Investor Presentaiton
Top-tier talent
Differentiated process
Scalable platform
Adding unmatched portfolio breadth over the last two years
21 products - 25 diseases (1)
Inflammatory
bowel disease
Entyvio
Takeda
Approved
Infectious
disease
PREVYMIS
MERCK
Hereditary
angioedema
orladeyo (3)
bio cryst
AML, MDS
Migraine
Cystic fibrosis
IDHIFA
Bristol Myers Squibb™
Nurtec'ODT
biohaven
trikafta
☐ pharmaceuticals
VERTEX
Pre-approval
Roche
Evrysdi
2020
biohaven
☐ pharmaceuticals
SMA
zavegepant
Type 1
hyperoxaluria
OXLUMO
Solid tumors
2
Alnylam
PsO, PsA, IBD
CABOMETYX®
EXELIXIS
Takeda
IPSEN
Johnson & Johnson
seltorexant
Depression
Tremfya
Johnson & Johnson
2021
Roche
gsk
otilimab
RA
Hereditary
angioedema
orladeyo (3)
bio
cryst
Constellati n
PHARMACEUTICALS
bio cryst
(3)
BCX9930
PNH, others
Migraine
bio
cryst
(3)
BCX9930
PNH, others
gantenerumab
Alzheimer's
CPI-0209
Liquid tumors
AML: acute myeloid leukemia; MDS: myelodysplastic syndromes; PSO: plaque psoriasis; PSA; psoriatic arthritis; IBD: inflammatory bowel disease; SMA: spinal muscular atrophy; PNH:
paroxysmal nocturnal hemoglobinuria; RA: rheumatoid arthritis
pelabresib
Myelofibrosis
ROYALTY PHARMA 1. Includes January 2022 aficamten royalty acquisition, which is not shown here. 2. Other products included in cystic fibrosis deal not shown include Symdeko, Orkambi and Kalydeco. 3.
Purchase of incremental royalties on Orladeyo and BCX9930 in November 2021 is not included in product/disease total.
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