Herantis Pharma Plc · OMXHEX:HRTIS
Herantis Pharma — Investor Presentation (November 2021)
30 slides from Herantis Pharma's investor presentation, November 2021
Slide 1: Corporate Update November 2021
Corporate Update November 2021 Our mission is to break boundaries of standard therapeutic approaches. Our drug candidates aim to revolutionize the treatment of Parkinson’s Disease and other Neurodegenerative diseases. 1
Slide 2: Disclaimer
Disclaimer By accepting to receive this presentation from Herantis Pharma Plc (the "Company"), you agree to be bound by the following conditions. This presentation and any oral communication in connection with it (the "Information") are strictly confidential and are being provided to you solely for
Slide 3: Herantis Focused On Parkinsons And Other Neurodegenerative Diseases
Herantis Focused On Parkinsons And Other Neurodegenerative Diseases • Headquartered Helsinki, listed Finland & Sweden - IPO 2014 (Finland) 2019 (Sweden), €67m to date • Two programs: i. rhCDNF a ground-breaking biological therapy. rh Already used safely in human Phase I study via surgical intracrani
Slide 4: Parkinson's Disease – Urgent Need For Disease Modifying Treatments
Parkinson’s Disease – Urgent Need For Disease Modifying Treatments • Exploding incidence of Parkinson’s worldwide … of ‘pandemic proportions’ - combination of man-made and genetic factors • Massive burden of disease for the world - > 10 million patients worldwide, costs €14bn annually EU alone, hous
Slide 5: More Than A Decade Of Innovation Coming to Fruition
More Than A Decade Of Innovation Coming to Fruition 2011 – 2017 2017 2020 2020 2020 – 21 • rhCDNF • Phase I safety • Alternative • Phase I study • Novel biomarker manufacturing study starts routes of successfully data generated rhCDNF and preclinical administration completed toxicology explored 2005
Slide 6: rhCDNF and HER-096 Targets Core Pathology Of Parkinson's – Proteostasis & UPR
rhCDNF and HER-096 Targets Core Pathology Of Parkinson’s – Proteostasis & Unfolded Protein Response (UPR) • Proteostasis regulates proteins in body and influences the fate of every protein from synthesis to degradation eg 𝜶𝜶-synuclein • Its failure is implicated with the development of…
Slide 7: Biomarker Focus
Biomarker Focus
Slide 8: CNS Drug Development is Shifting to Biomarker-driven Development Strategies
CNS Drug Development is Shifting to Biomarker-driven Development Strategies • Diseases take a long time to develop, so without biomarkers it can take years to see clinical results - Biomarkers provide a window into onset of disease, its progression, and response to therapy - Enables more rapid & eff
Slide 9: Why Biomarker Development has Become so Important in Drug Development?
Why Biomarker Development has Become so Important in Drug Development? • Mechanism-based, targeted therapies have successfully paved the way for precision medicine in oncology • All therapeutic areas are transitioning to and leading pharma companies have already adopted biomarker-driven clinical tri
Slide 10: Incorporation of Biomarkers to the Drug Development Process is Critical for Success
Incorporation of Biomarkers to the Drug Development Process is Critical for Success NO BIOMARKER STRATEGY BIOMARKER-LED Biomarkers not co-developed with the Biomarker assays developed to therapeutic characterize disease models and monitor response to therapy Traditional patient selection methods Str
Slide 11: Parkinson's Disease is a Heterogenous Disease with Multiple Etiological Subtypes
Parkinson’s Disease is a Heterogenous Disease with Multiple Etiological Subtypes Types of biomarkers used in clinical development of disease-modifying therapies • Biomarker-driven phenotyping for targeted clinical trials • Pharmacodynamic biomarkers for measuring treatment response • Prognostic biom
Slide 12: Three Biomarker Modalities Explored in CDNF Phase 1 Study
Three Biomarker Modalities Explored in CDNF Phase 1 Study Brain imaging biomarker: Digital biomarker: Fluid-based biomarkers: Dopamine transporter Parkinson’s CSF biomarker PET imaging KinetiGraph (PKG) discovery study Improvement 12 © 2014-2021 HERANTIS PHARMA Plc. All rights reserved. www.herantis
Slide 13: Biomarker Fingerprint in PD Patients Receiving rhCDNF
Biomarker Discovery Data: Targeted Cerebrospinal Proteomics Study Revealed a Biomarker Fingerprint in PD Patients Receiving rhCDNF IMMUNE RESPONSE OXIDATIVE STRESS LYSOSOMES, AUTOPHAGY & TRAFFICKING MITOCHONDRIA NEURONAL ADHESION, SYNAPSES & DEGENERATON AMYLOID PRECURSORS LIPOPROTEINS EXOSOMES IRON
Slide 14: An Example of a Biological Responder
An Example of a Biological Responder: Clinical Improvement Associated with a Change in CSF Biomarker Profile after Switching from Placebo to rhCDNF • 62-year old male • Disease duration: 10 years (from first motor symptoms) 3 • 6 months placebo, followed by 6 months CDNF CSF Proteomic profile 4 1 Ge
Slide 15: A Biomarker Signature: The Twenty CSF Proteins
A Biomarker Signature: The Twenty CSF Proteins Showing Similar Pattern of Change in the Biological Responders Have Common Functional Links Functional association: Gene Ontology (GO) term enrichment analysis of the 20 CSF proteins Markers that changed at least 2-fold vs baseline in at least three pat
Slide 16: Expert Scientific Advisory Board To Work With Herantis On Biomarker-Driven Development
Expert Scientific Advisory Board To Work With Herantis On Biomarker-Driven Development • Anders Gersel Pedersen, M.D., Ph.D. • Daniele Bravi, M.D., Ph.D. • Chairman of the Board • Specialist Neurologist in movement • Globally renowned and respected expert in disorders CNS drug development. Previousl
Slide 17: Update on rhCDNF and HER-096 Programs
Update on rhCDNF and HER-096 Programs
Slide 18: CDNF Is Essential For Enteric Dopamine Neuron Development And Survival
CDNF Is Essential For Enteric Dopamine Neuron Development And Survival • Dopaminergic neurons occur in highest concentration in brain and gut areas of body ○ Role of CDNF can be observed in both brain and gut dopamine neurons • CDNF deficient mice show degeneration of gut dopamine neurons ○ Results
Slide 19: Subcutaneously Administered HER-096 Effectively Penetrates the Blood-Brain Barrier
Subcutaneously Administered HER-096 Effectively Penetrates the Blood-Brain Barrier with Therapeutic Concentrations and Extended Half-life in the Brain DUAL (BRAIN AND PLASMA) MICRODIALYSIS STUDY IN MICE HER-096 (ng/ml) Kp,uu,brain = 0.216 Brain ISF T1/2 = 1.3 h STUDY: Mice with microdialysis probes
Slide 20: xCDNF (HER-096) Protection Of Dopamine Neurons, Reduces α-Synuclein Aggregates
xCDNF (HER-096) Impressive Protection Of Dopamine Neurons + Significantly Reduces 𝜶𝜶-Synuclein Aggregates In Vivo High protection of dopamine neurons, plus almost complete eradication of 𝛂𝛂-synuclein aggregates Improvement Improvement +11% +11% +46% aSyn injection + CBE start Analysis 0 1 2 3 4…
Slide 21: xCDNF (HER-096) Potent Impact On Key Pathologies Of Parkinsons
xCDNF (HER-096) Potent Impact On Key Pathologies Of Parkinsons Attenuates Unfolded Protein Response And ER Stress, Thus Reduces Cell Death >80% reduction of key neuroinflammatory/microgliosis marker, and 85% and 52% reductions of activated IRE1 and ATF6 -70% -84% -15% -85% -24% -52% STUDY: Test comp
Slide 22: xCDNF (HER-096) Convincing Protection Of Dopamine Neurons In Therapeutic Dosing Regimen
xCDNF (HER-096) Convincing Protection Of Dopamine Neurons, Reduction Of 𝜶𝜶- Synuclein And Neuroinflammation In Therapeutic Dosing Regimen aSyn injection HER-096 + CBE start start Analysis 0 1 2 3 4 5 6 weeks Dopamine neurons number in SN Alpha-synuclein deposition Neuroinflammation
Slide 23: HER-096 Has Superior Pharmacological Properties When Compared to Other UPR Modulating Compounds
HER-096 Has Superior Pharmacological Properties When Compared to Several Other UPR Modulating Compounds DOPAMINE NEURON SURVIVAL ALPHA-SYNUCLEIN AGGREGATION Sunitinib is an FDA-approved multi-target kinase inhibitor that modulates IRE1α • HER-096 has superior potency and significantly wider kinase a
Slide 24: Uptake of HER-096 by Multiple Cell Types of the Brain Following Subcutaneous Injection
Uptake of HER-096 by Multiple Cell Types of the Brain Following Subcutaneous Injection Vehicle-injected HER-096-injected 8000 areaCP-C-096-Atto647N of HER-096 signal * 6000 of vehicle) vehicle) 4000 (% of (% Totalarea 2000 Total HER-096-injected 0 ) at g; c. s. tr g/k n) e; io m in 0 al dm (3 is (s
Slide 25: Planned Development Timelines for HER-096 and rhCDNF
Planned Development Timelines for HER-096 and rhCDNF HER-096: Phase 1 HER-096: Phase 1b HER-096: Preclinical & tox in HV in early PD patients Observational biomarker study in early PD patients (no dosing) Non-invasive rhCDNF: Non-invasive rhCDNF: Non-invasive rhCDNF: Intranasal formulation & device
Slide 26: Enormous Commercial Potential For rhCDNF &/Or HER-096 In Parkinsons
Enormous Commercial Potential For rhCDNF &/Or HER-096 In Parkinsons • Disease modifying treatment rhCDNF/HER-096 for Parkinson’s disease could reach more than €8 bn in total annual revenue • Herantis business model is focused on partnering with large pharma; we continue to dialogue with potential pa
Slide 27: Desperate & Amplifying Need For Disease Modifying Therapies
Desperate & Amplifying Need For Disease Modifying Therapies (DMT’s) Like rhCDNF and xCDNF • Successful innovation urgently needed to address Parkinson’s pandemic • Other drug classes eg mAbs hold/held a lot of promise for neurodegenerative diseases, but facing challenges • target α-synuclein protein
Slide 28: Timelines & Some Of The Key Planned Milestones
Timelines & Some Of The Key Planned Milestones Commercialization Approval Parkinson's Disease and other Intranasal administration 2H 2021 rhCDNF: neurodegenerative diseases formulation milestones Phase III (Safety & Efficacy) HER-096: Pre-clinical efficacy and 2H 2021 Parkinson's Disease Phase II (
Slide 29: Summary
Summary • Unparalleled biomarker data, and clear biomarker program ahead - Strong impact on markers of disease; clear correlation with clinical & imaging improvements • Potent neuroprotection - Restores proteostasis, needed for neuronal survival, convincingly crosses BBB • Significant market opportu
Slide 30: Thank You!
Corporate Presentation Marking A Decade of Successful Research on CDNF Thank You!
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