Fourth-Quarter and Full-Year 2019 Orphan and Rheumatology Segment Results
A Leading, High-Growth Biopharmaceutical Company
Executing on Our Strategy
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•
Maximizing the value of our key
growth drivers KRYSTEXXA and
TEPEZZA
Expanding our pipeline for
sustainable growth
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Significant Progress in 2019
Announced impressive TEPEZZA
Phase 3 data; submitted BLA
12-0 FDA Advisory Committee
vote for TEPEZZA
Invested in TEPEZZA pre-launch
activities
Completed KRYSTEXXA MIRROR
OL trial
Initiated KRYSTEXXA MIRROR RCT
Initiated KRYSTEXXA PROTECT
trial
Significantly strengthened capital
structure
Opened new South San Francisco
R&D and manufacturing facility
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.
Progress and Expected
Milestones in 2020
TEPEZZA Jan. 21 approval;
continued launch execution
Advance KRYSTEXXA
immunomodulation strategy:
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Announced 79% complete
response rate in MIRROR OL
Complete MIRROR RCT
enrollment
Complete KRYSTEXXA PROTECT
trial enrollment
Initiate KRYSTEXXA shorter-
infusion duration trial
Initiate TEPEZZA diffuse cutaneous
scleroderma exploratory trial
TEPEZZA permanent J-code Oct. 1
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Expected Milestones
Beyond 2020
New KRYSTEXXA data
•
readouts:
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MIRROR RCT
PROTECT
Advancing toward peak U.S.
net sales expectations:
KRYSTEXXA: >$1B (1)
TEPEZZA: >$1B(1)
Pre-clinical gout candidates
advance into clinical
development
Potential upside from future
pipeline assets
BLA: Biologics License Application.
FDA: U.S. Food and Drug Administration.
MIRROR OL: Open-label initial trial with 14 enrolled patients evaluating the use of KRYSTEXXA in combination with methotrexate to increase the response rate.
MIRROR RCT: Registrational, randomized, placebo-controlled 135-patient trial evaluating the use of KRYSTEXXA in combination with methotrexate to increase the response rate.
PROTECT: Clinical trial evaluating the effect of KRYSTEXXA on serum uric acid levels in kidney transplant patients with uncontrolled gout.
(1) Horizon estimate.
HORIZON
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