Santhera Pharmaceuticals Holding AG · SIX:SANN
Santhera Pharmaceuticals — Investor Presentation (October 2023)
36 slides from Santhera Pharmaceuticals's investor presentation, Oct 27, 2023
Slide 1: Santhera Pharmaceuticals Corporate Presentation
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases Corporate Presentation October 27, 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 Own commercialization of lead asset in EU core countries Strong rare disease development capabilities 2 AGAMREE® (vamorolone) in Duchenne muscular dystrophy approv
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 in the U.S. on Oct 26
Lead asset AGAMREE® in DMD approved by FDA in the U.S. on Oct 26 Positive CHMP opinion with expected approval in Dec 2023 by EC as well as in UK by MHRA Molecule Indication IND Ph 1 PoC Pivotal Filing Market Milestones and remarks North America & China partnerships Duchenne VISION-DMD muscular dystr
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: Positive CHMP opinion and full FDA approval
Positive opinion by CHMP for Full approval by FDA for treatment of patients 4 years treatment of patients 2 years and older on Oct 13 and older on Oct 26 7 Corporate Presentation October 2023 EC: European Commission; CHMP: Committee for Medicinal Products for Human Use
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 options, projected to be worth Focused expert centres 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. and
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 Su
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 • Approval obtained in U.S. on Oct 26 and EC marketing authorization expected in late 2023 • Filings under review in UK with decision expected in Q4-2023 • Orphan drug exclusivity in U.S. (7 years) and Europe (12 years incl. ped. ext
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 U.S. and infections, chronic treatment specifical
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 time1 Lonodelestat is a highly potent, reversible and selective NE inhibitor • Effective in pico-molar range (Ki 0.05nM) inhibiting free
Slide 34: 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 35: 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, 2023*) • Recent milestones • Net (loss) for the period (23.3) • 07-2023: AGAMREE® U.S. licensing to Catalyst Raxone ®/idebenone divestment to Chiesi •
Slide 36: Santhera Pharmaceuticals
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases October 2023
Santhera Pharmaceuticals — Investor Presentation (October 2023) on 6ix