Santhera Pharmaceuticals Holding AG · SIX:SANN
Santhera Pharmaceuticals — Investor Presentation (September 2023)
23 slides from Santhera Pharmaceuticals's investor presentation, Sep 15, 2023
Slide 1: Santhera Pharmaceuticals
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases Presentation by Dario Eklund, CEO Sep 2023 Version S
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: Presentation Overview
Presentation Overview (1) Santhera in a snapshot (2) Duchenne muscular dystrophy (DMD) and the need for a better foundational therapy (3) Vamorolone and commercial strategy Headquarters near Basel, Switzerland (4) Financial status 3 Investor Presentation Sep 2023
Slide 4: Commercial stage company in attractive DMD market
Commercial stage company in attractive DMD market • SIX Swiss Exchange listed company (SANN) • Lead asset vamorolone in DMD (and beyond) • Safer alternative to corticosteroids • Regulatory decisions in Q4-2023 (US, EU, UK) ▪ Recent Catalyst US deal valued at up to USD 231 million plus royalties • Ca
Slide 5: Duchenne muscular dystrophy (DMD)
Duchenne muscular dystrophy (DMD) • Genetic disease causing progressive muscle weakness • Loss of ambulation in early teenage years • Respiratory failure and cardiac complications • Life expectancy in the late twenties • 30 – 35,000 patients in US and EU, combined • Corticosteroids standard of care
Slide 6: Vamorolone can fill the need for a better foundational therapy in DMD
Vamorolone can fill the need for a better foundational therapy in DMD ESTABLISHED SAFETY ISSUES VAMOROLONE EFFICACY OF WITH STEROIDS OFFERING STEROIDS ESTABLISHED TOO LATE NEW FOUNDATIONAL TOO LITTLE DISSOCIATIVE THERAPY TOO SHORT STEROID CLASS 6 Investor Presentation Sep 2023
Slide 7: Vamorolone dissociative properties
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 cortic
Slide 8: Primary endpoint was met in pivotal clinical trial (VISION-DMD)
Primary endpoint was met in pivotal clinical trial (VISION-DMD) Rise time (sec)1,2 BL w 24 % Change VAM 6 mg/kg/d 6.0 4.6 - 23% Placebo 5.4 5.5 + 2% TTSTAND velocity (rises/sec)1 0.08 Change from Baseline (SEM) 0.06 0.04 0.02 p=0.002 0.00 0 6 12 24 -0.02 Weeks -0.04 Placebo (N=28) VAM 6 mg/kg/d (N=2
Slide 9: Vamorolone has no negative impact on bone biomarkers
Vamorolone has no negative impact on bone biomarkers Example shown for biomarker of bone formation (VISION-DMD at 24 weeks) P1NP (ng/ml) 100 Mean (SEM) Change from Baseline 50 0 -50 -100 -150 -200 Data on File : VAM-2021-007, PDN, prednisone; SEM, standard error of mean; VAM, vamorolone. 9 Investor
Slide 10: Vamorolone leads to biomarker recovery after switch from prednisone
Vamorolone leads to biomarker recovery after switch from prednisone Example shown for biomarker of bone formation (VISION-DMD) P1NP (ng/ml) 150 Mean estimated change from Baseline 100 50 0 0 24 48 -50 -100 -150 -200 Data from VISION-DMD Pivotal Study on File 2022, PDN, prednisone; VAM, vamorolone. 1
Slide 11: Switching from prednisone to vamorolone recovers normal growth
Switching from prednisone to vamorolone recovers normal growth Growth trajectory (VISION-DMD) Change in Height z-score1 0.3 VAM 2 + 6 mg/kg/d (N=56) 0.2 Change from Baseline (SEM) 0.1 0 0 24 48 -0.1 PDN - VAM 2 + 6 mg/kg/d (N=30) -0.2 Data from VISION-DMD Pivotal Study: Safety Population 2 (SAF-2);
Slide 12: Vamorolone unlike other steroids preserves normal, long-term growth
Vamorolone unlike other steroids preserves normal, long-term growth Growth trajectory VISION-DMD vs FOR-DMD (natural history) Change from baseline in Height (cm) 8 Vamo 2mg (N=28) Mean height change from baseline (cm) 7 Vamo 6mg (N=28) Prednisone 0.75mg (N=50) 6 Deflazacort 0.9mg (N=56) Normal boys
Slide 13: Vamorolone leads to fewer and less severe spinal fractures
Vamorolone leads to fewer and less severe spinal fractures 35 Vamorolone long-term extension study vs FOR-DMD at 30 months1 30 25 Reduction of number of patients with 20 vertebral fractures by approx. 50% 15 10 5 0 % pts with vertebral fractures Number of vertebral fractures Total Spinal Deformity I
Slide 14: Summary of value proposition for vamorolone in DMD
Summary of value proposition for vamorolone in DMD Vamorolone • Durable efficacy comparable to standard of care • Preserved bone health (unlike deleterious effect of standard steroids) • Improved safety profile compared to prednisone evident at 24 weeks • Long-term treatment profile being further ex
Slide 15: Potential approvals in Q4-2023 allow Q1-2024 launches in US and EU
Potential approvals in Q4-2023 allow Q1-2024 launches in US and EU 2022 2023 2024 2025 Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 H1 Potential FDA Approval Launch 2 Filing Oct 26th 1 EMA Potential Launch 3 Filing Approval MHRA Potential Filing Launch 4 Approval Potential Filing Orphan drug exclusivity in US (7 y
Slide 16: Santhera holds global rights to vamorolone in all indications
Santhera holds global rights to vamorolone in all indications • North America • Partnership with Catalyst (NA) • China • Partnership with Sperogenix (CN) • Europe • Own commercialization (next slide) • Rest of world • Further out-licensing opportunities 16 Investor Presentation Sep 2023
Slide 17: Catalyst is our commercial partner of choice for North America
Catalyst is our commercial partner of choice for North America • Founded 2002, IPO 2006 • Market capitalization USD 1.4 billion as of June 22, 2023 (CPRX, Nasdaq) • Proven commercial capabilities: targeted total revenues 2023 of USD 375 – 385 million – Two marketed drugs, Firdapse (amifampridine) an
Slide 18: Catalyst Pharmaceuticals: Transformative deal valued up to USD 231 million + royalties
Catalyst Pharmaceuticals: Transformative deal valued up to USD 231 million + royalties • Upfront of USD 75 million represents more than Santhera’s market capitalization pre-signing – Provides funding for EU launch and for healthy balance sheet through repayment of short-term debt – Additional equity
Slide 19: Santhera commercial launch in key European geographies
Santhera commercial launch in key European geographies • Early access programs starting in Q4-2023 in FR and UK • First launch in Germany in early Q1-2024 2 1 • Commercialization in core Western markets by Santhera 3 • Lean commercial organization with up to 60 incremental FTEs 4 • Peak sales of EUR
Slide 20: Vamorolone – a pipeline in a product with multi-indication potential
Vamorolone – a pipeline in a product with multi-indication potential Therapeutic Area Indication • Focus on patient population benefiting Neurology Becker Muscular Dystrophy* from a prolonged and safer steroid Myasthenia Gravis Ocular* treatment Chronic Inflammatory Demyelinating Polyneuropathy Pulm
Slide 21: 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 transaction • Net (loss) for the period (23.3) • 07-2023: Vamorolone US licensing to Catalyst • Cash (used) in operations (15.5) Raxon
Slide 22: Santhera is well positioned for future growth
Santhera is well positioned for future growth Future potential for vamorolone as pipeline within a product not factored in current valuation • Healthy balance sheet with low debt and sufficient cash for own commercialization in core EU markets • Non-dilutive income via own EU sales, licensing income
Slide 23: Santhera Pharmaceuticals
Santhera Pharmaceuticals Developing medicines to meet the needs of patients living with rare diseases Sep 2023 Version S
Santhera Pharmaceuticals — Investor Presentation (September 2023) on 6ix