Amorfix Life Sciences Leaders in Misfolded Protein Diseases Q4 2014 1
Amorfix Life Sciences Leaders in Misfolded Protein Diseases Q4 2014 1 Amorfix Life Sciences Founded 2004 by Dr. Neil Cashman 2005 Public listing on TSX (AMF) Corporate Headquarters Mississauga, Ontario CANADA Relocating to South San Francisco Q1 2015 Focus on Misfolded Protein Diseases Neurodegenerative diseases and cancer Proprietary platform technologies for development of targeted therapeutics and companion diagnostics Epitope Protection ProMIS Discovery Technology 2 Management and Board of Directors Management Team Board of Directors Robert Gundel, PhD, MBA • President and CEO William Wyman, MBA • Chairman of the Board • 30 years of Pharmaceutical industry experience • Booz Allen, Oliver Wyman Neil Cashman, MD • Founder and CSO William Copeland, MBA • Director • World renowned neurologist • Leader in misfolded protein diseases • Vinik Asset Management Warren Whitehead, MBA • CFO • 30 years of Pharmaceutical industry experience Johannes Roth, MA • Director • Five T Capital Stuart Hwang, PhD Elliot Goldstein, MD • Director • VP of Business Development • 25 years of Pharmaceutical industry experience • Immune Pharmaceuticals, Virtua, British Biotech, SmithKline 3 Amorfix Platform Technologies Amorfix Technology Disruptive Idea 1: Protein Misfolding and Disease Cancer Cancer Neurodegeneration Neurodegeneration 5 Disruptive Idea 2: Antibodies Can Selectively Target Misfolded Proteins while Sparing Native Isoforms Efficacy: Specific targeting of a pathogenic species Safety: Selective sparing of normal proteins – – – – – Preservation of normal function – Minimization of autoimmunity – Minimal regimens in therapeutic vaccines Neutralization of toxicity Blockade of propagation Acceleration of degradation Minimal “target distraction” 6 Amorfix: The Leader in Misfolded Protein Diseases Leveraging Platform Technology and Intellectual Property Licensing Deals with Upfront Payments, Milestones and Royalties CJD Chronic Wasting Disease Misfolded Protein Diseases Prion Disease Cancer ProMIS Discovery Epitope Protection Neurodegenerative Ovarian Lung Prostate Melanoma Lymphoma Leukemia Core technology can be used across multiple disease areas: - therapeutic antibodies - companion diagnostics - vaccines ALS Alzheimer’s disease Parkinson’s disease 7 ProMIS™ Algorithm: Rational Selection of DSEs ProMis™ Discovery – An Enabling Technology • A computational algorithm that predicts where proteins will misfold or partially unfold • Used to identify specific areas of misfolded proteins used to generate novel targeted therapeutics INPUT Calculate free energy of unfolding ΔGij for region starting at residue i with length j Loop over i = 125 to 228 - j OUTPUT Loop over j = 1 to 25 DSE Candidates Targeted Therapeutics High resolution structure of the natively folded protein OUTPUT: Unfolding Energy Landscape = Disease Specific Epitopes CANDIDATES 8 Amorfix Technology: Beauty in Simplicity Therapeutic Antibodies 1. ProMIS™ Discovery Technology Identifies unique areas on misfolded protein targets 2. These misfolded proteins are only expressed on the surface of tumor cells, not normal cells 3. Antibodies generated against these unique areas don’t ‘recognize’ the normal cell surface receptor and only bind to tumors. Normal Cell Surface Receptor Misfolded Cell Surface Receptor Antibodies Multiple ways to kill tumors with antibodies - Antibody alone - Antibody recruits immune system - Antibody drug conjugate Normal Cell Cancer Cell 9 Proof of Concept of Disruptive Ideas: Therapeutic Benefit of Targeting Misfolded Epitopes ALS Treatment with antibodies directed against misfolded SOD1 increases survival time. Vaccine treatment produces the same therapeutic results. Inhibition of misfolding propagation Ovarian Cancer Treatment with antibodies directed against misfolded PrP specifically targets tumors, not normal tissue, and significantly reduces tumor growth % increase in tumor volume (compared to day 0) SKOV-3 450 400 350 300 250 200 150 100 50 0 Vehicle Taxotere AMF-1c-120 0 5 10 15 20 Day After Treatment Initiation 25 Options for therapeutic antibodies: - Antibody alone; recruiting the body’s own immune defense systems - Antibody drug conjugates; precise delivery of toxins directly to the tumor 10 ProMIS™ Discovery Partnering Activity • Currently in discussions with eight big pharma organizations about platform technology deals and Research Agreements. • Amorfix provides partner with patentable disease specific epitopes (targets) for the generation of therapeutic antibodies • Deals include upfront cash payments, development milestone payments and royalties on sales • We expect to complete the first deal end of 2014/early 2015 11 Amorfix Epitope Protection™ Technology Finding the “Needle in the Haystack” Misfolded proteins are present in a million fold excess of normally folded proteins Technical hurdle: how to identify and measure these small amounts of misfolded proteins Amorfix Epitope Protection and AMFIA technologies solve this problem. Epitope Protection™ Technology NO2 Normal Monomers NO2 Chemical Modifier Disease Oligomers Aggregates NO2 NO2 NO2 NO2 NO2 NO2 NO2 NO2 NO2 NO2 Disaggregation NO2NO2 NO2 = epitope available for chemical modification 12 Alzheimer’s Disease Diagnostic Identification of Early-Stage Disease Alzheimer’s disease diagnostic technology: - Measures levels of aggregated A oligomers in CSF - A oligomers are a “hallmark” of Alzheimer’s disease Diagnostic sensitivity for early-stage disease = 90% Highest sensitivity in the industry Diagnostic sensitivity for AD individuals = 97% Separate Detection of A 40 and 42 No other company in the world has this capability. Enables research into Abeta oligomer’s role in early Alzheimer’s disease. Can be used as a biomarker to ‘monitor’ target engagement, drug mechanism of action and efficacy over time. Additional secondary endpoint in clinical trials to support observations of cognitive function. 128 Signal / Unspiked CSF Original A40/42 New A40 New A42 64 32 16 8 4 2 unspiked CSF 1 0.5 10 2 10 3 10 4 10 5 10 6 Dilution of 10% AD Brain Homogenate 13 AD Diagnostic Partnering Option 1 Exclusive worldwide license to develop and commercialize • • • Upfront payment Milestone payments Royalties on sales Non-exclusive license for use of technology Option 2 • • • Option 3 Amorfix commercializes the test • • • Upfront technology access fee Tech Transfer based milestone payments Yearly maintenance payments Go with CLIA approved lab or CRO Clinical validation of the assay Service-based pricing In Discussions with Four Big Pharma Companies for Licensing the AD Diagnostic Technology Plan to complete the first deal in Q4 2014 14 One, Three, Ten Year Plan Evolving The Business Over Time Fully Integrated Platform Technology Business Year 10 - Cutting edge technology across a wide variety of businesses - Large number of collaborations using our technology platforms to develop products - Continue to improve and understand misfolded proteins as a means to expand the therapeutic arena Partially Integrated Drug Development Business Year 3 - Develop strength in Alzheimer’s, ALS and certain cancers Increase the number of partners using our technology platforms Expand capabilities and technology toolbox (e.g. protein crystallography) Begin to expand beyond neurodegenerative disease and cancer Revenue generated from partnered programs Obtain US Listing Building The Platform Technology Based Business Year 1 - ProMIS Discovery technology Epitope Protection Move operations to San Francisco Bay Area Improve visiblilty to investors and partners Obtain funding to achieve three year plan 15 Current Partnerships May 2010 High Affinity Rabbit Antibodies. Amorfix pays technology access upfront, milestone payments and royalties. November 2011 Research Agreement (50/50 cost/profit split). Urease-antibody drug conjugate for Ovarian cancer July 2012 Exclusive license for Preclinical AD diagnostic. They do all sales, marketing and perform the assay. Amorfix gets double-digit % of sales. November 2012 Multiple cancer antibody targets. Modest technology access fee paid. April 2013 Research agreement. Lung cancer targets. No money exchanged. Kyowa Hakko Kirin January 2014 Non-exclusive license to AD CSF assay. Funded R&D for assay optimization and validation. . 16 Short-Term Priority List Bring in non-dilutive revenue • Close at least one ProMIS discovery deal • License AD diagnostic • Complete other partnering deals Get the message out/Rebranding the company • Publish data in top tier scientific journal • Hire IR/PR firm • Present at high visibility conferences Stops the need for continued PP’s Validates our technology Sets stage for more deals Gets the company on radar screen Increases share price Sets stage to bring in more cash Secure additional financing • Angel investors • VC firms • Public offering Stabilizes the company Enables further growth of the business Generates further value 17 Financing Strategy/Options • Generate non-dilutive revenue through partnering deals for ProMIS Discovery technology and the AD assay. • Venture Capital funding, Angel Investors • Financing Goal: $10M 18
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