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Introduction
Frederick C. Beddingfield III, MD, PhD, FAAD
Chief Medical Officer
KYTHERA Biopharmaceuticals, Inc.
Clinical Associate Professor of Medicine,
Division of Dermatology
University of California at Los Angeles
CO-3
Disproportionate/excess
convexity or fullness
Causes
Genetics
Lifestyle
Aging
Can be resistant to
weight loss
CO-4
United States
Antifungal Amphotericin B (Amphocin®)
Influenza vaccines (Fluarix® and Flulaval®)
Ex-US
Solubilizing excipient for
phosphatidylcholine
Treatment of fat emboli
CO-8
Requested Indication
Agenda
Derek H. Jones, MD, FAAD
ATX-101 Administration
Skincare & Laser Physicians, Los Angeles
ATX-101 Administration
Derek H. Jones, MD, FAAD
Founder and Director
Skincare & Laser Physicians
Clinical Associate Professor of Medicine,
Division of Dermatology
University of California at Los Angeles
CO-14
ATX-101 Administration
Enrolled Patients
CO-18
Summary
Identical
Independent
Double-blind
Randomized
Placebo-controlled (vehicle)
All sites located in US or Canada
CO-21
Scale 0 1 2 3 4
Submental
Absent Mild Moderate Severe Extreme
Convexity
No Minimal Prominent Marked
Extreme
Localized Localized Localized Localized
Description Submental
Submental Submental Submental Submental
Convexity
Fat Evident Fat Fat Fat
Representative
Photographs
CO-26
Baseline Follow-up
Post-
platysmal
fat
Pre-
platysmal
fat
0 1 2 3 4 5 6 7 8 9 10
CO-33
Secondary Endpoint:
PR-SMFIS Total Scale Score
1. How happy are you with the appearance of your chin fat?
3. How self-conscious are you about the appearance of your chin fat?
4. How embarrassed are you about the appearance of your chin fat?
Disposition
Study 22 Study 23
Placebo ATX-101 Placebo ATX-101
ITT - Randomized (N) 250 256 258 258
Treated 99.6% 100% 99.6% 99.6%
Completed Primary Endpoint Visit 93% 91% 89% 86%
Completed Study 91% 88% 87% 83%
Discontinued from Study 9% 12% 13% 17%
Patient convenience 5% 8% 6% 6%
Lost to follow-up 2% 3% 6% 8%
Adverse event <1% <1% 1% 2%
Other* 1% <1% <1% 1%
*Administrative decision; Patient noncompliance
Cochran-Mantel-Haenszel method of imputation for missing data stratified by the site
CO-35
Efficacy
Frederick C. Beddingfield III, MD, PhD, FAAD
Chief Medical Officer
KYTHERA Biopharmaceuticals, Inc.
Clinical Associate Professor of Medicine,
Division of Dermatology
University of California at Los Angeles
CO-37
Efficacy Agenda
Secondary Endpoints:
• MRI Assessment
• Improvement in Visual and Emotional Impact (PR-SMFIS)
80%
Responder 60%
Rate
12 Weeks
After Last 40%
Treatment
p < 0.001 p < 0.001
19%
20% 13%
0% 3%
0%
N=250 N=256 N=258 N=258
Study 22 Study 23
CO-39
0%
N=250 N=256 N=258 N=258
Study 22 Study 23
CO-40
100%
80%
ATX-101
Responder ≥1 grade on CR-SMFRS
Rate
60%
≥1 grade on PR-SMFRS
(% of
Subjects)
40%
Placebo
20% ≥1 grade on CR-SMFRS
≥1 grade on PR-SMFRS
0%
Eval Eval Eval Eval Eval 4 weeks 12 weeks 24 weeks
Tx 1 Tx 2 Tx 3 Tx 4 Tx 5 after last tx after last tx after last tx
Treatment Evaluation Timepoints
Evaluations occurred 4 weeks after each treatment
CO-41
Efficacy Agenda
Secondary Endpoints:
• MRI Assessment
• Improvement in Visual and Emotional Impact (PR-SMFIS)
80%
Patients
Achieving 60% p < 0.001 p < 0.001
≥ 10% 46%
Reduction 40%
in MRI 40%
Volume
(%) 20%
5% 5%
0%
N=111 N=113 N=112 N=113
Study 22 Study 23
CO-43
8 8
Placebo Placebo
Mean 6 6.2 6 5.8
PR-SMFIS
Total
Scale 4 4
Score 3.9
3.6
ATX-101
2 ATX-101 2
No 0 0
Impact Baseline 12 Wks after Baseline 12 Wks after
Last Tx Last Tx
Efficacy Agenda
Secondary Endpoints:
• MRI Assessment
• Improvement in Visual and Emotional Impact (PR-SMFIS)
Non-Responder
Age 44
Baseline
BMI 27.7 kg/m2
Weight 179 lbs
CR-SMFRS Moderate (2)
PR-SMFRS Moderate (2)
Satisfaction Dissatisfied (1)
Conclusions
Safety
Paul F. Lizzul, MD, PhD, FAAD
Senior Medical Director
KYTHERA Biopharmaceuticals, Inc.
Private Practice, Westlake Village, CA
CO-50
Safety Agenda
Overview of SAEs
Safety Agenda
Overview of SAEs
Severe 21 1% 90 2%
Patients who experienced 1 or more adverse events are counted for the worst, or maximum, severity.
CO-56
P = Placebo; N = 504
100%
A = ATX-101; N = 515
80%
Mild
60% Moderate
%
of Severe
Patients 40%
20%
0%
P A P A P A P A P A P A P A P A P A P A
CO-58
Severity of Events
Severe 21 1% 90* 2%
*N=55 patients
Patients who experienced 1 or more adverse events are counted for the worst, or maximum, severity.
CO-59
Safety Agenda
Overview of SAEs
Safety Agenda
Overview of SAEs
Safety Agenda
Overview of SAEs
No Unexpected Findings in
Long-Term Post-Treatment Safety
* 2 mg/cm2
CO-69
Education/Training
and Summary
Frederick C. Beddingfield III, MD, PhD, FAAD
Chief Medical Officer
KYTHERA Biopharmaceuticals, Inc.
Clinical Associate Professor of Medicine,
Division of Dermatology
University of California at Los Angeles
CO-71
74
CO-75
ATX-101 Benefit-risk