Targeted Therapeutics for Inflammatory Disease Jefferies Global Healthcare Conference June 2015
Forward Looking Statements / Safe Harbor This presentation and the accompanying oral commentary contain forward-looking statements that involve substantial risks and uncertainties. All statements other than statements of historical facts contained in this presentation and the accompanying oral commentary, including statements regarding our future financial condition, business strategy and plans and objectives of management for future operations, are forward looking statements. In some cases, you can identify forward-looking statements by terminology such as believe, will, may, estimate, continue, anticipate, intend, should, plan, might, approximately, expect, predict, could, potentially or the negative of these terms or other similar expressions. Forward looking statements appear in a number of places throughout this presentation and the accompanying oral commentary and include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things, our ongoing and planned preclinical development and clinical trials, the timing of and our ability to make regulatory filings and obtain and maintain regulatory approvals for AQX-1125 and our future product candidates, our intellectual property position, the degree of clinical utility of AQX-1125 and our future product candidates, particularly in specific patient populations, our ability to develop commercial functions, expectations regarding clinical trial data, our results of operations, cash needs, financial condition, liquidity, prospects, growth and strategies, the industry in which we operate and the trends that may affect the industry or us. Forward-looking statements involve known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. In evaluating these statements, you should specifically consider various factors, including the risks outlined under the caption Risk Factors set forth in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2015, which we filed with the Securities and Exchange Commission ( SEC ) on May 12, 2015 and other reports and filings we will make with the SEC from time to time. Forward-looking statements represent our management s beliefs and assumptions only as of the date of this presentation. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. 2
A Novel Class of Anti-Inflammatory Therapies AQX-1125, lead SHIP1 activator: Broad anti-inflammatory potential Positive Preclinical and Clinical PoC Ongoing Phase 2 trials: FLAGSHIP (COPD Exacerbations) Top Line Mid-2015 LEADERSHIP (BPS/IC) Top Line Mid-2015 KINSHIP (Atopic Dermatitis) by Q1 2016 Worldwide rights and patent coverage to 2028 Diverse library of next-generation compounds targeting SHIP1 3
Experienced Management David Main, President & CEO INEX Pharmaceuticals, QLT Stephen Shrewsbury, CMO Sarepta, MAP, Chiron, Glaxo Kamran Alam, CFO Angiotech, AnorMED, PwC Lloyd Mackenzie, VP Technical Ops QLT, Inflazyme David Mitchell, VP Global Regulatory Affairs & QA AbbVie, Biogen Idec, Bayer Schering 4
Well Capitalized with Large Pharma Investment ~$34.4M cash on hand as at March 31 st, 2015 Cash to mid-2016 and data from all 3 Phase 2 trials. Backed by Large Pharma Investment: Analyst coverage: 5
SHIP1 - Nature s Path to Resolve Inflammation Potential Next-Gen Anti-Inflammatory Drugs PI3K All cells PI-4,5-P 2 PIP 3 SHIP1 Immune cells PI-3,4-P 2 PTEN All cells SHIP1 Activators Cancer Cell growth and survival Inflammation Cell activation and function 6
Focused Strategy & Broad Opportunities Phase 2 trials supported by preclinical and clinical PoC Mucosal Inflamma-on Unmet Need Short Dura-on Trials Acute Endpoints Exacerba-ons, Flares, Pain Expand into Chronic Endpoints Lung/Airway COPD Exacerbations (Moderate-Severe) Asthma Non-CF Bronchiectasis Churg-Strauss Syndrome Idiopathic Pulmonary Fibrosis Chronic Rhinosinusitis Epidermal Atopic Dermatitis Urinary Tract BPS/IC Glomerulonephritis Gastrointestinal Eosinophilic Esophagitis Crohn's Disease Ulcerative Colitis 7
Clinical Development in Large, Underserved Markets Research/Preclinical Phase 1 Phase 2 AQX-1125 SHIP1 Activator Next Gen SHIP1 Activator Completed Ongoing Future SAD / MAD / FE Chronic Obstructive Pulmonary Disease (COPD) PoC Asthma PoC Chronic Obstructive Pulmonary Disease Exacerbations (COPD) Bladder Pain Syndrome/Interstitial Cystitis (BPS/IC) Atopic Dermatitis Other Respiratory, Urology, Gastrointestinal Inflammation Immuno-oncology 8
AQX-1125 Clinical Program 9
Desirable Once Daily, Oral Pharmacokinetics Single Ascending Dose PK Concentration (ng/ml) 10000.0 1000.0 100.0 10.0 1.0 542 mg 400 mg 200 mg 100 mg 50 mg 17 mg t 1/2 =~21 h 0.1 0 20 40 60 80 100 Time (h) 10
Phase 2a Proof of Concept Achieved COPD Two different challenge models for multiple inflammatory diseases LPS challenge; model of the inflammatory mechanisms seen in COPD Cross-over trial; 40 volunteers, once daily x 7 Sputum neutrophils, p<0.1; trend analysis other cell counts Asthma Allergen challenge; model of inflammatory mechanisms in allergic response Cross-over trial; 22 mild asthmatics, once daily x 7 Late Asthmatic Response (LAR), p<0.05; trend analysis for sputum cell counts 11
COPD PoC: AQX-1125 Inhibits Sputum Neutrophils AQX-1125 met primary endpoint in healthy volunteers CI (2.25-8.12) CI (0.82-3.16) 12
COPD PoC: AQX-1125 Compares Favorably Robust Inhibition of Inflammation Drug Class % Neutrophil Inhibition AQX-1125 SHIP1 activator 62% Ph-797804 p38 MAP kinase Inhibitor 50% Roflumilast (Daxas, Daliresp) PDE4 inhibitor 39% Prednisolone / Fluticasone Corticosteroids No effect 13
Current AQX-1125 Phase 2 Development 14
COPD Exacerbations Remain Major Problem AQX-1125 s properties and tolerability provide competitive differentiation Approx. 600 million COPD cases globally with 65 200 million moderate to severe. 25 million affected in the US. Despite numerous therapies, majority of moderate to severe patients still suffer exacerbations High unmet need for effective oral anti-inflammatories that reduce exacerbations, decrease hospitalization and slow/prevent disease progression 15
FLAGSHIP: Targeting Exacerbations Objective: Evaluate AQX-1125 in COPD patients post exacerbation ~400 moderate to severe COPD patients from EU, AUS NZ and USA IND Accepted by FDA in June, 2014 Endpoints: Primary: Sized to detect effect of AQX-1125 (200 mg capsule q.d.) vs. placebo on recurrent COPD exacerbations over 12 weeks (reduction of AAC for EXACT-PRO*) utilizing ediaries Secondary: CAT, PFT, safety, PK, healthcare utilization (rescue meds / hospitalization) Key Milestones: First Pa2ent Top- line data Full data Q4 2013 Mid- 2015 (Enrollment Complete) At Scien2fic Mee2ng TBD *EXACT- PRO= EXAcerba2ons of Chronic obstruc2ve pulmonary disease Tool Pa2ent Reported Outcome 16
EXACT: Comprehensive COPD Symptom Assessment Offers robust and data rich evaluation of response to therapy Developed by Specialists in Outcome Measurement & Pharma Consortium AZ, Almirall, Bayer, BI, Forest, GSK, Merck, Novartis, Ortho-McNeil, Pfizer, Sepracor FDA guidance received January 2014; EMA guidance received April 2015 Designed to standardize the method for evaluating the frequency, severity, and duration of acute exacerbations of COPD Patient reported outcome (PRO) daily questionnaire utilizing ediaries Developed with considerable regulatory consultation (FDA and EMA) 17
BPS/IC: Getting to the Source of the Pain AQX-1125 s distribution and anti-inflammatory properties compelling Up to 14 million may be affected in the US Disease driven by chronic inflammation and pain following damage to bladder lining Common treatment approach: direct instillation for short-term relief of symptoms Oral administration of AQX-1125 results in bladder exposure systemically and through urine. 18
LEADERSHIP: Potential Once Daily, Oral Pain Relief Objective: Evaluate AQX-1125 in subjects with BPS/IC pain ~70 moderate to severe patients from Canadian and US sites IND Accepted by FDA in May, 2014 Endpoints: Primary: Sized to detect effect of AQX-1125 (200 mg capsule once daily) vs. placebo on reduction of mean pain score (11 point NRS*) at 6 weeks vs. baseline utilizing ediaries Secondary: urinary symptoms, safety, PK, QOL Key Milestones: First Pa2ent Top- line data Full data Q3 2013 Mid- 2015 (Enrollment Complete) At Scien2fic Mee2ng TBD *NRS= Numerical Ra2ng Scale 19
A Novel Approach to Atopic Dermatitis Link between SHIP1 and dermatitis creates opportunity for AQX-1125 Estimated 17.8 million affected with all forms of AD in the US (2/3 with moderate to severe form). Global prevalence of AD varies between 1%-20%. Disease characterized by excessive dermal immune cell activation and mobilization. Severe AD (11% of AD population) treated with corticosteroids, cytotoxics and immunosuppressants not suitable for long term treatment. Large market and limited competition for oral, well tolerated treatment. 20
KINSHIP: Potential Oral Relief of AD Symptoms Objective: Evaluate AQX-1125 in subjects with mild to moderate AD ~50 mild to moderate patients from Canadian sites Endpoints: Primary: Sized to detect effect of AQX-1125 (200 mg capsule once daily) vs. placebo on change from baseline in Total Lesion Symptom Score (TLSS) after 12 weeks of treatment Secondary: AD symptoms, safety, PK Key Milestones: First Pa2ent Top- line data Full data Q4 2014 Q1 2016 (Enrollment Complete) At Scien2fic Mee2ng TBD 21
Key Milestones: Near-term data, expanded market opportunities and pipeline advancement COPD Exacerbations First patient: Q4 2013 (Enrollment Complete) Top-line Data: Mid-2015 Scientific Meeting: TBD Atopic Dermatitis: First patient: Q4 2014 (Enrollment Complete) Top-line Data: Q1 2016 Scientific Meeting: TBD BPS/IC First patient: Q3 2013 (Enrollment Complete) Top-line Data: Mid-2015 Scientific Meeting: TBD Additional AQX-1125 Trials: CRS Trial Initiated: 2015 Next Generation Compounds: Lead Selection: H2 2015 22
Targeted Therapeutics for Inflammatory Disease