CTP-543 Phase 2a Topline Results in

Patients with Moderate-to-Severe

Alopecia Areata

4 mg and 8 mg Dosing Cohorts

November 12, 2018

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2

Alopecia Areata: A Serious Medical Disease

• A devastating and poorly treated autoimmune disease

• Up to 650,000 patients affected with alopecia areata (AA) in the U.S. at any given time*

• Chronic condition affecting women, men and children of all ages

• Disease profoundly impacts patients; associated with anxiety, depression and other

autoimmune conditions

• No FDA-approved treatment options

3*Fricke M. Clinical, Cosmetic and Investigational Dermatology, 2015.

CTP-543: Phase 2a Design

• Double-blind, randomized, placebo-controlled trial in adult patients with moderate-to-

severe alopecia areata

• Entry criteria of at least 50% hair loss as measured by Severity of Alopecia Tool (SALT)

• AA patients sequentially randomized to receive one of three doses of CTP-543

(4, 8 and 12 mg twice daily) or placebo for 24 weeks

• 4 and 8 mg Cohorts completed; 12 mg Cohort is currently enrolling

• Interim top line analysis for 4 and 8 mg Cohorts

• Primary Endpoint: 50% relative reduction in SALT between Week 24

and baseline

4

SALT Scoring

Interim Analysis

Demographics

PlaceboCTP-543

4 mgCTP-543

8 mg

Randomized Population 36 30 38

Efficacy Population 35 28 38

Age: Mean (SD) 37 (12) 36 (11) 37(14)

Males, N (%) 12 (33) 8 (27) 12 (32)

Females, N (%) 24 (67) 22 (73) 26 (68)

Race: N(%)

White 27 (75) 25 (83) 26 (68)

Black or African American 5 (14) 2 (7) 7 (18)

Asian 2 (6) 2 (7) 2 (5)

Other 2 (6) 1 (3) 3 (8)5

Interim Analysis

Baseline AA Characteristics

PlaceboCTP-543

4 mgCTP-543

8 mg

Episode Duration: Yr, Mean 3.6 6 3.8

SALT score, Mean (SD) 85.0 (19.4) 88.8 (16.2) 89.1 (16.4)

AA Patchy, N(%) 19 (52.8) 16 (53.3) 16 (42.1)

AA Totalis, N(%) 5 (13.9) 2 (6.7) 6 (15.8)

AA Universalis, N(%) 12 (33.3) 12 (40.0) 14 (36.8)

AA Ophiasis, N(%) 0 (0) 0 (0) 2 (5.3)

6

Interim Analysis

Most Common Treatment Emergent Adverse Events by Patient

7

Preferred Term PlaceboCTP-543

4 mg

CTP-543

8 mg

Headache 4 (11.1%) 5 (17.2%) 10 (26.3%)

Nausea 4 (11.1%) 4 (13.8%) 4 (10.5%)

Acne 2 (5.6%) 4 (13.8%) 4 (10.5%)

Cough 0 4 (13.8%) 1 (2.6%)

Diarrhoea 3 (8.3%) 3 (10.3%) 1 (2.6%)

Nasopharyngitis 1 (2.8%) 3 (10.3%) 3 (7.9%)

Folliculitis 0 3 (10.3%) 2 (5.3%)

Blood creatine phosphokinase

increased1 (2.8%) 3 (10.3%) 2 (5.3%)

Oropharyngeal pain 0 3 (10.3%) 1 (2.6%)

Upper respiratory tract infection 6 (16.7%) 2 (6.9%) 2 (5.3%)

• No Serious Adverse Events Reported

• Only 3 Grade 3/4 hematology events; distributed equally across placebo, 4mg, and 8mg groups

Interim Analysis

Primary Analysis: Responders at Week 24

8

8.6 %

21%

47%

0

10

20

30

40

50

% P

atie

nts

pe

r T

rea

tme

nt

4 mg BIDPlacebo 8 mg BID *** P < 0.001 vs PBO

± P < 0.05 vs 4 mg

Patients with ≥ 50% Change in SALT Relative to Baseline

Interim Analysis

Responders by Visit

9

0

10

20

30

40

50

Week 4 Week 8 Week 12 Week 16 Week 20 Week 24

% P

atie

nts

pe

r T

rea

tme

nt

Placebo 4 mg BID 8 mg BID

***

Patients with ≥ 50% Change in SALT Relative to Baseline

*** P < 0.001 vs PBO

** P < 0.01 vs PBO

* P < 0.05 vs PBO

± P < 0.05 vs 4 mg

**

21%

8.6%

47%

Interim Analysis

Relative Change in SALT by Visit

10

-10

0

10

20

30

40

50

Week 4 Week 8 Week 12 Week 16 Week 20 Week 24

Me

an

% R

ela

tive

Ch

an

ge

fro

m B

aselin

e

Placebo 4 mg BID 8 mg BID

***

All Treated Patients Per Cohort

*** P < 0.001 vs PBO

* P < 0.05 vs PBO

± P < 0.05 vs 4 mg

***

***

39%

14%

7%

CTP-543 Phase 2a: Patient SALT Improvement

11

0

10

20

30

40

50

≥ 50% ≥ 75% ≥ 90%

% o

f P

atie

nts

pe

r T

rea

tme

nt

Relative Change in SALT from Baseline to Week 24

Placebo

4 mg BID

8 mg BID

Interim Analysis.

29%

16%

47%

21%

8.6%

14%

5.7%

*** P < 0.001 vs PBO

* P < 0.05 vs PBO

± P < 0.05 vs 4 mg

***

Baseline

12

Week 12 Week 24

Interim Analysis

Response Over Treatment Period

Interim Analysis

Response Over Treatment Period

13

Baseline Week 12 Week 24

Interim Analysis

Conclusion

• The primary efficacy endpoint was met

‒ 47% of patients treated with 8 mg BID of CTP-543 achieved a ≥ 50% reduction in their overall SALT

score compared to 8.6% placebo (p < 0.001)

‒ 21% of patients treated with 4 mg BID of CTP-543 achieved a ≥ 50% reduction in their overall SALT

score compared to 8.6% placebo (ns)

‒ 8 mg BID dose group was significantly different than the 4 mg BID dose group (p < 0.05)

• Significant changes in SALT score were observed starting at 12 weeks for the 8 mg cohort

(p < 0.05)

• Treatment generally well-tolerated with no serious adverse events

• Company intends to discuss complete Phase 2a results and development plan with FDA in

2019

‒ Dosing in 12 mg Cohort ongoing

14

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For additional information contact:

Justine Koenigsberg

ir@concertpharma.com

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