Santhera Pharmaceuticals Holding AG · SIX:SANN
Santhera Pharmaceuticals — Investor Presentation (December 2023)
39 slides from Santhera Pharmaceuticals's investor presentation, Dec 22, 2023
Slide 1: Santhera Pharmaceuticals Corporate Presentation
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases Corporate Presentation December 22, 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) Global headquarters near Basel (Switzerland) with internationally experienced leadership team Own commercialization of lead asset in EU core countries Strong rare disease development capabilities AGAMREE® (vamorolone) in Duchenne muscular dystrophy approved S
Slide 4: Santhera pipeline with two assets and broad therapeutic potential
Santhera pipeline with two assets and broad therapeutic potential Opportunities beyond current active program in Duchenne muscular dystrophy (DMD) AGAMREE® (vamorolone) foundational therapy in DMD Lonodelestat targeting inflammation pulmonary disease • U.S. FDA full approval on Oct 26, 2023, for DMD
Slide 5: Lead asset AGAMREE in DMD approved by FDA and EMA
Lead asset AGAMREE® in DMD approved by FDA and EMA Launch expected in Q1-2024 in the U.S. and EU Molecule Indication IND Ph 1 PoC Pivotal Filing Market Milestones and remarks North America & China partnerships Duchenne Approved in US and EU muscular dystrophy Vamorolone1 Trial under FDA grant to par
Slide 6: AGAMREE (vamorolone) in Duchenne muscular dystrophy and potentially other inflammatory disorders
AGAMREE® (vamorolone) in Duchenne muscular dystrophy and potentially other inflammatory disorders 6
Slide 7: Full approval by FDA and EC
Full approval by FDA for treatment of patients 2 years and older on Oct 26 Full approval by EC for treatment of patients 4 years and older on Dec 18 MHRA decision expected soon as part of EC reliance procedure 7 Corporate Presentation December 2023 EC: European Commission; CHMP: Committee for Medici
Slide 8: 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 Focused expert options, projected to be worth centers treating DMD Centers HCPs > USD 4 billion by 2023* patients in U.S. ~90 ~450 EU and U.S. • Approx. 30,000 – 35,000 patients in U.S.
Slide 9: AGAMREE can fill the need for a better foundational therapy in DMD
AGAMREE® can fill the need for a better foundational therapy in DMD Corticosteroids delay disease progression by 2-3 years, but associated toxicities limit their use ESTABLISHED SAFETY ISSUES VAMOROLONE EFFICACY OF WITH STEROIDS OFFERING STEROIDS Classical corticosteroids Classical corticosteroids S
Slide 10: 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 11: 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 12: AGAMREE (vamorolone) dissociative properties
AGAMREE® (vamorolone) dissociative properties Subtle but impactful difference in chemical structure separates vamorolone from classical steroids1-5 Like corticosteroids, efficacy maintained by potent anti-inflammatory action • Retained inhibition of NF-кB pro-inflammatory transcription factor Unlike
Slide 13: Comprehensive AGAMREE (vamorolone) development
Comprehensive AGAMREE® (vamorolone) development2-9 200 patient-years exposure in 160 DMD boys treated with vamorolone for up to 6 years1 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* Pivo
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 years 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 Comparison vs Rank Endpoint Difference MCID P-value placebo Primary TTSTAND velocity vam 6mg/kg 0.06 rises/
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; Time to Stand (TTSTAND), 6 Minute W
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 data1 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: Phase
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 Bone Health Unlike classical corticosteroids, vamorolone does not have a negative impact Biomarkers of bone formation1 Biomarkers of bone remodelling1 Osteocalcin (ng/ml) P1NP (ng/ml) CTX1 (pg/ml) Osteocalcin (ng/mL
Slide 23: Rapid recovery of bone biomarkers after switching from prednisone
Bone biomarker data from VISION-DMD study supports findings on long-term bone health Bone Health Rapid recovery of bone biomarkers after switching from prednisone Biomarkers of bone formation1 Biomarkers of bone remodelling1 Osteocalcin (ng/ml) P1NP (ng/ml) CTX1 (pg/ml) 400 20 150 Estimated mean cha
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: AGAMREE (vamorolone) clinical data value proposition
AGAMREE® (vamorolone) clinical data value proposition • Durable efficacy comparable to standard of care with AGAMREE® 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 ef
Slide 27: Full approval by FDA and positive CHMP opinion for AGAMREE in DMD
Full approval by FDA and positive CHMP opinion for AGAMREE® in DMD • Approvals obtained in 2023: U.S. on Oct 26, EU on Dec 18 (and UK decision expected soon) • Orphan drug exclusivity in U.S. (7 years) and Europe (12 years incl. pediatric extension) • Patent protection at least until 2040 (U.S.) and
Slide 28: Santhera commercial launch in key European geographies
Santhera commercial launch in key European geographies Santhera aims to market vamorolone in DMD itself in core territory with population of ~ 340 million • First launch in Germany in Q1-2024 • Staged roll-out across the key European markets • Strong and growing stakeholder support 2 1 • Lean commer
Slide 29: Santhera commercial set-up with central hub structure at headquarters
Santhera commercial set-up with central hub structure at headquarters Headquarter core functions collaborate with license partners and support own lean own country teams as well as distribution partners License Partners • Catalyst • Sperogenix Patient Medical Advocacy Affairs Santhera Organizations
Slide 30: Market opportunity to change the foundational therapy in DMD
Market opportunity to change the foundational therapy in DMD AGAMREE® can adress the shortcomings of current standard of care corticosteroid use • Current corticosteroid use • With 60-70% of patients on steroid treatment, currently up Issues with current steroid use to 8,000 boys/men are being treat
Slide 31: Vamorolone in Becker muscular dystrophy
Vamorolone in Becker muscular dystrophy 31
Slide 32: 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 33: 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 34: Lonodelestat in cystic fibrosis and potentially other inflammatory pulmonary disorders
Lonodelestat in cystic fibrosis and potentially other inflammatory pulmonary disorders 34
Slide 35: 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 U.S. and infections, chronic treatment specifical
Slide 36: 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 time1 Lonodelestat is a highly potent, reversible and selective NE inhibitor • Effective in pico-molar range (Ki 0.05nM) inhibiting free
Slide 37: 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 38: Santhera financial status
Santhera financial status Santhera Pharmaceuticals is listed on the Swiss Stock Exchange SIX: Ticker SANN • Key figures (CHF million* as of June 30, prior closing • Recent milestones Catalyst agreement in July 2023) • 07-2023: AGAMREE® U.S. licensing to Catalyst • Net (loss) for the period (23.3) R
Slide 39: Santhera Pharmaceuticals
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases December 22, 2023
Santhera Pharmaceuticals — Investor Presentation (December 2023) on 6ix