Global Leader in Bacteriophage Development to Combat Infectious Diseases

Transcription

1 Global Leader in Bacteriophage Development to Combat Infectious Diseases September 13, 2016

2 Safe Harbor Statement This presentation contains "forward-looking" statements that involve risks, uncertainties and assumptions. If the risks or uncertainties materialize or the assumptions prove incorrect, our results may differ materially from those expressed or implied by such forward-looking statements. All statements other than statements of historical fact could be deemed forward-looking, including, but not limited to: the potential future of antibiotic resistance; the expected timing of patient dosing; the expected timing of collecting and reporting data; the expected timing of additional clinical trials, including Phase II clinical trials; the drug product candidates to be supplied by AmpliPhi for clinical trials; the activities to be performed by specific parties in connection with clinical trials; the potential use of bacteriophages to treat bacterial infections; timing for manufacturing scale-up; research and development plans; the development of bacteriophage-based therapies; the ability to select combinations of phages to formulate product candidates; the ability to manufacture product candidates; the safety and efficacy of product candidates; collaborations with third parties and the potential markets for product candidates; potential market growth; and any statements of assumptions underlying any of the items mentioned. These statements are based on estimates and information available to us at the time of this presentation and are not guarantees of future performance. Actual results could differ materially from our current expectations. You should not rely upon forward-looking statements as predictions of future events. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, we undertake no obligation to update publicly any forward-looking statements for any reason to conform these statements to actual results or to changes in our expectations. We refer you to the documents that we file from time to time with the Securities and Exchange Commission (the SEC ), specifically our Annual Report on Form 10-K, our Quarterly Reports on Form 10-Q and our Current Reports on Form 8-K. These documents, including the sections therein entitled Risk Factors, identify important factors that could cause the actual results to differ materially from those contained in forward-looking statements. We have filed a registration statement (including a preliminary prospectus) with the SEC for the offering to which this presentation relates. Before you invest, you should read the prospectus in that registration statement and other documents we have filed with the SEC for more complete information about our company and the offering. This presentation shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or other jurisdiction. 2

3 What happens when antibiotics stop working? 3

4 The Looming Antibiotic Resistant Era 10,000,000 annual deaths Year 2050 $100 trillion cumulative cost 3.5% global GDP reduction If we fail to act, this could become our reality 4 Source: O Neill Report

5 5 The World Awaits a Response

6 AmpliPhi Biosciences Our mission is to combat the rising tide of antibiotic-resistant infections Adaptable Bacteriophage Platform Technology Radically different than traditional antibiotics Potential to selectively target any species of bacteria Clinical Stage Results from two Phase I clinical trials expected in H First phage therapy Phase II trial planned for 2017 Manufacturing Know-How In-house, dedicated cgmp phage manufacturing facility Proprietary processes are a significant barrier to entry Proven Track Record Established leadership in bringing early stage products to market Efficient use of funds to reach clinical stage 6

7 Why Phage? Harnessing an Ancient Predator-Prey Relationship Phages are the most abundant and diverse organisms on Earth Multiple species of phage for every species of bacteria Humans evolved with phage in and on us We Can Engineer Phage Products to Combat Evolving Bacterial Pathogens Develop phage cocktails to maximize efficacy and minimize treatment resistance frequency Hybridize and synthesize traits to create super phages 7

8 8 How Phage Destroy Bacteria

9 Product Pipeline

10 Product Pipeline Indication Partner Discovery Preclinical Phase I Phase II Phase III AB-SA01 (3-Phage cocktail targeting Staphylococcus aureus) Chronic Rhinosinusitis (CRS) Wounds AB-PA01 (4-Phage cocktail targeting Pseudomonas aeruginosa) Cystic Fibrosis (CF) Expected Start H Chronic Rhinosinusitis (CRS) AB-CD01 (Clostridium difficile) C. diff Infection 10 Data expected H2 2016

11 S. aureus in Chronic Rhinosinusitis (CRS) Addressing an unmet medical need and alleviating severe economic burden Epidemiology ~30 million cases in US each year Rising prevalence Quality of Life Market Opportunity Worse than both congestive heart failure and back pain ~300,000 patients resort to invasive surgery each year Economic Burden $4.3 billion direct treatment costs Estimated annual US burden of $22 billion AB-SA01 Non-invasive Potentially - Penetrates biofilms - Relieves rhinitis 11

12 Current Phase I Trials Primary Endpoints: Safety Tolerability I. Chronic Rhinosinusitis (University of Adelaide) Days Patient Secondary Observations: Bacterial load Symptoms 2 Doses: Low Dose BID High Dose BID Primary Endpoints: Safety Tolerability 2 Doses: Low Dose QD High Dose QD Cohort 1 & 2 Completed II. Intact Skin (US Army) Days Subject Fully enrolled and no drug-related serious adverse events 12

13 Pseudomonas in Cystic Fibrosis (CF) Addressing an unmet medical need and alleviating severe economic burden Epidemiology Pseudomonas are cause of infection in ~80% of CF patients ages High overlap with CRS Quality of Life Repeated antibiotic use selects for resistant strains Recurrent infections severely compromise lung function Market Opportunity Economic Burden Mean annual costs following initial infection increased by ~$18,500 per patient AB-PA01 Demonstrated activity of over 85% against a global reference panel of CF isolates Penetrates biofilm Survives nebulization 13

14 C. difficile Infections Addressing an unmet medical need and alleviating severe economic burden Epidemiology Disease often arises after systemic antibiotic therapy; 20-40% recurrence One of the most common hospital acquired infections Quality of Life Severe diarrhea and physical pain Can limit antibiotic treatment for other conditions Market Opportunity Economic Burden $4.8B in acute care costs 250,000 hospitalizations per year (USA) 14,000 deaths per year (USA) AB-CD01 Preserves beneficial microbiome Kill and replace Adjunctive to antibiotic therapy 14

15 Company Overview

16 The AmpliPhi Team Management M. Scott Salka Chief Executive Officer Aspyrian, Ambit Steve Martin Chief Financial Officer Apricus, Stratagene Wendy Johnson ichief Operating Officer ProQuest, Salmedix Alex Gaidamaka, Ph.D., D.V.M. VP CMC Merial, Sanofi Pasteur Board of Directors Jeremy Curnock Cook Bioscience Managers Louis Drapeau InSite Vision, Nektar Mike Perry, D.V.M., Ph.D. Novartis, Bay City Capital Vijay Samant Vical, Merck Paul Grint, M.D. Regulus, Pfizer M. Scott Salka Wendy Johnson Team of ~20 in R&D and Manufacturing 16

17 Scalable Technology Platform Arsenal CRISPR Antimicrobial peptides Armed v3.0 Hybridized Traits Enhance biofilm destruction Expand cocktail footprint with fewer phage Increase efficiency of kill Engineered v2.0 Wild-Type v1.0 17

18 Establishing & Protecting the Space Patents 42 US and Foreign Patents issued covering: - Bacterial resensitization to antibiotics through phage administration - Methods to design phage cocktails Many more pending Manufacturing Proprietary Manufacturing Hosts Know-how 18

19 Synergy with Existing (sometimes ineffective) Antibiotics Patent Protection Major market protection for the sequential administration of phage and antibiotics Why might this work? Bacteria have limited degrees of freedom to out-maneuver therapies Prosecuting both biofilm-dependent and independent induced sensitivity Broad coverage in EU, Australia, Japan; US protection for Pseudomonas Penetrate biofilm, pressure to drop efflux pumps and/or plasmids 19

20 In-House cgmp-certified Bacteriophage Manufacturing Facility Ljubljana, Slovenia: ~6,000 sq. feet: - Clean rooms - QC & Process Dev - GMP storage Conducting: Fermentation (40-liter scale) Purification Aseptic fill Product QC and release Stability APHB cgmp Bioreactor 20

21 Financial Snapshot and Significant Events Cash at June 30, 2016 $7.1M As of June 30, 2016: Common stock - shares outstanding 11.1M Stock options and warrants outstanding 3.2M Fully diluted share count 14.3M Closing stock price Sept 12- NYSE MKT (APHB) $1.70/share Average daily trading volume 190,000 Biocontrol and SPH Acquisitions Scott Salka appointed as CEO $13M Financing Uplisted to NYSE MKT cgmp in Slovenia Shipped AB-SA01 & Initiated AB-PA01 Manufacturing First cohort successfully completed for CRS First-in-man dose of AB- SA01 for CRS $5M Financing Opened US IND & Initiated Phase I Study 21

22 Key Milestones S. aureus (CRS and Wound) Complete both Phase I trials* Wound: Evaluate indication following Phase I CRS: Initiate Phase II study CRS: Report Phase II Data* P. aeruginosa (CF and CRS) Optimize and scale-up manufacture of clinical trial material Complete toxicology studies CF: Initiate Phase I study CRS: Initiate Phase I study CF: Report Phase I Data* C. difficile Isolate additional lytic phages Continue preclinical studies Engineer phage & manufacturing host Initiate manufacturing Continue manufacturing 22 *Clinical Data Expected

23 AmpliPhi Biosciences Our mission is to combat the rising tide of antibiotic-resistant infections Adaptable Bacteriophage Platform Technology Radically different than traditional antibiotics Potential to selectively target any species of bacteria Clinical Stage Results from two Phase I clinical trials expected in H First phage therapy Phase II trial planned for 2017 Manufacturing Know-How In-house, dedicated cgmp phage manufacturing facility Proprietary processes are a significant barrier to entry Proven Track Record Established leadership in bringing early stage products to market Efficient use of funds to reach clinical stage 23

24 For additional information Investor Relations AmpliPhi Biosciences