Fourth-Quarter and Full-Year 2019 Orphan and Rheumatology Segment Results
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TEPEZZA: New Development Program Evaluating Diffuse Cutaneous Scleroderma
No Approved Therapies for this Rare Autoimmune Disease
Diffuse Cutaneous Scleroderma
Chronic autoimmune disease marked by
fibrosis, including hardening of skin and
internal organ involvement
Rare disease with no approved or
effective treatments
Patients typically suffer extensive fibrosis
that can progress to internal organ
damage
Primarily managed by rheumatologists
(1) Horizon estimate.
IGF-1R: Insulin-like growth factor 1 receptor.
HORIZON
.
BED
Epidemiology
☑
Scientific Rationale
~100K patients in U.S. diagnosed with
scleroderma (1)
Of U.S. diagnosed patients, ~30K patients
are diagnosed with diffuse cutaneous
scleroderma (1)
Evaluating efficacy based on TEPEZZA
mechanism of action, which is to block
IGF-1R
Literature suggests that targeting IGF-1R
could have an impact on the fibrotic
process
Initiating exploratory trial to evaluate
objective biomarker and clinical
endpoints
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