Santhera Pharmaceuticals Holding AG · SIX:SANN
Santhera Pharmaceuticals — Investor Presentation (March 2023)
37 slides from Santhera Pharmaceuticals's investor presentation, March 2023
Slide 1: Santhera Pharmaceuticals Corporate Presentation
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases Corporate Presentation March 2023
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 near Boston (USA) with team growing under new leadership Regulatory filings completed for vamorolone in Duchenne muscular dystrophy 2 N
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 pulmonary disease • US NDA and EU MAA accepted and under revie
Slide 5: Pipeline offers promising therapeutic options in rare disease areas
Pipeline offers promising therapeutic options in rare disease areas US NDA and EU MAA, both accepted and under review for potential approval in Q4-2023 Molecule Indication IND Ph 1 PoC Pivotal Filing Market Milestones and remarks Oct-22: MAA filing validated by EMA Duchenne VISION-DMD Jan-23: NDA fi
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 ~90 ~450 EU and US • Approx. 30,000 – 35,000 patients in US and EU4+U
Slide 8: The need for a better foundational steroid therapy in DMD
The need for a better foundational steroid therapy in DMD EFFICACY OF SAFETY ISSUES VAMOROLONE STEROIDS WITH STEROIDS OFFERING Classical corticosteroids Classical corticosteroids Subtle difference in demonstrate efficacy are associated with steroid ring structure with delay in significant side effec
Slide 9: Corticosteroids delay disease progression in DMD by 2–3 years
Corticosteroids delay disease progression in DMD by 2 – 3 years4,6 Established endpoints and consistent evidence base through several clinical studies age (years) Corticosteroids are the standard of care 0 5 10 15 20 25 30 Impaired ability to hop, run, jump, rise from floor • DMD progression is sequ
Slide 10: Corticosteroid treatment is associated with well-defined toxicities
Corticosteroid treatment is associated with well-defined toxicities …up to 65% of DMD patients discontinue treatment early due to adverse events1-3 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% Cu
Slide 11: 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 age1-4 Reasons (%) for Discontinuing Steroid Treatment4 Problems with side effects Not enough benefits Did not like use of long-term medication
Slide 12: 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 13: Comprehensive vamorolone development
Comprehensive vamorolone development 2-9 200 patient-years exposure in 160 DMD boys treated with vamorolone for up to 6 years 1 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024 2025 Ph 1 (N=86) Ph 2a (N=48) Ph 2a Long-term Extension Expanded Access Program (N=153) Long term follow-up* Pivotal VISIO
Slide 14: 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. 48 weeks Vamorolone 6 mg/kg/day Vamorolone 2 mg/kg/day Screening Placebo Transition Vamorolone 6 mg/kg/day Placebo Transition Vamorolone 2 mg/kg/day Prednisone
Slide 15: Primary endpoint met with high statistical significance at 24 weeks
Primary endpoint met with high statistical significance at 24 weeks Consistent and robust efficacy shown by primary endpoint and majority of secondary endpoints for both vamorolone doses Rank Endpoint Comparison vs placebo Difference P-value Primary TTSTAND velocity vam 6mg/kg/d 0.06 rises/s 0.002 T
Slide 16: Primary endpoint met with clinically relevant treatment difference
Primary endpoint met with clinically relevant treatment difference Observed difference of 0.06 rises/sec is expected to delay the time to loss of ambulation by 2-3 years1 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 0
Slide 17: Comparable efficacy of vamorolone 6 mg/kg/d vs prednisone 0.75 mg/kg/d
Comparable efficacy of vamorolone 6 mg/kg/d vs prednisone 0.75 mg/kg/d Difference between groups in percentual change from baseline at week 24 (post hoc analysis) FAVORS PREDNISONE FAVORS VAMOROLONE PDN: Prednisone 0.75 mg/kg/d; VAM: Vamorolone at 2 and 6 mg/kg/d; PLA: Placebo; Time to Stand (TTSTAN
Slide 18: 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 19: The FOR-DMD study provides external comparator data
The FOR-DMD study provides external comparator data 1 Pre-specified analyses in double-blind, randomized, academic-run, independent study DMD boys 4- <7 VBP15-LTE: Phase 2a, open-label long-term extension up to 30 months (2-6 mg/kg/day) Steroid naive N=121 (Pivotal Phase 2b, 48-wks) Vision-DMD: Phas
Slide 20: VISION-DMD pre-specified analyses vs FOR-DMD external control
VISION-DMD pre-specified* analyses vs FOR-DMD external control Propensity matched cross study comparison shows comparable efficacy for vamorolone 6 mg/kg/d versus standard of care corticosteroid treatment TTSTAND velocity (rises/sec) 80 6-MWT (m) Mean (SEM) change from baseline 70 Mean (SEM) change
Slide 21: Fewer and less severe spinal fractures with vamorolone
Fewer and less severe spinal fractures with vamorolone compared to classical corticosteroids over 2.5 years Bone Health Vamorolone long-term extension (LTE) study vs FOR-DMD, matched comparison, central reading using modified Genant grades1 35 30 25 Reduction of number of patients with 20 vertebral
Slide 22: Bone biomarker data from VISION-DMD supports long-term bone health
Bone biomarker data from VISION-DMD study supports findings on long-term bone health Unlike classical corticosteroids, vamorolone does not have a negative impact Bone Health Biomarkers of bone formation 1 Biomarkers of bone remodelling 1 Osteocalcin (ng/ml) P1NP (ng/ml) CTX1 (pg/ml) Osteocalcinp<0.0
Slide 23: Bone biomarker data: rapid recovery after switching from prednisone
Bone biomarker data from VISION-DMD study supports findings on long-term bone health Rapid recovery of bone biomarkers after switching from prednisone Bone Health Biomarkers of bone formation 1 Biomarkers of bone remodelling 1 Osteocalcin (ng/ml) P1NP (ng/ml) CTX1 (pg/ml) 400 20 150 Estimated mean c
Slide 24: Vamorolone allows for normal bone development and growth
Vamorolone allows for normal bone development and growth Comparison to natural history data and in patients switching from prednisone Bone Health Vamorolone did not stunt growth unlike other Switching from prednisone to vamorolone recovers corticosteroids used in DMD normal growth trajectory (VISION
Slide 25: Fewer and less severe behavioral problems reported with vamorolone
Fewer and less severe behavioral problems reported with vamorolone Comparison of behavioral problems reported for vamorolone vs prednisone at week 24 Prednisone Vamorolone Vamorolone VISION-DMD Study Placebo 0.75 mg/kg/d 2 mg/kg/d 6 mg/kg/d N = 29 N = 31 N = 30 N = 28 Behavior problems AESIs, n (%)
Slide 26: Vamorolone value proposition
Vamorolone value proposition • Durable efficacy comparable to standard of care with vamorolone 6mg/kg/day • Statistically robust efficacy vs placebo at 24 weeks for both 2mg/kg/day and 6mg/kg/day • No loss of efficacy when switching from prednisone to vamorolone • Long-term efficacy of vamorolone 6m
Slide 27: Vamorolone program in DMD submitted in US, EU and UK
Vamorolone program in DMD submitted both in US, EU and UK FDA and EMA filing both accepted and under review on pivotal VISION-DMD study and supportive long-term data Filing based on pivotal VISION-DMD study and additional long-term extension data compared to FOR-DMD natural history study • Positive
Slide 28: Vamorolone in Becker muscular dystrophy
Vamorolone in Becker muscular dystrophy 28
Slide 29: Becker muscular dystrophy disease profile and corticosteroid use
Becker muscular dystrophy (BMD) disease profile and corticosteroid use Genetics Cause Patients Symptoms Medical need X-linked recessive form Partial loss of function Higher life expectancy Progressing muscle No approved treatment of muscular dystrophy of dystrophin with a and lower prevalence weakne
Slide 30: Vamorolone holds promise in BMD based on data generated for DMD
Vamorolone holds promise in BMD based on data generated for DMD 24 weeks Vamorolone potential benefits in BMD1,2 NCT05166109 Vamorolone Sponsor ReveraGen 1. Anti-inflammatory agent with reduced side 500 mg/day or 250 mg if <50 kg body weight Safety and effects via dissociative character of vamorolon
Slide 31: Lonodelestat in cystic fibrosis and potentially other inflammatory pulmonary disorders
Lonodelestat in cystic fibrosis and potentially other inflammatory pulmonary disorders 31
Slide 32: 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 33: 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 1 Lonodelestat is a highly potent, reversible and selective NE inhibitor • Effective in pico-molar range (Ki 0.05nM) inhibiting Lono
Slide 34: 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 1 Linear dose relationship and well tolerated doses
Slide 35: Successful Phase 1 program paves way for further clinical development
Successful Phase 1 program paves way for further clinical 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 ther
Slide 36: Financial overview & upcoming milestones
Financial overview & upcoming milestones Key figures* – Jun 30, 2022 Capital structure – Mar 1, 2023 CHF million Listed SIX (SANN) • Net (loss) for the period (29.7) • Number of shares outstanding (excl treasury) 85,021,816 • Cash (used) in operations (12.0) • Market capitalization CHF 67.5 million
Slide 37: Santhera Pharmaceuticals
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases March 2023
Santhera Pharmaceuticals — Investor Presentation (March 2023) on 6ix