Fourth-Quarter and Full-Year 2019 Orphan and Rheumatology Segment Results
Expanding Our Pipeline
Added Two Programs to Maximize Our Current Portfolio
MEDICINE / PROGRAM
KRYSTEXXA Immunomodulation
DESCRIPTION
MIRROR Randomized Controlled Trial
KRYSTEXXA Nephrology
KRYSTEXXA Shorter-Infusion Duration (1)
TEPEZZA Thyroid Eye Disease
TEPEZZA Diffuse Cutaneous Scleroderma (1)
HZN-003 Next-Gen Uncontrolled Gout
HZN-007 Next-Gen Uncontrolled Gout (2)
HemoShear Gout Discovery Collaboration
PROTECT trial in kidney transplant
patients with uncontrolled gout
Open-label trial
• OPTIC-X: Phase 3 extension trial
Exploratory trial
• Optimized uricase and optimized
PEGylation for uncontrolled gout
Optimized uricase and PASylation
for uncontrolled gout
Exploration of novel approaches
to treating gout
(1) New trial expected to begin in 2020.
(2) Being developed under a collaboration agreement with XL Protein GmbH.
MIRROR: Trials 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.
OPTIC-X: Open-label extension trial of the Phase 3 trial evaluating TEPZZA for the treatment of thyroid eye disease.
HI HORIZON
PRE-CLINICAL
PHASE 1
PHASE 2
PHASE 3
PHASE 3b/4
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