically
Slide 24: Lonodelestat targets elastase, a protease responsible for lung damage
Lonodelestat targets elastase, a protease responsible for lung damage Pathological levels of neutrophil elastase (NE) during inflammation destroy lung tissue over time Lonodelestat is a highly potent, reversible and selective NE inhibitor • Effective in pico-molar range (Ki 0.05nM) inhibiting Lonode
Slide 25: Effect on inflammatory biomarker at a safe dose established in Phase 1
Effect on inflammatory biomarker at a safe dose established in Phase 1 Single and multiple ascending dose (SAD & MAD) studies supported by the Cystic Fibrosis Foundation and successfully completed with lonodelestat Phase 1 SAD in healthy volunteers (Barth et al. 2020) Linear dose relationship and we
Slide 26: Successful Phase 1 program paves way for further clinic development
Successful Phase 1 program paves way for further clinic development Key achievements in CF development program Next steps in CF • Safe dose regimen identified • Preparation of Phase 2a program in patients • Effect on inflammatory biomarker established currently non-eligible for CFTR modulator therap
Slide 27: Financial overview
Financial overview • Cash runway through mid-2022 • CHF 21 million cash & cash equivalents and CHF 8 million available facility at Dec 31, 2021 • CHF 42 million (net proceeds) financing completed September 2021 • Comprising CHF 20 million share issue; CHF 15 million Bond issue; CHF 10 million loan f
Slide 28: Santhera Pharmaceuticals
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases MAR 2022
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