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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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)
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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
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For additional information contact:
Justine Koenigsberg
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