Targeted Therapeutics for Inflammatory Disease 2016 Jefferies Healthcare Conference
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 Year End Report on Form 10-K for the year-end ended March 31, 2016, which we filed with the Securities and Exchange Commission ( SEC ) on April 10, 2016 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
Corporate Highlights First in class drug targeting novel enzyme (SHIP1) with broad antiinflammatory potential Positive Phase 2 results in Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS) Well capitalized to advance Phase 3 program Competitive advantage in large, underserved market suitable for independent commercialization in the US, partner RoW 3
SHIP1 - Nature s Path to Suppress Inflammation Potential Next-Gen Anti-Inflammatory Drugs ü Restricted expression to hematopoietic derived cells limits off tissue toxicity PI-4,5-P 2 PI3K All cells PIP 3 SHIP1 Immune cells PI-3,4-P 2 ü Plays key role in regulating cell migration and activation PTEN All cells SHIP1 Activators ü SHIP1 activators redirect signalling, not block it Cancer Cell growth and survival Inflammation Cell activation and function ü Non-catalytic binding site imparts target selectivity and limits off target toxicity 4
A Novel First in Class Anti-Inflammatory Therapy AQX-1125, a SHIP1 activator: Broad anti-inflammatory potential Favourable ADME: Once daily oral administration T½ 21hrs Dose proportional PK, No food effect High bioavailability; Not metabolized ~60/40 Liver/Renal elimination as unchanged parent Well tolerated in 7 completed clinical trials >360 subjects dosed 5
Lead Indication: Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS): A Severe and Debilitating Inflammatory Disease Chronic erosion of the bladder lining, exposing the bladder wall to irritation and inflammation Associated with significant pain and urinary symptoms with a dramatic impact on quality of life One of the most challenging conditions known to the urology community 1) NIH-NIDDK Publica1on No. 11-3220 Sept 2011; 2) Bogart et al. 2011 Urology. 2011. 77(3):576-80; 3) Hepner et al Urology, 80(2) 280-285 6
Unmet Medical Need Estimated prevalence in the US of ~5-12M ~3.3-7.9M women 1, ~2.1-4.6M men 2 Relatively low diagnosis rate (~8%) 3 In current practice, 1:10 male to female ratio typically observed Expected to increase with improved disease definition and the emergence of new treatment options 3 Limited treatment options No new FDA approved oral treatment in 20 years 4 Other treatments are invasive (including bladder instillation) 4 1) Berry. J Urol. 2011;186:540; 2) Suskind. J Urol. 2013;189:141; 3) ClearView Analysis: Physician Interviews; 4) 2014 AUA Treatment Guidelines 7
IC/BPS Heightened Awareness 8
Phase 2 LEADERSHIP Trial: A Comprehensive IC/BPS Trial Population and Entry Demographics: 69 female patients across US and Canadian sites with moderate to severe IC/BPS symptoms: Mean pain > 5/10 Mean BPIC/SS and O Leary Sant ICSI/PI symptom scores 19 or 8 respectively On background medication (excluding opiates) Primary Endpoint: - Reduction of average daily pain at 6 weeks with once daily AQX-1125 vs. placebo Pre-specified Secondary Endpoints: - Maximum daily pain based on an 11-point NRS recorded by ediary - Average and maximum daily pain score measured by 11-point NRS recorded at clinic - Multiple urological and QoL symptom assessments (O Leary-Sant ICSI/PI, BPIC-SS) - Voiding frequency over a 24 hour period Safety, Pharmacokinetics 9
LEADERSHIP: Greater Average Daily Pain Reduction Over 6 Weeks (ediary) # Average Daily Pain (11-Point NRS, ediary) # Primary Endpoint Average Daily Pain at 6 weeks did not meet sta:s:cal significance 10
LEADERSHIP: Significantly Reduced Maximum Daily Pain Over 6 Weeks (ediary) Maximum Daily Pain (11-point NRS, ediary) 11
LEADERSHIP: Summary of Key Primary and Secondary Endpoints at 6 Weeks Endpoint (Change from Baseline at 6 weeks) Placebo (n=32) (mean change from baseline) AQX-1125 (n=37) (mean change from baseline) Difference in LSM AQX-1125 - Placebo p value PAIN (11-point NRS) Average Daily Pain (e-diary) -1.4-2.4-1.0 0.061 Maximum Daily Pain (e-diary) 1-1.4-2.6-1.3 0.030* Average Daily Pain (clinic) -1.1-2.6-1.6 0.008* Maximum Daily Pain (clinic) -1.1-2.8-1.6 0.028* SYMPTOM QUESTIONNAIRES O Leary-Sant IC Symptom Index (ICSI) -1.4-3.8-2.7 0.005* O Leary-Sant IC Problem Index (ICPI) -1.6-3.6-2.5 0.014* Combined O Leary-Sant ICSI/ICPI -3.0-7.3-5.1 0.007* BPIC-SS -4.0-8.8-5.4 0.011* VOIDING FREQUENCY Number of Voids/24h (e-diary) -0.8-3.6-2.8 0.040* 1 Primary endpoint for 301 trial *StaTsTcally Significant 12
AQX-1125: Bladder Exposure Through Blood & Urine 1000000 Concentration (ng/ml) 100000 10000 1000 100 10 LEADERSHIP, 4 Weeks, Plasma Phase 2a LPS Challenge, 7 Days, Plasma LEADERSHIP, 6 Weeks, Plasma LEADERSHIP, 4 Weeks, Urine LEADERSHIP, 6 Weeks, Urine 13
Overall Safety Summary of Oral AQX-1125 (200mg) Across Ph 2 Trials with at least 6 Weeks Daily Dosing LEADERSHIP (AQX-1125-201) IC/BPS pts (6-week dosing) COMBINED SAFETY DATA Ph2 Trials with 200mg daily dosing for 6 or 12 weeks (LEADERSHIP, KINSHIP, FLAGSHIP) Placebo N=32 n* (%) AQX-1125 N=37 n* (%) Placebo N=260 n* (%) AQX-1125 N=263 n* (%) TEAE 25 (78) 22 (60) 143 (55) 148 (56) GI Disorders 1 11 (34) 11 (30) 42 (16) 54 (21) Eye Disorders 2 3 (9) 2 (5) 25 (10) 18 (7) SAEs 0 (0) 0 (0) 18 (7) 12 (5) Deaths 0 (0) 0 (0) 1 (0.4) 1 (0.4) TEAEs Leading to DisconTnuaTon 1 (3) 2 (5) 12 (5) 15 (6) TEAEs shown are those reported from the start of dosing through to the end of the 28-day follow-up *n = number of subjects with 1 event. Subjects counted once per SOC, PT and Treatment group. 1. GI disorders are specifically included as adverse events of interest, due to their increased incidence rela^ve to other events in earlier Phase 1 clinical studies (at greater than two-fold higher doses of 450 542 mg; none of which were serious). 2. Eye disorders are specifically included as adverse events of interest since there were non-clinical ophthalmologic findings. 14
IC/BPS Commercial Opportunity 15
Untapped Market with Growth Potential 12 M Up to 12 M 5 M 4 M 2.1 M Effective therapy has potential to unlock market Men Women 3 M 2 M 1 M 3.3 M Elmiron FY sales $279 M M US Prevalence Diagnosed Treated 16
Specialist Driven Prescribing Suitable for Independent Commercialization in the US, Partner RoW Prescribing Physician Elmiron Scripts % of Scripts TOTAL 495,100 100% Urologists 239,858 48% Obstetrics/Gynecologists 97,882 20% Family Practice 33,491 7% Osteopathic Medicine 28,450 6% Nurse Practitioner 26,889 5% Internal Medicine 26,710 5% Physician Assistant 14,737 3% Specialty Unspecified 10,118 2% Source: IMS Health Report, 2010 17
IC/BPS Phase 3 Trial Timelines Ini^a^on (Q3/16) 12 Weeks Dosing with one of Two Doses of AQX-1125 or Placebo Top Line Data (Q4/17) Single Dose AQX-1125 for Safety Database 301 Treatment Phase 40 wk Open Label Extensions 302 Treatment Phase 14 wk OLE 12 Weeks Dosing with Single Dose of AQX-1125 or placebo 18
Focused Strategy with Broader Opportunities Building on the Success of LEADERSHIP with AQX-1125 Potential Development with AQX-1125 Urinary Tract IC/BPS Glomerulonephritis Prostatitis Gastrointestinal Ulcerative Colitis Crohn's Disease Eosinophilic Esophagitis 19
Prospective Near-Term Milestones Near-term data, expanded market opportunities and pipeline advancement Abstracts at EAU, AUA and CUA Initiate Mouse Carcinogenicity Study Publication of Leadership Data Initiation of IC/BPS - 301 Pivotal Trial Tablet Manufacturing for Leadership 301 Initiate ADME Trial Initiate Rat Carcinogenicity Study Safety & Other Required Phase 1 Trials ( 1 Renal, 1 Hepatic and TQT) Report ADME results IC/BPS - 301 Top-Line Data (Q4) Potential New AQX-1125 Indication 2016 2017 1 Ini:a:on and Timing Capital Dependent 20
Financial & Stock Information ~ $107.3M cash as at March 31 st, 2016 ~ $21.3M operating expenses in 2015 17.2M Shares Outstanding (~18.1M fully diluted) Cash to fund first Phase 3 trial and supporting activities NASDAQ : AQXP 21