Neurogene (NGNE) 8-K Aquinox Presents at 2015 International Society for the Study of BPS Conference

Safety and Efficacy of AQX-1125 in

Interstitial Cystitis/Bladder Pain Syndrome

Results of the Phase 2 LEADERSHIP Trial

Dr. Stephen B. Shrewsbury

MD

Chief Medical Officer, Aquinox Pharmaceuticals (Canada) Inc.

September 18

, 2015

ESSIC, Rome

Exhibit 99.2

th

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 5 clinical trials completed

•

>360 subjects dosed

2

LEADERSHIP Trial: Endpoints & Selection Criteria

Primary

Endpoint:

AQX-1125

vs

placebo

on

average

daily

pain

score

utilizing

eDiaries

Secondary Endpoints: Difference in the change from baseline in:

-

Average

pain score at clinic

-

Maximum

daily pain on eDiary

-

O’Leary-Sant

Interstitial Cystitis Symptom Index and Problem Index (ICSI/PI)

-

Bladder Pain/Interstitial Cystitis Symptom Score (BPIC-SS)

-

Voiding frequency over a 24 hour period

Safety, Pharmacokinetics

Selection Criteria

•

Key Inclusion: Diagnosed>12m/

15y; +ve

cystoscopy within 36m; BPIC-SS

19 V1/2;

ICSI/PI

8 V2; Average pain

5 over 7d; 

8 voids in 24h

•

Key Exclusion: Pelvic

floor pain (

5/10); UTI in 30d;

1+ hematuria; H/o chronic

substance abuse, dependency or abuse of opiates;

intravesical

Tx

in 60d

ClinicalTrials.Gov: NCT 01882543

3

LEADERSHIP Trial: Subject Disposition

4

Screened

N=137

Randomized to

Treatment

N=69

Placebo

N=32

Discontinued, N=3

(Adverse event, N=2)

(Other, N=1)

Completed, N=29

AQX-1125

N=37

Discontinued, N=4

(Adverse event, N=2)

(Withdrew consent, N=2)

Completed, N=33

Screen Failures, N=68

(Pain score, N=19)

(Withdrew consent, N=11)

(Prohibited meds, N=10)

(UTI, N=7)

(Cystoscopy, N=4)

(Pelvic floor pain, N=3)

(IC > 15yrs, N=2)

(BMI, N=2)

(Substance abuse, N=2)

(BPIC-SS score, N=2)

(Other, N=6)

LEADERSHIP Trial: Baseline Demographics

5

Placebo

(N=32)

AQX-1125

(N=37)

Age

(years;

mean

±

SD)

53.1

(12.9)

52.1

(14.9)

Duration

of

Diagnosis

(months;

mean

±

SD)

76.3

(57.3)

63.5

(54.4)

Number

on

at

Least

One

Concomitant

Medication

for

Pain

1

(%)

22

(68.8)

22

(59.5)

-

Number on Pentosan

Polysulphate

Sodium

(%)

9

(28.1)

12

(32.4)

-

Number on Amitriptyline (%)

9

(28.1)

9

(24.3)

-

Others

14

(43.8)

13

(35.1)

Average

Daily

Pain

(mean

±

SD)

6.7

(1.01)

6.4

(0.88)

Maximum

Pain

(mean

±

SD)

7.9

(1.05)

7.6

(1.04)

O’Leary-Sant

ICSI/PI

(mean

±

SD)

30.2

(4.40)

27.3

(5.04)

-

ICSI

(mean

±

SD)

16.1

(2.87)

14.4

(2.94)

-

ICPI

(mean

±

SD)

14.1

(1.92)

12.9

(2.36)

BPIC-SS

(mean

±

SD)

31.6

(3.28)

29.6

(3.62)

Voiding

Frequency,

24h

(mean

±

SD)

20.3

(11.97)

17.0

(7.97)

1

Includes PPS, Amitriptyline and Others.  Some patients on more than one medication.

6

LEADERSHIP

Trial:

Average

Pain

(eDiary)

-

Reduced

Average

Daily Pain

(11-Point NRS,

eDiary)

BPIC-SS

Voiding Frequency (24 hrs)

Average Daily Pain (eDiary)

Average Pain (Clinic)

Maximum Daily Pain (eDiary)

O’Leary-Sant

ICSI

LEADERSHIP

Trial:

Pain

and

Symptoms

at

6

Weeks

-

Reduced

7

O’Leary Sant

ICPI

LEADERSHIP

Trial:

Responder

Rates

-

Improved

Endpoint

(at Week 6)

Reduction

AQX-1125

(N=37)

%

(n)

Placebo

(N=32)

% (n)

Proportion of

responders

AQX-1125:Placebo

1) Avg

NRS Pain

(e-Diary)

30%

51.4%

(19)

28.1% (9)

1.83

50%

29.7%

(11)

15.6%

(5)

1.90

2) Max NRS Pain

(e-Diary)

30%

46.0% (17)

25.0% (8)

1.84

50%

35.1% (13)

12.5% (4)

2.81

8

LEADERSHIP Trial: Safety & PK Summary

AEs

Placebo

N=32

AQX-1125

N=37

N (%)

N (%)

Total

AEs

25 (78.1)

19 (51.4)

GI Disorders

11 (34.4)

12 (32.4)

Eye Disorders

3 (9.4)

2 (5.4)

SAEs

0 (0.0)

0 (0.0)

Deaths

0 (0.0)

0 (0.0)

AEs Leading to

Discontinuation

2 (6.3)

2 (5.4)

*AEs by subject. Subjects counted once per SOC, PT and Treatment group

9

Treatment Emergent Adverse Events*                          Pharmacokinetics (Plasma & Urine)

10

100

1000

10000

100000

1000000