Targeted Therapeutics for Inflammatory Disease R&D Day

Targeted Therapeutics for

Inflammatory Disease

R&D Day October 7, 2015

Forward Looking Statements / Safe Harbor

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,

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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

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Risk Factors set forth in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2015, which we filed with the Securities and Exchange Commission ( SEC ) on August 6, 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

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Registration & Breakfast

8:30AM Welcome Remarks David Main, President & CEO

8:35AM BPS/IC Prevalence, Diagnosis & Treatment

Dr. Robert Evans, M.D.

Associate Professor of Urology at Wake Forest Baptist Health University

Clinical Instructor at Wake Forest School of Medicine Department of Urology

9:05AM LEADERSHIP Trial Review and Clinical Update

Dr. Stephen Shrewsbury, M.D.

Senior VP of Clinical Development and CMO

Commercial Opportunity David Main 9:35AM Q&A Panel & Open Discussion 10:00AM Event Concludes

Focused Strategy and Defined Path

pivotal trials Competitive advantage Well capitalized Broad opportunity Independent commercialization

Experienced Management

INEX Pharmaceuticals, QLT

Stephen Shrewsbury, CMO

Sarepta, MAP, Chiron, Glaxo

Angiotech, AnorMED, PwC

Lloyd Mackenzie, VP Technical Ops

David Mitchell, VP Global Regulatory Affairs & QA

AbbVie, Biogen Idec, Bayer Schering

Key Near-Term Milestones

Near-term data, expanded

market opportunities and pipeline advancement

ESSIC Meeting (Sept 18, 2015)

R&D Day Complete (Q2/15) (Oct 7, 2015)

LEADERSHIP Top- Initiation of BPS/IC 301 Pivotal

Line Results (Q3/15) Trial

KINSHIP Top-Line KINSHIP Full Results: Meeting

Secondary Results Next

Generation Lead Selection (Q3/15) ? 2015 2016

KINSHIP: Potential Relief of Atopic Dermatitis Symptoms

Evaluate AQX-1125 in subjects with

~50 mild to moderate patients from Canadian sites

Primary: Effect of AQX-1125 (200 mg capsule, oral once daily) vs. placebo on change from baseline in Total Lesion Symptom Score (TLSS) after 12 weeks of treatment

Secondary: AD symptoms, safety, PK

First Patient Q4 2014

Top?line data Q4 2015 (Enrollment Complete) Full data

At Scientific Meeting TBD

Associate Professor of

Wake Forest Baptist Medical School

Associate Professor of

Urology, Wake Forest Baptist Medical School

Jefferson Medical College MD

Private Urology practice ~ 20 years

Associate Professor of Urology 6 years, NC

Co-Director, Continence & Pelvic Pain Center, NC

ICA Physician Award, 2000

GAG Society Urologist of the Year, 2004 & 2008

Key urology publications, 15 since 2002

multicenter urology RCTs in last 2 years

NIDDK/MAPP research (pending) NIH 2015-17

Dr. Stephen Shrewsbury

A Novel First in Class Anti-Inflammatory Therapy

AQX-1125, a SHIP1 activator:

Broad anti-inflammatory potential

Once daily oral administration

Dose proportional PK, No food effect

bioavailability; Not metabolized ~60/40 Liver/Renal elimination as unchanged parent

completed clinical trials

SHIP1 Nature s Path to Resolve Inflammation

Potential Next-Gen Anti-Inflammatory Drugs

PI-4,5-P PIP SHIP1 PI-3,4-P

Cell growth and Cell activation survival and function

Preclinical Validation of SHIP1 Target

Demonstrated activity at mucosal surfaces such as Airway, GI, Bladder

Lungs SHIP1 Knockout (KO) Mouse Model

Increased GM progenitors

Infiltration of lungs with Wild KO macrophages/neutrophils Type Airway remodeling/fibrosis

KO mouse is viable & fertile, ~40% survival by 14 weeks Mixed inflammatory infiltrates

Granuloma Colitis phenotype

Inflammation in IC/BPS

distribution and anti-inflammatory properties are compelling

AQX-1125 inhibits mast cell degranulation,

inflammatory processes, vascular leakage, and tissue remodelling

AQX-1125 may be disease modifying through

reduction of bladder inflammation and inhibition of subsequent injury

Source: GlobalData, Cleveland Clinic

Journal of Medicine, 2012

Bladder Insult More Injury Epithelial Layer Damage Pentosan Polysulphate Sodium

Mast Cell Activation and Histamine Release Potassium Leakage into Interstitium

Activation of C-Fibres and Release of Substance P

Antihistamine Therapy Tricyclic Antidepresent (TCA) Therapy

AQX-1125 Inhibits Cyclophosphamide-Induced