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BioArctic AB Sachs Associates 11th Annual European Life Sciences - PowerPoint PPT Presentation

BioArctic AB Sachs Associates 11th Annual European Life Sciences CEO Forum Gunilla Osswald, PhD, CEO February 26, 2018 Disclaimer This presentation has been prepared and produced by BioArctic AB (publ) (BioArctic) solely for the


  1. BioArctic AB Sachs Associates 11th Annual European Life Sciences CEO Forum Gunilla Osswald, PhD, CEO February 26, 2018

  2. Disclaimer • This presentation has been prepared and produced by BioArctic AB (publ) (“BioArctic”) solely for the benefit of investment analysis of BioArctic and may not be used for any other purpose. Unless otherwise stated, BioArctic is the source for all data contained in this presentation. Such data is provided as at the date of this presentation and is subject to change without notice. • This presentation includes forward-looking statements. These forward-looking statements involve known and unknown risks, uncertainties and other factors, which may cause BioArctic’s actual results, performance, achievements or industry results to be materially different from those expressed or implied by these forward-looking statements. Forward-looking statements speak only as of the date of this presentation and BioArctic expressly disclaims any obligation or undertaking to release any update of, or revisions to, any forward- looking statement in this presentation, as a result of any change in BioArctic’s expectations or an y change in events, conditions or circumstances on which these forward-looking statements are based. • This presentation does not constitute or form part of, and should not be construed as, an offer or invitation for the sale of or the subscription of, or a solicitation of any offer to buy or subscribe for, any securities, nor shall it or any part of it or the fact of its distribution form, or be relied on in connection with, any offer, contract, commitment or investment decision relating thereto, nor does it constitute a recommendation regarding the securities of BioArctic. • The information in this presentation has not been independently verified. • No regulatory body in Sweden or elsewhere has examined, approved or registered this presentation. 2 | BioArctic AB, 2018

  3. Snapshot of BioArctic Company overview Investment highlights  Research oriented biopharma company  Highly educated organization with proven track focusing on development of drugs in areas with record of bringing drugs from idea to market a large unmet medical need, such as  Innovative portfolio of differentiated first- Alzheimer’s and Parkinson’s Disease, and generation disease modifying agents in Complete Spinal Cord Injury Alzheimer’s and Parkinson’s Disease, diagnostics  Founded in 2003 by Prof. Lars Lannfelt and and pioneering Complete Spinal Cord Injury Dr. Pär Gellerfors treatment  Flexible organization with approx. 30 FTEs  Strategic collaborations with Eisai and AbbVie complemented with consultants and close validating highly innovative research collaborations with external partners organization and unique product candidates  Headquartered in Stockholm, Sweden  Attractive combination of fully financed partner projects and cutting-edge, well funded,  Listed on Nasdaq Stockholm Mid Cap proprietary R&D pipeline with substantial since October 2017 market and out-licensing potential 3 | BioArctic AB, 2018

  4. Long-standing and Extensive Partnerships Eisai collaboration and license agreements AbbVie collaboration agreement Description of agreements Milestone / royalty potential Description of agreements Milestone / royalty potential • Two previous research • Research collaboration • Total potential value of the • The total aggregated value collaborations regarding (entered Sep 2016) agreement is up to USD of the research disease modifying therapies regarding alpha-synuclein 755m incl. an up-front fee, collaborations and license for Alzheimer’s Disease that antibodies as disease option exercise fee, and agreements is approx. EUR resulted in two licenses of modifying therapies for success-based milestones 218m in signing fee and the A β oligomer/protofibril Parkinson’s Disease incl. plus tiered royalties milestones plus high single antibodies BAN2401 and BAN0805 to IND, follow-up digit royalties • BioArctic has received an BAN2401 Back-up compounds and diagnostic • BioArctic has received USD 80m up-front payment • Third research collaboration • BioArctic primarily for the research approx. EUR 47m for the ongoing regarding a new responsible for performing collaboration research collaborations, target as a disease all pre-clinical activities signing fees and milestones modifying therapy for • Option for AbbVie for Alzheimer’s Disease a license to develop and commercialize the antibodies Strategic collaborations with pharmaceutical industry validating potential value and commercialization potential for BioArctic with proven track record of delivering on research collaborations 4 | BioArctic AB, 2018

  5. Strategic Partnerships and Cutting-Edge Proprietary R&D PRODUCT CANDIDATE INDICATION PARTNER DISCOVERY PRE-CLINICAL PHASE 1 PHASE 2 PHASE 3 BAN2401 1) Alzheimer’s Disease (anti-A β antibody) BAN2401 Down’s Syndrome 2) (anti-A β antibody) Traumatic Brain Injury NEURODEGENERATIVE DISEASES BAN2401 Back-up Alzheimer’s Disease (anti-A β antibody) AE1501 Alzheimer’s Disease (undisclosed) AD15 1502 02 Alzheimer’s Disease (undisclosed) AD1503 Alzheimer’s Disease (undisclosed) BAN0805 Parkinson’s Disease (anti-alpha-synuclein antibody) Imaging and biochemical Alzheimer’s Disease DIAGNOSTICS & biomarkers (A β ) TECHNOLOGY Imaging and biochemical Parkinson’s Disease biomarkers (alpha-synuclein) BBB-technology Multiple application areas (blood-brain barrier) SC0806 SPINE Complete Spinal Cord Injury (FGF1/device) 1) Partner with Eisai on BAN2401 for treatment of AD. Since 2014, Eisai partnered with Biogen in AD. 2) Dementia and cognitive impairment associated with Down’s syndrome. Source: Company data 5

  6. Disease Modifying Agents and Reliable Diagnostics/Biomarkers for Neurodegenerative Diseases New therapy focus on disease pathogenesis – efforts to delay the neurodegenerative process Neurodegenerative disease therapy TODAY Neurodegenerative disease therapy TOMORROW ILLUSTRATIVE ILLUSTRATIVE Biomarkers used as diagnostic tools to identify and to monitor the therapeutic effect of disease modifying treatments Clinical diagnosis DISEASE PROGRESSION DISEASE PROGRESSION Agents that modify disease pathology Symptomatic (thereby slowing or delaying treatment the onset and disease progression) AGE AGE Significant unmet medical need to be addressed by disease modifying agents and reliable diagnostics/biomarkers 6

  7. Protein Misfolding is Disease Causing in a Number of Neurodegenerative Diseases Including AD and PD Monomers Oligomers/Protofibrils Mature fibrils Plaque/Lewy bodies Oligomer/ Protofibril selective antibodies NEUROTOXIC FORMATIONS SOLUBLE FORMS INSOLUBLE FORMS A β P l a q u e i n A D A l p h a - s y n u c l e i n L e w y b o d i e s i n P D Source: Company information. 7 | BioArctic AB, 2018

  8. BAN2401 – Learnings from Previous Clinical Trials in AD Incorporated in Phase 2b Study Design Last interim analyses performed and final results in H2 2018 Important parameters Right patient Right dose & Right Right target Right safety population exposure measurements • Address the soluble protofibrils – • Early AD – MCI due to AD & Mild • Selecting doses with exposures • More sensitive cognition scales • Well tolerated with a benign a toxic form of amyloid AD above preclinical IC50 safety profile • Biomarkers for disease • Identify right patients – • Adaptive design testing several • Low cardiovascular risks and progression and disease biomarkers doses and dose regimens modification amyloid related imaging abnormalities (ARIA) etc. Phase 2b study design Patient inclusion Treatment 12 months Treatment 18 months Multinational Placebo Primary Secondary endpoints: recruitment: endpoints: • ∆ from baseline in ADCOMS • 100 clinical centers 2.5 mg/kg bi-weekly • ∆ from baseline at 18 months included Double-blind, placebo in ADCOMS at Inclusion • ∆ from baseline in brain • Inclusion criteria: controlled, parallel- 5 mg/kg bi-weekly 12 months completed with amyloid as measured by MMSE >22-30   group study with • Safety and amyloid PET at 12 and 18 856 • Stable concomitant Bayesian adaptive 10 mg/kg bi-weekly tolerability months  patients  medication design • ∆ from baseline in total • Positive amyloid 5 mg/kg once every four weeks hippocampal volume at 6, 12 PET/CSF and 18 months 10 mg/kg once every four weeks Full read-out of study after 18 months treatment in H2 2018 Source: Company information. Note: ADCOMS = Alzheimer’s Disease Composite Score, a evaluation tool developed by Eisai. 8 | BioArctic AB, 2018

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