Fourth-Quarter and Full-Year 2019 Orphan and Rheumatology Segment Results
Today We Are in Our Strongest Position Ever
Key Takeaways
TEPEZZA U.S. commercial launch
underway
First and only FDA-approved medicine for
patients with thyroid eye disease
Highly experienced team working with
stakeholders since July 2019
Encouraging early launch progress
Advancing KRYSTEXXA
immunomodulation strategy
•
OMIRROR OL trial demonstrated 79 percent
of patients achieved complete response (1)
MIRROR RCT underway to potentially
expand prescribing information
·
Maximizing growth drivers through
additional R&D programs
•
KRYSTEXXA shorter-infusion trial; designed to
improve patient convenience
TEPEZZA exploratory trial in diffuse cutaneous
scleroderma
Strongest position ever
Peak U.S. annual net sales expectations for our
key growth drivers KRYSTEXXA and TEPEZZA of
>$1B each (2)
• Strong capital structure; significantly reduced
gross debt in 2019 with 0.7x net leverage; below
target of 2.0x(3)
•
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.
(1) MIRROR open-label response rate of 79 percent compared to Phase 3 blinded, placebo-controlled clinical trial data of 42 percent.
(2) Horizon estimate.
(3) Net leverage as of Dec. 31, 2019.
HORIZON
5View entire presentation