Santhera Pharmaceuticals Holding Ag · SIX:SANN
Santhera Pharmaceuticals — Investor Presentation (October 2024)
35 slides from Santhera Pharmaceuticals's investor presentation, Oct 1, 2024
Slide 1: Santhera Pharmaceuticals Corporate Presentation
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases Corporate Presentation October 1, 2024
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 Western EU countries Strong rare disease development capabilities AGAMREE® (vamorolone) in Duchenne muscular dystrophy Santher
Slide 4: Value driver in DMD with broad potential
Value driver in DMD with broad potential AGAMREE® (vamorolone) foundational therapy in DMD • U.S. FDA full approval on October 26, 2023; US launch on March 13, 2024 • EC full approval on December 18, 2023; German launch on Jan 15, 2024 • MHRA full approval on January 11, 2024 • Potential as alternat
Slide 5: Lead asset AGAMREE in DMD approved by FDA, EMA and MHRA
Lead asset AGAMREE® in DMD approved by FDA, EMA and MHRA Launch in Germany on January 15, 2024, by Santhera and on March 13, 2024, in the U.S. by Catalyst NDA filed in China and priority review granted to partner Sperogenix on March 27, 2024 Molecule Study / Indication Proof of Concept Pivotal Filin
Slide 6: AGAMREE (vamorolone) in Duchenne muscular dystrophy and potentially in other disorders
AGAMREE® (vamorolone) in Duchenne muscular dystrophy and potentially in other disorders where current steroid use has limitations 6
Slide 7: DMD offers attractive opportunity in well-defined orphan disease market
DMD offers attractive opportunity in well-defined orphan disease market The DMD indication with few current Focused expert treatment options is a fast-growing centers treating DMD Centers HCPs multi-billion market patients in U.S. ~90 ~450 EU and U.S. • Approx. 30,000 – 35,000 patients in U.S.
Slide 8: 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 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: 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 12: Comprehensive AGAMREE (vamorolone) development
Comprehensive AGAMREE® (vamorolone) development 2-9 200 patient-years exposure in 160 DMD boys treated with vamorolone for up to 7 years1 2015 2016 2017 2018 2019 2020 2021 2022 2023 2024 2025 Ph 1 Ph 2a Ph 2a Long-term Extension Expanded Access Program (N=153) Long-term follow-up (N=86) (N=48) Pivo
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 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 14: 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 15: 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 16: 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 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: 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 19: 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 20: 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 21: 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 22: Bone biomarker data: rapid recovery 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 23: 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 24: 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 25: AGAMREE (vamorolone) clinical data value proposition
AGAMREE® (vamorolone) clinical data value proposition • Durable efficacy comparable to standard of care with AGAMREE® 6 mg/kg/day • Statistically robust efficacy vs placebo at 24 weeks for both 2 mg/kg/day and 6 mg/kg/day • No loss of efficacy when switching from prednisone to vamorolone • Long-term
Slide 26: Full approval by FDA, EMA and MHRA for AGAMREE in DMD
Full approval by FDA, EMA and MHRA for AGAMREE® in DMD • Regulatory filings under review in Switzerland and China • Orphan drug exclusivity in U.S. (7 years) and Europe (12 years incl. pediatric extension) • Patent protection at least until 2040 (U.S.) and 2035 (EU) 2023 2024 2025 2026 Q3 Q4 Q1 Q2 Q
Slide 27: Santhera commercial launch in key European geographies
Santhera commercial launch in key European geographies Santhera aims to market vamorolone in DMD itself in territory with population of ~ 410 million • First launch in Germany in January 2024 • Staged roll-out across the key European markets • Strong and growing stakeholder support • Lean commercial
Slide 28: 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 29: 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 • AGAMREE ® opportunity for change Issues with current steroid use • Replacing current corticosteroid treatment initiation Too late Too little Too soon • S
Slide 30: Vamorolone in Becker muscular dystrophy
Vamorolone in Becker muscular dystrophy 30
Slide 31: 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 32: Vamorolone holds promise in BMD based on data generated for DMD
Vamorolone holds promise in BMD based on data generated for DMD Vamorolone designated orphan drug status by FDA in January 2024 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 w
Slide 33: Half-year 2024 financial results and update (Sept 12, 2024)
Half-year 2024 financial results and update (Sept 12, 2024) 33 Corporate Presentation Oct, 2024 https://www.santhera.com/assets/files/financial_reports/2023_SANN_AnnualReport2023_final.pdf
Slide 34: Santhera financial status
Santhera financial status Santhera Pharmaceuticals is listed on the Swiss Stock Exchange SIX: Ticker SANN • Cash runway • Recent milestones AGAMREE® for DMD • August 2024: CHF 52 million post financing and • 07-2023: North American licensing to Catalyst settlement of convertible bonds • 01-2024: Lau
Slide 35: Santhera Pharmaceuticals
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases October 1, 2024
Santhera Pharmaceuticals — Investor Presentation (October 2024) on 6ix