Fourth-Quarter and Full-Year 2019 Orphan and Rheumatology Segment Results
Transformed Our R&D Organization to Deliver on Our Future Promise
Tremendous Progress in Two Years
2018
2019
2020+
•
.
Built experienced R&D leadership
team with deep drug development expertise;
agile execution and proven track record
Added leadership team with broad expertise
and capabilities
Experience across broad range
of therapeutic areas and rare diseases
• 100+ molecules developed across team
.
Opened new South San Francisco facility to
support expanding organization
Accelerated delivery on near-term
transformative priorities
including TEPEZZA development and
improvement of KRYSTEXXA profile
TEPEZZA: Accelerated development and
BLA submission; 2020 FDA approval nearly
2 months before PDUFA date
• KRYSTEXXA: Optimizing product profile
targeting response rate
• Initiated HemoShear collaboration to
discover new targeted therapies for gout
Maximizing and expanding pipeline
of early and late stage medicines
to drive sustainable growth
⚫ TEPEZZA: Evaluating additional indications,
including a new program for diffuse
cutaneous scleroderma
• KRYSTEXXA: Continuing to optimize product
•
profile and convenience
Growing pipeline through M&A
FDA: U.S. Food and Drug Administration.
BLA: Biologics License Application.
PDUFA: Prescription Drug User Fee Act.
HI HORIZON
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